Mucopolysaccharidosis (MPS)
Conditions
Keywords
Mucopolysaccharidosis (MPS) IIIA, Recombinant Human Heparan N-Sulfatase (rhHNS), Sanfilippo Syndrome Type A (MPS IIIA), Lysosomal Storage Disease (LSD), Intrathecal Drug Delivery Device (IDDD)
Brief summary
Sanfilippo syndrome, or Mucopolysaccharidosis (MPS) III, is a rare lysosomal storage disease (LSD) caused by loss in activity of 1 of 4 enzymes necessary for degradation of the glycosaminoglycan (GAG) heparan sulfate (HS) in lysosomes. MPS IIIA results from deficiency of the enzyme heparan N-sulfatase (sulfamidase). MPS IIIA symptoms arise on average at 7 months of age, with the average age of diagnosis at 4.5 years for the majority of patients. The central nervous system (CNS) is the most severely affected organ system in patients with MPS IIIA, evidenced by deficits in language development, motor skills, and intellectual development. In addition, there are abnormal behaviors including but not limited to aggression and excess motor activity/hyperactivity that contribute to disturbances in sleep.Overall, individuals with MPS IIIA have a marked developmental delay and significantly reduced lifespan of 15 years of age on average. The purpose of this study is to determine the safety and tolerability of rhHNS via ascending doses administered via an a surgically implanted intrathecal drug delivery device (IDDD) intrathecal (IT) route once monthly (or every two weeks) for 6 months in patients with MPS IIIA.
Detailed description
No effective, disease-modifying therapies are currently approved as treatments for this devastating and disabling disease. Shire Human Genetic Therapies (Shire HGT) is developing a sulfamidase enzyme replacement therapy (ERT)rhHNS for patients with MPS IIIA. recombinant human heparan-N-sulfatase (rhHNS) is being administered into the cerebrospinal fluid (CSF) via an surgically implanted intrathecal drug delivery device (IDDD), because when administered intravenously (IV) it does not cross the blood brain barrier (BBB). This study is a multicenter, multiple-dose, dose escalation study designed to evaluate the safety, tolerability, and clinical activity of up to 3 dose levels (10mg,45mg and 90mg monthly for 6 months) of rhHNS administered via an IDDD in patients with Sanfilippo syndrome Type A ages greater than or equal to 3 years of age. Patients who have completed all study requirements in this study will be invited to participate in an open-label extension study that will be designed to evaluate long term safety and clinical outcomes of intrathecal administration of rhHNS.
Interventions
BIOLOGICALRecombinant human heparan N-sulfatase (rhHNS)
Sponsors
Shire
Study design
Allocation
RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
3 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Each patient had to meet the following criteria to be eligible for the study: 1. a.) Patients had a documented deficiency in sulfamidase enzyme activity of ≤10% of the lower limit of the normal range as measured in fibroblasts or leukocytes. AND either b or c b.) Patients had a normal enzyme activity level of at least 1 other sulfatase (to rule out multiple sulfatase deficiency) as measured in fibroblasts or leukocytes. c.) Patients had 2 documented mutations. 2. The patient was ≥3 years of age and had a developmental age ≥1 year. 3. Patients must have been medically stable, in the opinion of the Investigator, to accommodate the protocol requirements, including travel, assessments, and IDDD surgery, without placing an undue burden on the patient/patient's family. 4. The patient's parent(s) or legal guardian must have voluntarily signed an Institutional Review Board/Independent Ethics Committee-approved informed consent form after all relevant aspects of the study have been explained and discussed with the patient, the patient's parent(s), or legal guardian. The patients, patient's parents or legal guardian's consent and patient's assent as appropriate, must have been obtained.
Exclusion criteria
Patients who met any of the following criteria were excluded from the study: 1. The patient had significant non-MPS IIIA related CNS impairment or behavioral disturbances that would confound the scientific integrity or interpretation of study assessments, as determined by the investigator. 2. The patient had MPS IIIA behavioral-related issues, as determined by the investigator, that would have precluded performance of study neurocognitive and developmental testing procedures 3. The patient was pregnant, breast feeding, or was a female patient of childbearing potential who would not or could not comply with the use of an acceptable method of birth control such as condoms, barrier method, oral contraception, etc. 4. The patient was blind and/or deaf. 5. The patient had any known or suspected hypersensitivity to anesthesia or was thought to have an unacceptably high risk for anesthesia due to airway compromise or other conditions. 6. The patient or the patient's family had a history of neuroleptic malignant syndrome, malignant hyperthermia, or other anesthesia-related concerns. 7. The investigator may have chosen to exclude patients who have had complications resulting from prior lumbar punctures. 8. The patient had a CNS shunt. 9. The patient had skeletomuscular/spinal abnormalities or other contraindications for the surgical implantation of the IDDD. 10. The patient had a history of poorly controlled seizure disorder. 11. The patient was currently receiving psychotropic or other medications, which in the investigator's opinion, would have been likely to substantially confound test results and the dose and regimen of which cannot be kept constant throughout the study. 12. The patient could not sustain absence from aspirin, non-steroidal medications, or medications that affected blood clotting within 1 week prior to a relevant study related procedure (eg, device implantation if applicable), or had ingested such medications within 1 week before any procedures in which any change in clotting activity would have been deleterious. 13. The patient had received treatment with any investigational drug or a device intended as a treatment for MPS IIIA within the 30 days prior to, or during the study, or was enrolled in another study that involved an investigational drug or device (screening through safety follow-up contact). 14. The patient received a hematopoietic stem cell or bone marrow transplant. 15. The patient's parent(s), or patient's legal guardian(s) was/were unable to provide consent or the patient could not provide assent, as appropriate, due to, but not limited to, the inability to understand the nature, scope, and possible consequences of the study, or did not agree to comply with the protocol defined schedule of assessments.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Treatment Emergent Serious Adverse Events (SAE) | Baseline to week 30 (follow-up) | An adverse event (AE) was defined as any untoward medical occurrence in a clinical investigation participant administered as a pharmaceutical product that did not necessarily have a causal relationship with this treatment. A serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged in-patient hospitalization; life threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent AEs (TEAEs) were defined as all adverse events (AEs) from the time of the surgery for IDDD implantation to the last follow up contact, 30 (±7) days after the end of study (EOS) procedures. |
| Number of Treatment Emergent Adverse Events (TEAE) | Baseline to week 30 (follow-up) | An adverse event (AE) was defined as any untoward medical occurrence in a clinical investigation participant administered as a pharmaceutical product that did not necessarily have a causal relationship with this treatment. A serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged in-patient hospitalization; life threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent AEs (TEAEs) were defined as all adverse events (AEs) from the time of the surgery for IDDD implantation to the last follow up contact, 30 (±7) days after the end of study (EOS) procedures. |
| Summary of Anti-rhHNS Antibody Status in Cerebrospinal Fluid (CSF) by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Baseline, Week 26 | Participants with positive, negative and missing status were reported. |
| Summary of Anti-rhHNS Antibody Status in Serum by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Baseline, Week 26 | Participants with positive, negative and missing status were reported. |
| Number of Participants With Intrathecal Drug Device (IDDD) Failures at Week 26 | Week 26 | Participants with IDDD failures were reported. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | Baseline, Week 22, Week 26/EOS | CHQ-PF50 which was designed to measure the physical and psychosocial well-being of children 5 years to 18 years of age, consists of 13 health concepts including 11 multi-item and 2 single item scales: Physical Function (PF), Role/Social-Emotional/Behavioral (REB), Role/Social-Physical (RP), bodily pain (BP), General Behavior (BE), Mental Health (MH), Self Esteem (SE), General Health Perceptions (GH), Change in Health (CH), Parental Impact-Emotional (PE), Parental Impact-Time (PT), Family Activities (FA), and Family Cohesion (FC). Transformed scores for all subscales range from 0 to 100, with a higher score indicating better health. Physical and Psychosocial Summary measures (SM) were scored with the use of norm-based methods that standardize the scores to a mean (± Standard Deviation) of 50 ± 10 on the basis of an assessment of the general United States population. Higher values represent better health. |
| Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | Baseline, Week 22, Week 26/EOS | ITQOL is a generic, validated health status measure for children aged 2 months up to 5 years, including items and scales to measure aspects of physical functioning, development, pain, mood, behavior, general health and impact on parents. The ITQOL consists of 97 items that are scored, summed, and transformed on a scale from 0 (worst health) to 100 (best health). The score range for all individual subscales is 0 (worst health) to 100 (best health). Abbreviation: Overall Health (OH), Physical Abilities (PA), Growth And Development (GAD), Bodily Pain (BP), Temperament And Moods (TAM), General Behavior (GEB), Global Behavior (GLB), Getting Along (GA), General Health Perceptions (GHP), PI-Emotion (PIE), PI-Time (PIT), Family Cohesion (FC). |
| Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Child Form 87 (CHQ-CF87) at Week 26 (EOS) | Baseline, Week 26/EOS | CHQ-CF87 form was designed to be a self-report for participants 10 years and older. It consists of 87 questions and contains the same scales as the PF-50, (with the omission of the parental impact scales and there are no psychosocial and physical summary scores derived). As per statistical analysis plan, the outcome was to be assessed only if greater than (\>) 50 percent (%) of participants were available for the evaluation. |
| Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | Baseline, Week 22, Week 26/EOS | Children's sleep habits rating scale consisting of 35 item parent questionnaire sleep screening tool. Items are scored on a 3-point scale from 1 (rarely) to 3 (usually) with a Total Sleep Disturbance score (TSDS) ranging from 35-105. Eight subscale scores are totaled to create the TSDS. The subscales are: Bedtime Resistance (BR)(6 items, score = 6-18), Sleep Duration (SD)(3 items, score = 3-9), Parasomnias (P)(7 items, score = 7-21), Sleep Disordered Breathing (SDB)(3 items, score = 3-9), Night Waking (NW)(3 items, score = 3-9), Daytime Sleepiness (DS)(8 items, score = 8-24), Sleep Anxiety (SA)(4 items, score = 4-12), and Sleep Onset Delay (SOD)(1 item, score = 1-3). The questionnaire was designed for children aged 4 through 12 years. A higher score is indicative of more disturbed sleep. |
| Number of Participants With Accumulation of Recombinant Human Heparan N-Sulfatase (rhHNS) in Cerebrospinal Fluid (CSF) at Week 22 | Baseline, Week 22 | Cerebrospinal fluid samples were collected from participants through an implanted IDDD or via lumbar puncture (LP) immediately prior to each administration of HGT-1410. |
| Change From Baseline in Concentration of Heparan Sulfate and Heparan Sulfate Derivatives in Cerebrospinal Fluid (CSF) at Week 6, 10, 14, 18, 22 and 26(EOS) | Baseline, Week 6, 10, 14, 18, 22 and 26(EOS) | Levels of heparan sulfate and its derivatives were evaluated using the proprietary Sensi-Pro (SP) high-performance liquid chromatography (HPLC) based assay. Abbreviation: SP Total Heparan Sulfate (SPTHS), SP Non-Reducing End Assay (SPNREA), |
| Change From Baseline in Brain Magnetic Resonance Imaging (MRI) at Week 22 | Baseline, Week 22 | Brain MRI was measured for grey matter volume (GMV) , white matter volume (WMV) and Intracranial cerebrospinal fluid Volume (ICSFV) (Ventricles + Additional CSF Space). |
| Change From Baseline in Developmental Quotient (DQ) Using Bayley Scales of Infant Development Third Edition (BSID III) and Kaufman Assessment Battery for Children Second Edition (KABC II) at Week 22 | Baseline, Week 22 | BSID-III was used to assess the motor (fine and gross), language (receptive and expressive), and cognitive development of infants and toddlers. This measure consists of a series of developmental play tasks. Raw scores of successfully completed items are converted to scale scores and to composite scores. The mean composite score is 100 and the standard deviation (SD) is 15.Higher scores are indicative of decreased development. KABC-II was an individually administered measure of the processing and reasoning abilities of children and adolescents between the ages of 3 and 18 years and is an alternative to BSID-III. BSID-III DQ score was based on the Cognitive domain. The DQ score was calculated from the data obtained from either BSID-III/KABC-II mental age equivalent of the child in months divided by the calendar age in months (multiplied by 100 to give percentage points). |
| Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | Baseline, Week 22 | ABR assessments were to be conducted under anesthesia and measured the electrical response evoked by acoustic stimuli as sound is processed along the auditory pathway. The Inter-peak Latencies (IPL) were calculated by subtracting the absolute latencies (AL). The Inter-aural Latencies (IAL) were calculated by subtracting the absolute wave V latencies of the right and left ear. IAL, IPL and AL were reported. Abbreviation: Right Ear (RE), Left Ear (LE), Wave (W), Wave V (WV) |
| Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | Baseline, Week 22 | ABR assessments were to be conducted under anesthesia and measured the electrical response evoked by acoustic stimuli as sound is processed along the auditory pathway. Data for change from baseline in ABR amplitudes by left ear (LE) and right ear (RE) were reported. |
| Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | Baseline, Week 22 | ABR assessments were to be conducted under anesthesia and measured the electrical response evoked by acoustic stimuli as sound is processed along the auditory pathway. Data for change from baseline in ABR amplitudes(A), log-transformed amplitudes (LTA), square-root transformed amplitudes (STA) by left ear (LE) and right ear (RE) wave V/I in ratio was reported. |
| Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | Baseline, Week 22 | ABR assessments were to be conducted under anesthesia and measured the electrical response evoked by acoustic stimuli as sound is processed along the auditory pathway. Data for change from baseline in ABR log-transformed latencies (LTL) by left and right ear were reported. |
| Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | Baseline, Week 22 | ABR assessments were to be conducted under anesthesia and measured the electrical response evoked by acoustic stimuli as sound is processed along the auditory pathway. Data for change from baseline in ABR log-transformed amplitudes (LTA) by left and right ear were reported. |
| Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | Baseline, Week 22 | ABR assessments were to be conducted under anesthesia and measured the electrical response evoked by acoustic stimuli as sound is processed along the auditory pathway. Data for change from baseline in ABR square-root transformed latency (STL) by left and right ear were reported. |
| Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | Baseline, Week 22 | ABR assessments were to be conducted under anesthesia and measured the electrical response evoked by acoustic stimuli as sound is processed along the auditory pathway. Data for change from baseline in ABR square-root transformed amplitude (STA) by left and right ear were reported. |
| Change From Baseline in Mean Auditory Brainstem Response (ABR) at Week 22 | Baseline, Week 22 | ABR assessments were to be conducted under anesthesia and measured the electrical response evoked by acoustic stimuli as sound is processed along the auditory pathway. Mean ABR air and bone conduction threshold were assessed. Mean ABR bone conduction threshold was not possible to be reported as there was insufficient data to be analysed. |
| Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | Baseline, Week 22, Week 26 | FPSS is a sanfilippo-specific disability assessment which assesses motor function, expressive/speech language, and cognitive function on a 0 to 3 point scale. A score of 3 points is assigned for normal function, 2 points for beginning of regression, 1 point for severe level of regression, and 0 points for lost skills. The total disability score (TDS) is the average (0-3) of the motor skills (MS), speech abilities (SA), and cognitive function (CF) scores. Lower scores indicate developmental regression. |
| Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | Baseline, Week 22, Week 26/EOS | SBRS a parent-scored behavioral inventory measuring: comprehensive language skills, expressive language skills, tantrums, mood and emotions, and other behaviors not otherwise classified. Subscale items are scored 0=Never, 1=Occasionally(5-10%), 2=Sometimes (25%), 3=About half the time, 4=Often(75%), 5=Almost always (90%), 6=Always. Summary scores are the sum of responses within a given domain. Higher values = undesirable behavior. Total score range listed below with the abbreviations. Current Communication (CC)(0-42), Past Communication (PC)(0-42), Orality (0-36), Body Movements (BM)(0-30), Interaction With Objects (IWO)(0-24), Activity And Routines (AAR)(0-36), Emotional Function (EF)(0-18), Safety-consciousness (SC)(0-18), Fearfulness (0-36), Social Interaction (SI)(0-36), Eye Contact (EC)(0-18), Emotional Engagement (EE)(0-18), Comfort Seeking (CS)(0-30), Attention (0-18), Self-control/Compliance (SCC)(0-18), Mood, Anger/Aggression (MAA)(0-42), Self-gratification (SG)(0-24). |
| Change From Baseline in Developmental Quotient (DQ) Using Vineland Adaptive Behavioral Scales Second Edition (VABS-II) at Week 22 | Baseline, Week 22 | VABS-II measures adaptive behaviors, including the ability to cope with environmental changes, to learn new everyday skills, and to demonstrate independence. It is an instrument that supports the diagnosis of intellectual and developmental disabilities in participants. This test measures 5 key domains: communication, daily living skills, socialization, motor skills, and the adaptive behavior composite (a composite of the other four domains). Scoring is 'Usually' = 2, 'Sometimes'/Partially' = 1 or 'Never' = 0. The raw scores will be converted to domain standard scores (mean 100, SD 15). Higher scores indicate undesirable behavior. The Overall DQ score was calculated from the mean age-equivalent score obtained by averaging out the age equivalent scores for all the sub-domains except for Gross and Fine motor skills. |
| Change From Baseline in Movement Assessment Battery for Children Second Edition (MABC-2) at Week 26 (EOS) | Baseline, Week 26/EOS | Movement Assessment Battery for Children, Second Edition (MABC-II) was to be used to identify, describe and guide the treatment of motor impairment in children from 3.0 to 16:11 years of age. As per statistical analysis plan, the outcome was to be assessed only if greater than (\>) 50 percent (%) of participants were available for the evaluation. |
Countries
Netherlands, United Kingdom
Participant flow
Pre-assignment details
Study enrolled 12 participants and 4 participants were included in each of the 3 dose groups.
Participants by arm
| Arm | Count |
|---|---|
| HGT-1410 10 mg HGT-1410/rhHNS 10 mg monthly via an IDDD (every 28 \[±7 days\]) for a total of 6 months. | 4 |
| HGT-1410 45 mg HGT-1410/rhHNS 45 mg monthly via an IDDD (every 28 \[±7 days\]) for a total of 6 months. | 4 |
| HGT-1410 90 mg HGT-1410/rhHNS 45 mg dose every 14 \[±2 days\] for a monthly total dose of 90 mg via an IDDD for 6 months. | 4 |
| Total | 12 |
Baseline characteristics
| Characteristic | HGT-1410 10 mg | HGT-1410 45 mg | HGT-1410 90 mg | Total |
|---|---|---|---|---|
| Age, Continuous | 9.15 years STANDARD_DEVIATION 4.7 | 9.07 years STANDARD_DEVIATION 9.8 | 10.64 years STANDARD_DEVIATION 8.7 | 9.618 years STANDARD_DEVIATION 7.2941 |
| Sex: Female, Male Female | 1 Participants | 2 Participants | 1 Participants | 4 Participants |
| Sex: Female, Male Male | 3 Participants | 2 Participants | 3 Participants | 8 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — | — / — |
| other Total, other adverse events | 4 / 4 | 4 / 4 | 4 / 4 |
| serious Total, serious adverse events | 3 / 4 | 3 / 4 | 1 / 4 |
Outcome results
Primary
Number of Participants With Intrathecal Drug Device (IDDD) Failures at Week 26
Participants with IDDD failures were reported.
Time frame: Week 26
Population: ITT population.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| HGT-1410 10 mg | Number of Participants With Intrathecal Drug Device (IDDD) Failures at Week 26 | 3 participants |
| HGT-1410 45 mg | Number of Participants With Intrathecal Drug Device (IDDD) Failures at Week 26 | 2 participants |
| HGT-1410 90 mg | Number of Participants With Intrathecal Drug Device (IDDD) Failures at Week 26 | 1 participants |
Primary
Number of Treatment Emergent Adverse Events (TEAE)
An adverse event (AE) was defined as any untoward medical occurrence in a clinical investigation participant administered as a pharmaceutical product that did not necessarily have a causal relationship with this treatment. A serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged in-patient hospitalization; life threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent AEs (TEAEs) were defined as all adverse events (AEs) from the time of the surgery for IDDD implantation to the last follow up contact, 30 (±7) days after the end of study (EOS) procedures.
Time frame: Baseline to week 30 (follow-up)
Population: Safety population.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| HGT-1410 10 mg | Number of Treatment Emergent Adverse Events (TEAE) | 71 events |
| HGT-1410 45 mg | Number of Treatment Emergent Adverse Events (TEAE) | 42 events |
| HGT-1410 90 mg | Number of Treatment Emergent Adverse Events (TEAE) | 55 events |
Primary
Number of Treatment Emergent Serious Adverse Events (SAE)
An adverse event (AE) was defined as any untoward medical occurrence in a clinical investigation participant administered as a pharmaceutical product that did not necessarily have a causal relationship with this treatment. A serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged in-patient hospitalization; life threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent AEs (TEAEs) were defined as all adverse events (AEs) from the time of the surgery for IDDD implantation to the last follow up contact, 30 (±7) days after the end of study (EOS) procedures.
Time frame: Baseline to week 30 (follow-up)
Population: Safety population was defined as all enrolled participants who received at least 1 dose (full or partial) of study drug.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| HGT-1410 10 mg | Number of Treatment Emergent Serious Adverse Events (SAE) | 5 events |
| HGT-1410 45 mg | Number of Treatment Emergent Serious Adverse Events (SAE) | 3 events |
| HGT-1410 90 mg | Number of Treatment Emergent Serious Adverse Events (SAE) | 2 events |
Primary
Summary of Anti-rhHNS Antibody Status in Cerebrospinal Fluid (CSF) by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group
Participants with positive, negative and missing status were reported.
Time frame: Baseline, Week 26
Population: Intent to treat (ITT) population was defined as all enrolled participants who received at least 1 dose (full or partial) of study drug.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| HGT-1410 10 mg | Summary of Anti-rhHNS Antibody Status in Cerebrospinal Fluid (CSF) by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Negative (Baseline) | 4 participants |
| HGT-1410 10 mg | Summary of Anti-rhHNS Antibody Status in Cerebrospinal Fluid (CSF) by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Positive (Baseline) | 0 participants |
| HGT-1410 10 mg | Summary of Anti-rhHNS Antibody Status in Cerebrospinal Fluid (CSF) by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Missing (Baseline) | 0 participants |
| HGT-1410 10 mg | Summary of Anti-rhHNS Antibody Status in Cerebrospinal Fluid (CSF) by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Negative (Week 26) | 1 participants |
| HGT-1410 10 mg | Summary of Anti-rhHNS Antibody Status in Cerebrospinal Fluid (CSF) by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Positive (Week 26) | 0 participants |
| HGT-1410 10 mg | Summary of Anti-rhHNS Antibody Status in Cerebrospinal Fluid (CSF) by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Missing (Week 26) | 3 participants |
| HGT-1410 45 mg | Summary of Anti-rhHNS Antibody Status in Cerebrospinal Fluid (CSF) by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Missing (Week 26) | 3 participants |
| HGT-1410 45 mg | Summary of Anti-rhHNS Antibody Status in Cerebrospinal Fluid (CSF) by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Negative (Baseline) | 4 participants |
| HGT-1410 45 mg | Summary of Anti-rhHNS Antibody Status in Cerebrospinal Fluid (CSF) by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Negative (Week 26) | 1 participants |
| HGT-1410 45 mg | Summary of Anti-rhHNS Antibody Status in Cerebrospinal Fluid (CSF) by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Positive (Week 26) | 0 participants |
| HGT-1410 45 mg | Summary of Anti-rhHNS Antibody Status in Cerebrospinal Fluid (CSF) by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Positive (Baseline) | 0 participants |
| HGT-1410 45 mg | Summary of Anti-rhHNS Antibody Status in Cerebrospinal Fluid (CSF) by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Missing (Baseline) | 0 participants |
| HGT-1410 90 mg | Summary of Anti-rhHNS Antibody Status in Cerebrospinal Fluid (CSF) by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Positive (Baseline) | 0 participants |
| HGT-1410 90 mg | Summary of Anti-rhHNS Antibody Status in Cerebrospinal Fluid (CSF) by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Missing (Baseline) | 0 participants |
| HGT-1410 90 mg | Summary of Anti-rhHNS Antibody Status in Cerebrospinal Fluid (CSF) by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Missing (Week 26) | 2 participants |
| HGT-1410 90 mg | Summary of Anti-rhHNS Antibody Status in Cerebrospinal Fluid (CSF) by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Negative (Week 26) | 2 participants |
| HGT-1410 90 mg | Summary of Anti-rhHNS Antibody Status in Cerebrospinal Fluid (CSF) by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Negative (Baseline) | 4 participants |
| HGT-1410 90 mg | Summary of Anti-rhHNS Antibody Status in Cerebrospinal Fluid (CSF) by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Positive (Week 26) | 0 participants |
Primary
Summary of Anti-rhHNS Antibody Status in Serum by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group
Participants with positive, negative and missing status were reported.
Time frame: Baseline, Week 26
Population: ITT population
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| HGT-1410 10 mg | Summary of Anti-rhHNS Antibody Status in Serum by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Positive (Week 26) | 2 participants |
| HGT-1410 10 mg | Summary of Anti-rhHNS Antibody Status in Serum by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Missing (Baseline) | 0 participants |
| HGT-1410 10 mg | Summary of Anti-rhHNS Antibody Status in Serum by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Missing (Week 26) | 1 participants |
| HGT-1410 10 mg | Summary of Anti-rhHNS Antibody Status in Serum by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Negative (Week 26) | 1 participants |
| HGT-1410 10 mg | Summary of Anti-rhHNS Antibody Status in Serum by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Positive (Baseline) | 0 participants |
| HGT-1410 10 mg | Summary of Anti-rhHNS Antibody Status in Serum by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Negative (Baseline) | 4 participants |
| HGT-1410 45 mg | Summary of Anti-rhHNS Antibody Status in Serum by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Missing (Week 26) | 2 participants |
| HGT-1410 45 mg | Summary of Anti-rhHNS Antibody Status in Serum by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Negative (Baseline) | 3 participants |
| HGT-1410 45 mg | Summary of Anti-rhHNS Antibody Status in Serum by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Positive (Baseline) | 1 participants |
| HGT-1410 45 mg | Summary of Anti-rhHNS Antibody Status in Serum by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Missing (Baseline) | 0 participants |
| HGT-1410 45 mg | Summary of Anti-rhHNS Antibody Status in Serum by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Negative (Week 26) | 2 participants |
| HGT-1410 45 mg | Summary of Anti-rhHNS Antibody Status in Serum by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Positive (Week 26) | 0 participants |
| HGT-1410 90 mg | Summary of Anti-rhHNS Antibody Status in Serum by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Negative (Week 26) | 2 participants |
| HGT-1410 90 mg | Summary of Anti-rhHNS Antibody Status in Serum by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Positive (Baseline) | 1 participants |
| HGT-1410 90 mg | Summary of Anti-rhHNS Antibody Status in Serum by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Negative (Baseline) | 3 participants |
| HGT-1410 90 mg | Summary of Anti-rhHNS Antibody Status in Serum by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Positive (Week 26) | 0 participants |
| HGT-1410 90 mg | Summary of Anti-rhHNS Antibody Status in Serum by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Missing (Baseline) | 0 participants |
| HGT-1410 90 mg | Summary of Anti-rhHNS Antibody Status in Serum by Recombinant Human Heparan N-Sulfatase (rhHNS) Dose Group | Missing (Week 26) | 2 participants |
Secondary
Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio
ABR assessments were to be conducted under anesthesia and measured the electrical response evoked by acoustic stimuli as sound is processed along the auditory pathway. Data for change from baseline in ABR amplitudes(A), log-transformed amplitudes (LTA), square-root transformed amplitudes (STA) by left ear (LE) and right ear (RE) wave V/I in ratio was reported.
Time frame: Baseline, Week 22
Population: ITT population. Here, n = participants evaluable for specified category for each arm, respectively.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | A-RE, Wave V/I: Baseline (n= 2, 2, 2) | 1.34 ratio | Standard Deviation 0.94 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | A-RE, Wave V/I: Change at Week 22 (n= 2, 1, 1) | 0.14 ratio | Standard Deviation 0.269 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | A-LE, Wave V/I: Baseline (n= 2, 2, 2) | 1.33 ratio | Standard Deviation 0.764 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | A-LE, Wave V/I: Change at Week 22 (n= 2, 1, 1) | 0.37 ratio | Standard Deviation 0.453 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | LTA-RE, Wave V/I: Baseline (n= 2, 2, 2) | 0.146 ratio | Standard Deviation 0.7733 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | LTA-RE, Wave V/I: Change at Week 22 (n= 2, 1, 1) | 0.188 ratio | Standard Deviation 0.3011 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | LTA-LE, Wave V/I: Baseline (n= 2, 2, 2) | 0.195 ratio | Standard Deviation 0.6093 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | LTA-LE, Wave V/I: Change at Week 22 (n= 2, 1, 1) | 0.188 ratio | Standard Deviation 0.1787 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | STA-RE, Wave V/I: Baseline (n= 2, 2, 2) | 1.116 ratio | Standard Deviation 0.4212 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | STA-RE, Wave V/I: Change at Week 22 (n= 2, 1, 1) | 0.082 ratio | Standard Deviation 0.1409 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | STA-LE, Wave V/I: Baseline (n= 2, 2, 2) | 1.128 ratio | Standard Deviation 0.3385 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | STA-LE, Wave V/I: Change at Week 22 (n= 2, 1, 1) | 0.13 ratio | Standard Deviation 0.1448 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | STA-LE, Wave V/I: Change at Week 22 (n= 2, 1, 1) | 0.119 ratio | — |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | A-RE, Wave V/I: Baseline (n= 2, 2, 2) | 1.09 ratio | Standard Deviation 0.057 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | LTA-LE, Wave V/I: Baseline (n= 2, 2, 2) | 0.231 ratio | Standard Deviation 0.0112 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | STA-RE, Wave V/I: Baseline (n= 2, 2, 2) | 1.044 ratio | Standard Deviation 0.0271 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | A-RE, Wave V/I: Change at Week 22 (n= 2, 1, 1) | 0.42 ratio | — |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | LTA-RE, Wave V/I: Change at Week 22 (n= 2, 1, 1) | 0.336 ratio | — |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | STA-LE, Wave V/I: Baseline (n= 2, 2, 2) | 1.122 ratio | Standard Deviation 0.0063 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | A-LE, Wave V/I: Baseline (n= 2, 2, 2) | 1.26 ratio | Standard Deviation 0.014 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | LTA-LE, Wave V/I: Change at Week 22 (n= 2, 1, 1) | 0.202 ratio | — |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | LTA-RE, Wave V/I: Baseline (n= 2, 2, 2) | 0.086 ratio | Standard Deviation 0.0519 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | A-LE, Wave V/I: Change at Week 22 (n= 2, 1, 1) | 0.28 ratio | — |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | STA-RE, Wave V/I: Change at Week 22 (n= 2, 1, 1) | 0.188 ratio | — |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | A-LE, Wave V/I: Change at Week 22 (n= 2, 1, 1) | -0.15 ratio | — |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | LTA-RE, Wave V/I: Baseline (n= 2, 2, 2) | 0.589 ratio | Standard Deviation 0.2227 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | STA-RE, Wave V/I: Change at Week 22 (n= 2, 1, 1) | -0.996 ratio | — |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | LTA-RE, Wave V/I: Change at Week 22 (n= 2, 1, 1) | -3.245 ratio | — |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | LTA-LE, Wave V/I: Baseline (n= 2, 2, 2) | 0.572 ratio | Standard Deviation 1.1791 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | LTA-LE, Wave V/I: Change at Week 22 (n= 2, 1, 1) | -0.217 ratio | — |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | STA-LE, Wave V/I: Baseline (n= 2, 2, 2) | 1.449 ratio | Standard Deviation 0.8078 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | A-RE, Wave V/I: Baseline (n= 2, 2, 2) | 1.83 ratio | Standard Deviation 0.403 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | A-RE, Wave V/I: Change at Week 22 (n= 2, 1, 1) | -1.48 ratio | — |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | STA-RE, Wave V/I: Baseline (n= 2, 2, 2) | 1.347 ratio | Standard Deviation 0.1496 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | A-LE, Wave V/I: Baseline (n= 2, 2, 2) | 2.43 ratio | Standard Deviation 2.341 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitude Ratio | STA-LE, Wave V/I: Change at Week 22 (n= 2, 1, 1) | -0.09 ratio | — |
Secondary
Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes
ABR assessments were to be conducted under anesthesia and measured the electrical response evoked by acoustic stimuli as sound is processed along the auditory pathway. Data for change from baseline in ABR amplitudes by left ear (LE) and right ear (RE) were reported.
Time frame: Baseline, Week 22
Population: ITT population. Here, n = participants evaluable for specified category for each arm, respectively.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | RE, Wave I: Baseline (n= 2, 3, 3) | 0.49 microvolt (mcV) | Standard Deviation 0.042 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | RE, Wave I: Change at Week 22 (n= 2, 3, 3) | -0.23 microvolt (mcV) | Standard Deviation 0.042 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | RE, Wave III: Baseline (n= 2, 3, 3) | 0.33 microvolt (mcV) | Standard Deviation 0.156 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | RE, Wave III: Change at Week 22 (n= 2, 3, 2) | -0.05 microvolt (mcV) | Standard Deviation 0.099 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | RE, Wave V: Baseline (n= 2, 3, 3) | 0.64 microvolt (mcV) | Standard Deviation 0.403 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | RE, Wave V: Change at Week 22 (n= 2, 3, 3) | -0.28 microvolt (mcV) | Standard Deviation 0.354 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | LE, Wave I: Baseline (n= 2, 3, 4) | 0.4 microvolt (mcV) | Standard Deviation 0.028 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | LE, Wave I: Change at Week 22 (n= 2, 3, 3) | -0.1 microvolt (mcV) | Standard Deviation 0.17 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | LE, Wave III: Baseline (n= 2, 3, 4) | 0.22 microvolt (mcV) | Standard Deviation 0.106 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | LE, Wave III: Change at Week 22 (n= 2, 3, 3) | 0.05 microvolt (mcV) | Standard Deviation 0.057 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | LE, Wave V: Baseline (n= 2, 3, 4) | 0.52 microvolt (mcV) | Standard Deviation 0.269 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | LE, Wave V: Change at Week 22 (n= 2, 3, 4) | -0.13 microvolt (mcV) | Standard Deviation 0.24 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | LE, Wave V: Change at Week 22 (n= 2, 3, 4) | -0.02 microvolt (mcV) | Standard Deviation 0.134 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | RE, Wave I: Baseline (n= 2, 3, 3) | 0.42 microvolt (mcV) | Standard Deviation 0.118 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | LE, Wave I: Baseline (n= 2, 3, 4) | 0.44 microvolt (mcV) | Standard Deviation 0.199 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | LE, Wave III: Baseline (n= 2, 3, 4) | 0.38 microvolt (mcV) | Standard Deviation 0.194 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | RE, Wave I: Change at Week 22 (n= 2, 3, 3) | -0.17 microvolt (mcV) | Standard Deviation 0.147 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | RE, Wave V: Change at Week 22 (n= 2, 3, 3) | -0.09 microvolt (mcV) | Standard Deviation 0.145 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | LE, Wave V: Baseline (n= 2, 3, 4) | 0.48 microvolt (mcV) | Standard Deviation 0.123 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | RE, Wave III: Baseline (n= 2, 3, 3) | 0.35 microvolt (mcV) | Standard Deviation 0.195 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | LE, Wave I: Change at Week 22 (n= 2, 3, 3) | 0.02 microvolt (mcV) | Standard Deviation 0.221 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | RE, Wave V: Baseline (n= 2, 3, 3) | 0.47 microvolt (mcV) | Standard Deviation 0.144 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | RE, Wave III: Change at Week 22 (n= 2, 3, 2) | -0.12 microvolt (mcV) | Standard Deviation 0.117 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | LE, Wave III: Change at Week 22 (n= 2, 3, 3) | -0.09 microvolt (mcV) | Standard Deviation 0.188 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | RE, Wave III: Change at Week 22 (n= 2, 3, 2) | -0.16 microvolt (mcV) | Standard Deviation 0.156 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | RE, Wave V: Baseline (n= 2, 3, 3) | 0.32 microvolt (mcV) | Standard Deviation 0.101 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | LE, Wave III: Change at Week 22 (n= 2, 3, 3) | 0.02 microvolt (mcV) | Standard Deviation 0.047 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | RE, Wave V: Change at Week 22 (n= 2, 3, 3) | -0.14 microvolt (mcV) | Standard Deviation 0.201 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | LE, Wave I: Baseline (n= 2, 3, 4) | 0.26 microvolt (mcV) | Standard Deviation 0.133 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | LE, Wave I: Change at Week 22 (n= 2, 3, 3) | -0.01 microvolt (mcV) | Standard Deviation 0.135 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | LE, Wave V: Baseline (n= 2, 3, 4) | 0.29 microvolt (mcV) | Standard Deviation 0.17 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | RE, Wave I: Baseline (n= 2, 3, 3) | 0.26 microvolt (mcV) | Standard Deviation 0.097 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | RE, Wave I: Change at Week 22 (n= 2, 3, 3) | -0.03 microvolt (mcV) | Standard Deviation 0.216 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | LE, Wave III: Baseline (n= 2, 3, 4) | 0.21 microvolt (mcV) | Standard Deviation 0.102 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | RE, Wave III: Baseline (n= 2, 3, 3) | 0.24 microvolt (mcV) | Standard Deviation 0.113 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Amplitudes | LE, Wave V: Change at Week 22 (n= 2, 3, 4) | -0.01 microvolt (mcV) | Standard Deviation 0.085 |
Secondary
Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies
ABR assessments were to be conducted under anesthesia and measured the electrical response evoked by acoustic stimuli as sound is processed along the auditory pathway. The Inter-peak Latencies (IPL) were calculated by subtracting the absolute latencies (AL). The Inter-aural Latencies (IAL) were calculated by subtracting the absolute wave V latencies of the right and left ear. IAL, IPL and AL were reported. Abbreviation: Right Ear (RE), Left Ear (LE), Wave (W), Wave V (WV)
Time frame: Baseline, Week 22
Population: ITT population. Here, n = participants evaluable for specified category for each arm, respectively.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | LE,IPL I-III: Baseline (n= 3, 3, 4) | 2.32 millisecond (ms) | Standard Deviation 0.206 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | RE,IPL I-III: Change at Week 22 (n= 3, 3, 2) | -0.09 millisecond (ms) | Standard Deviation 0.147 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | RE,IPL III-V: Baseline (n= 3, 3, 3) | 2.04 millisecond (ms) | Standard Deviation 0.24 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | RE,IPL III-V: Change at Week 22 (n= 3, 3, 2) | -0.08 millisecond (ms) | Standard Deviation 0.185 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | RE,IPL I-V: Baseline (n= 3, 3, 3) | 4.34 millisecond (ms) | Standard Deviation 0.047 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | RE,IPL I-V: Change at Week 22 (n= 3, 3, 3) | -0.17 millisecond (ms) | Standard Deviation 0.269 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | RE,IPL I-III: Baseline (n= 3, 3, 3) | 2.29 millisecond (ms) | Standard Deviation 0.257 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | LE,IPL I-III: Change at Week 22 (n= 3, 3, 3) | -0.01 millisecond (ms) | Standard Deviation 0.129 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | LE,IPL III-V: Baseline (n= 3, 3, 4) | 2.06 millisecond (ms) | Standard Deviation 0.255 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | LE,IPL III-V: Change at Week 22 (n= 3, 3, 3) | -0.02 millisecond (ms) | Standard Deviation 0.075 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | LE,IPL I-V: Baseline (n= 3, 3, 4) | 4.39 millisecond (ms) | Standard Deviation 0.264 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | LE,IPL I-V: Week 22 (n= 3, 3, 3) | -0.03 millisecond (ms) | Standard Deviation 0.185 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | IAL: Baseline (n= 3, 3, 3) | -0.05 millisecond (ms) | Standard Deviation 0.061 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | IAL: Change at Week 22 (n= 3, 3, 3) | 0.04 millisecond (ms) | Standard Deviation 0.301 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-RE,WI: Baseline (n= 3, 3, 3) | 1.73 millisecond (ms) | Standard Deviation 0.237 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-RE,WI: Change at Week 22 (n= 3, 3, 3) | 0 millisecond (ms) | Standard Deviation 0.466 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-RE,WIII: Baseline (n= 3, 3, 2) | 4.02 millisecond (ms) | Standard Deviation 0.45 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-RE,WIII: Change at Week 22 (n= 3, 3, 2) | -0.09 millisecond (ms) | Standard Deviation 0.355 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-RE,WV: Baseline (n= 3, 3, 3) | 6.06 millisecond (ms) | Standard Deviation 0.283 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-RE,WV: Change at Week 22 (n= 3, 3, 3) | -0.17 millisecond (ms) | Standard Deviation 0.478 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-LE,WI: Baseline (n= 3, 3, 4) | 1.73 millisecond (ms) | Standard Deviation 0.13 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-LE,WI: Change at Week 22 (n= 3, 3, 3) | -0.18 millisecond (ms) | Standard Deviation 0.111 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-LE,WIII: Baseline (n= 3, 3, 4) | 4.05 millisecond (ms) | Standard Deviation 0.323 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-LE,WIII: Change at Week 22 (n= 3, 3, 3) | -0.19 millisecond (ms) | Standard Deviation 0.165 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-LE,WV: Baseline (n= 3, 3, 4) | 6.12 millisecond (ms) | Standard Deviation 0.278 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-LE,WV: Change at Week 22 (n= 3, 3, 4) | -0.21 millisecond (ms) | Standard Deviation 0.178 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | LE,IPL III-V: Baseline (n= 3, 3, 4) | 2.15 millisecond (ms) | Standard Deviation 0.225 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-LE,WI: Baseline (n= 3, 3, 4) | 1.68 millisecond (ms) | Standard Deviation 0.144 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | LE,IPL III-V: Change at Week 22 (n= 3, 3, 3) | 0.09 millisecond (ms) | Standard Deviation 0.091 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | LE,IPL I-V: Baseline (n= 3, 3, 4) | 4.68 millisecond (ms) | Standard Deviation 0.33 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-LE,WV: Baseline (n= 3, 3, 4) | 6.36 millisecond (ms) | Standard Deviation 0.412 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | LE,IPL I-V: Week 22 (n= 3, 3, 3) | -0.15 millisecond (ms) | Standard Deviation 0.07 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-LE,WI: Change at Week 22 (n= 3, 3, 3) | 0.16 millisecond (ms) | Standard Deviation 0.33 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | IAL: Baseline (n= 3, 3, 3) | -0.07 millisecond (ms) | Standard Deviation 0.058 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | IAL: Change at Week 22 (n= 3, 3, 3) | 0.28 millisecond (ms) | Standard Deviation 0.312 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-RE,WI: Baseline (n= 3, 3, 3) | 1.59 millisecond (ms) | Standard Deviation 0.156 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-LE,WIII: Baseline (n= 3, 3, 4) | 4.22 millisecond (ms) | Standard Deviation 0.189 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-RE,WI: Change at Week 22 (n= 3, 3, 3) | 0.46 millisecond (ms) | Standard Deviation 0.54 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-RE,WIII: Baseline (n= 3, 3, 2) | 4.12 millisecond (ms) | Standard Deviation 0.197 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-RE,WIII: Change at Week 22 (n= 3, 3, 2) | 0.35 millisecond (ms) | Standard Deviation 0.687 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | RE,IPL I-III: Baseline (n= 3, 3, 3) | 2.53 millisecond (ms) | Standard Deviation 0.153 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | RE,IPL I-III: Change at Week 22 (n= 3, 3, 2) | -0.11 millisecond (ms) | Standard Deviation 0.183 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-LE,WIII: Change at Week 22 (n= 3, 3, 3) | -0.08 millisecond (ms) | Standard Deviation 0.266 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | RE,IPL III-V: Baseline (n= 3, 3, 3) | 2.17 millisecond (ms) | Standard Deviation 0.261 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-RE,WV: Baseline (n= 3, 3, 3) | 6.3 millisecond (ms) | Standard Deviation 0.454 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | RE,IPL III-V: Change at Week 22 (n= 3, 3, 2) | -0.06 millisecond (ms) | Standard Deviation 0.304 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | RE,IPL I-V: Baseline (n= 3, 3, 3) | 4.71 millisecond (ms) | Standard Deviation 0.352 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | RE,IPL I-V: Change at Week 22 (n= 3, 3, 3) | -0.17 millisecond (ms) | Standard Deviation 0.187 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-RE,WV: Change at Week 22 (n= 3, 3, 3) | 0.29 millisecond (ms) | Standard Deviation 0.633 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | LE,IPL I-III: Baseline (n= 3, 3, 4) | 2.53 millisecond (ms) | Standard Deviation 0.153 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | LE,IPL I-III: Change at Week 22 (n= 3, 3, 3) | -0.24 millisecond (ms) | Standard Deviation 0.065 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-LE,WV: Change at Week 22 (n= 3, 3, 4) | 0.01 millisecond (ms) | Standard Deviation 0.324 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | LE,IPL I-III: Baseline (n= 3, 3, 4) | 2.4 millisecond (ms) | Standard Deviation 0.303 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | RE,IPL I-V: Baseline (n= 3, 3, 3) | 4.29 millisecond (ms) | Standard Deviation 0.238 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | RE,IPL I-III: Baseline (n= 3, 3, 3) | 2.33 millisecond (ms) | Standard Deviation 0.193 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | LE,IPL III-V: Change at Week 22 (n= 3, 3, 3) | 0.06 millisecond (ms) | Standard Deviation 0.04 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-LE,WI: Baseline (n= 3, 3, 4) | 1.78 millisecond (ms) | Standard Deviation 0.233 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-RE,WIII: Change at Week 22 (n= 3, 3, 2) | -0.03 millisecond (ms) | Standard Deviation 0.042 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | LE,IPL I-V: Baseline (n= 3, 3, 4) | 4.48 millisecond (ms) | Standard Deviation 0.394 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-LE,WIII: Change at Week 22 (n= 3, 3, 3) | 0.15 millisecond (ms) | Standard Deviation 0.306 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | RE,IPL I-III: Change at Week 22 (n= 3, 3, 2) | 0.07 millisecond (ms) | Standard Deviation 0.014 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | LE,IPL I-V: Week 22 (n= 3, 3, 3) | 0.17 millisecond (ms) | Standard Deviation 0.191 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-RE,WV: Change at Week 22 (n= 3, 3, 3) | 0.67 millisecond (ms) | Standard Deviation 1.109 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | RE,IPL I-V: Change at Week 22 (n= 3, 3, 3) | 0.2 millisecond (ms) | Standard Deviation 0.131 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | IAL: Baseline (n= 3, 3, 3) | 0.07 millisecond (ms) | Standard Deviation 0.081 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-LE,WI: Change at Week 22 (n= 3, 3, 3) | 0.04 millisecond (ms) | Standard Deviation 0.16 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | RE,IPL III-V: Baseline (n= 3, 3, 3) | 1.96 millisecond (ms) | Standard Deviation 0.119 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | IAL: Change at Week 22 (n= 3, 3, 3) | 0.46 millisecond (ms) | Standard Deviation 0.849 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-LE,WV: Baseline (n= 3, 3, 4) | 6.26 millisecond (ms) | Standard Deviation 0.612 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | LE,IPL III-V: Baseline (n= 3, 3, 4) | 2.09 millisecond (ms) | Standard Deviation 0.311 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-RE,WI: Baseline (n= 3, 3, 3) | 1.76 millisecond (ms) | Standard Deviation 0.012 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | LE,IPL I-III: Change at Week 22 (n= 3, 3, 3) | 0.11 millisecond (ms) | Standard Deviation 0.219 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | RE,IPL III-V: Change at Week 22 (n= 3, 3, 2) | 0.06 millisecond (ms) | Standard Deviation 0.085 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-RE,WI: Change at Week 22 (n= 3, 3, 3) | 0.47 millisecond (ms) | Standard Deviation 0.987 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-LE,WIII: Baseline (n= 3, 3, 4) | 4.18 millisecond (ms) | Standard Deviation 0.504 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-RE,WV: Baseline (n= 3, 3, 3) | 6.06 millisecond (ms) | Standard Deviation 0.227 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-RE,WIII: Baseline (n= 3, 3, 2) | 4.09 millisecond (ms) | Standard Deviation 0.181 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Latencies | AL-LE,WV: Change at Week 22 (n= 3, 3, 4) | 0.24 millisecond (ms) | Standard Deviation 0.227 |
Secondary
Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude
ABR assessments were to be conducted under anesthesia and measured the electrical response evoked by acoustic stimuli as sound is processed along the auditory pathway. Data for change from baseline in ABR log-transformed amplitudes (LTA) by left and right ear were reported.
Time frame: Baseline, Week 22
Population: ITT population. Here, n = participants evaluable for specified category for each arm, respectively.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-RE, Wave I: Baseline (n= 2, 3, 3) | -0.715 log transformed (amplitude [mcV]) | Standard Deviation 0.0867 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-RE, Wave I: Change at Week 22 (n= 2, 3, 3) | -0.659 log transformed (amplitude [mcV]) | Standard Deviation 0.2456 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-RE, Wave III: Baseline (n= 2, 3, 3) | -1.168 log transformed (amplitude [mcV]) | Standard Deviation 0.4901 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-RE, Wave III: Change at Week 22 (n= 2, 3, 2) | -0.116 log transformed (amplitude [mcV]) | Standard Deviation 0.2867 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-RE, Wave V: Baseline (n= 2, 3, 3) | -0.567 log transformed (amplitude [mcV]) | Standard Deviation 0.6834 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-RE, Wave V: Change at Week 22 (n= 2, 3, 3) | -0.474 log transformed (amplitude [mcV]) | Standard Deviation 0.5435 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-LE, Wave I: Baseline (n= 2, 3, 4) | -0.918 log transformed (amplitude [mcV]) | Standard Deviation 0.0708 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-LE, Wave I: Change at Week 22 (n= 2, 3, 3) | -0.409 log transformed (amplitude [mcV]) | Standard Deviation 0.6445 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-LE, Wave III: Baseline (n= 2, 3, 4) | -1.602 log transformed (amplitude [mcV]) | Standard Deviation 0.5149 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-LE, Wave III: Change at Week 22 (n= 2, 3, 3) | 0.265 log transformed (amplitude [mcV]) | Standard Deviation 0.3271 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-LE, Wave V: Baseline (n= 2, 3, 4) | -0.726 log transformed (amplitude [mcV]) | Standard Deviation 0.5418 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-LE, Wave V: Change at Week 22 (n= 2, 3, 4) | -0.217 log transformed (amplitude [mcV]) | Standard Deviation 0.4692 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-LE, Wave V: Change at Week 22 (n= 2, 3, 4) | -0.066 log transformed (amplitude [mcV]) | Standard Deviation 0.2771 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-RE, Wave I: Baseline (n= 2, 3, 3) | -0.893 log transformed (amplitude [mcV]) | Standard Deviation 0.2741 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-LE, Wave I: Baseline (n= 2, 3, 4) | -0.875 log transformed (amplitude [mcV]) | Standard Deviation 0.4212 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-LE, Wave III: Baseline (n= 2, 3, 4) | -1.057 log transformed (amplitude [mcV]) | Standard Deviation 0.4761 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-RE, Wave I: Change at Week 22 (n= 2, 3, 3) | -0.76 log transformed (amplitude [mcV]) | Standard Deviation 0.8475 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-RE, Wave V: Change at Week 22 (n= 2, 3, 3) | -0.214 log transformed (amplitude [mcV]) | Standard Deviation 0.3107 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-LE, Wave V: Baseline (n= 2, 3, 4) | -0.748 log transformed (amplitude [mcV]) | Standard Deviation 0.2486 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-RE, Wave III: Baseline (n= 2, 3, 3) | -1.17 log transformed (amplitude [mcV]) | Standard Deviation 0.6224 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-LE, Wave I: Change at Week 22 (n= 2, 3, 3) | -0.268 log transformed (amplitude [mcV]) | Standard Deviation 0.8163 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-RE, Wave V: Baseline (n= 2, 3, 3) | -0.792 log transformed (amplitude [mcV]) | Standard Deviation 0.2929 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-RE, Wave III: Change at Week 22 (n= 2, 3, 2) | -0.549 log transformed (amplitude [mcV]) | Standard Deviation 0.4627 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-LE, Wave III: Change at Week 22 (n= 2, 3, 3) | -0.374 log transformed (amplitude [mcV]) | Standard Deviation 0.7773 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-RE, Wave III: Change at Week 22 (n= 2, 3, 2) | -1.239 log transformed (amplitude [mcV]) | Standard Deviation 1.5038 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-RE, Wave V: Baseline (n= 2, 3, 3) | -1.19 log transformed (amplitude [mcV]) | Standard Deviation 0.3631 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-LE, Wave III: Change at Week 22 (n= 2, 3, 3) | 0.053 log transformed (amplitude [mcV]) | Standard Deviation 0.151 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-RE, Wave V: Change at Week 22 (n= 2, 3, 3) | -1.237 log transformed (amplitude [mcV]) | Standard Deviation 1.5458 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-LE, Wave I: Baseline (n= 2, 3, 4) | -1.445 log transformed (amplitude [mcV]) | Standard Deviation 0.5468 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-LE, Wave I: Change at Week 22 (n= 2, 3, 3) | 0.078 log transformed (amplitude [mcV]) | Standard Deviation 0.5695 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-LE, Wave V: Baseline (n= 2, 3, 4) | -1.372 log transformed (amplitude [mcV]) | Standard Deviation 0.5377 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-RE, Wave I: Baseline (n= 2, 3, 3) | -1.379 log transformed (amplitude [mcV]) | Standard Deviation 0.3627 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-RE, Wave I: Change at Week 22 (n= 2, 3, 3) | -0.24 log transformed (amplitude [mcV]) | Standard Deviation 1.018 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-LE, Wave III: Baseline (n= 2, 3, 4) | -1.643 log transformed (amplitude [mcV]) | Standard Deviation 0.5086 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-RE, Wave III: Baseline (n= 2, 3, 3) | -1.527 log transformed (amplitude [mcV]) | Standard Deviation 0.589 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Amplitude | LTA-LE, Wave V: Change at Week 22 (n= 2, 3, 4) | 0.006 log transformed (amplitude [mcV]) | Standard Deviation 0.3379 |
Secondary
Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies
ABR assessments were to be conducted under anesthesia and measured the electrical response evoked by acoustic stimuli as sound is processed along the auditory pathway. Data for change from baseline in ABR log-transformed latencies (LTL) by left and right ear were reported.
Time frame: Baseline, Week 22
Population: ITT population. Here, n = participants evaluable for specified category for each arm, respectively.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-RE, Wave I: Baseline (n= 3, 3, 3) | 0.54 log transformed (latency [ms]) | Standard Deviation 0.1326 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-RE, Wave I: Change at Week 22 (n= 3, 3, 3) | -0.006 log transformed (latency [ms]) | Standard Deviation 0.2585 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-RE, Wave III: Baseline (n= 3, 3, 3) | 1.387 log transformed (latency [ms]) | Standard Deviation 0.1121 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-RE, Wave III: Change at Week 22 (n= 3, 3, 2) | -0.019 log transformed (latency [ms]) | Standard Deviation 0.0882 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-RE, Wave V: Baseline (n= 3, 3, 3) | 1.802 log transformed (latency [ms]) | Standard Deviation 0.0461 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-RE, Wave V: Change at Week 22 (n= 3, 3, 3) | -0.028 log transformed (latency [ms]) | Standard Deviation 0.0794 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-LE, Wave I: Baseline (n= 3, 3, 4) | 0.546 log transformed (latency [ms]) | Standard Deviation 0.0769 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-LE, Wave I: Change at Week 22 (n= 3, 3, 3) | -0.106 log transformed (latency [ms]) | Standard Deviation 0.0653 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-LE, Wave III: Baseline (n= 3, 3, 4) | 1.397 log transformed (latency [ms]) | Standard Deviation 0.0804 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-LE, Wave III: Change at Week 22 (n= 3, 3, 3) | -0.046 log transformed (latency [ms]) | Standard Deviation 0.0394 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-LE, Wave V: Baseline (n= 3, 3, 4) | 1.81 log transformed (latency [ms]) | Standard Deviation 0.045 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-LE, Wave V: Change at Week 22 (n= 3, 3, 4) | -0.034 log transformed (latency [ms]) | Standard Deviation 0.0281 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-LE, Wave V: Change at Week 22 (n= 3, 3, 4) | 0.002 log transformed (latency [ms]) | Standard Deviation 0.0494 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-RE, Wave I: Baseline (n= 3, 3, 3) | 0.461 log transformed (latency [ms]) | Standard Deviation 0.0956 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-LE, Wave I: Baseline (n= 3, 3, 4) | 0.518 log transformed (latency [ms]) | Standard Deviation 0.0838 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-LE, Wave III: Baseline (n= 3, 3, 4) | 1.438 log transformed (latency [ms]) | Standard Deviation 0.0455 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-RE, Wave I: Change at Week 22 (n= 3, 3, 3) | 0.242 log transformed (latency [ms]) | Standard Deviation 0.2791 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-RE, Wave V: Change at Week 22 (n= 3, 3, 3) | 0.044 log transformed (latency [ms]) | Standard Deviation 0.0963 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-LE, Wave V: Baseline (n= 3, 3, 4) | 1.849 log transformed (latency [ms]) | Standard Deviation 0.0658 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-RE, Wave III: Baseline (n= 3, 3, 3) | 1.416 log transformed (latency [ms]) | Standard Deviation 0.0483 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-LE, Wave I: Change at Week 22 (n= 3, 3, 3) | 0.088 log transformed (latency [ms]) | Standard Deviation 0.1812 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-RE, Wave V: Baseline (n= 3, 3, 3) | 1.838 log transformed (latency [ms]) | Standard Deviation 0.0731 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-RE, Wave III: Change at Week 22 (n= 3, 3, 2) | 0.074 log transformed (latency [ms]) | Standard Deviation 0.148 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-LE, Wave III: Change at Week 22 (n= 3, 3, 3) | -0.02 log transformed (latency [ms]) | Standard Deviation 0.0624 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-RE, Wave III: Change at Week 22 (n= 3, 3, 2) | -0.008 log transformed (latency [ms]) | Standard Deviation 0.0108 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-RE, Wave V: Baseline (n= 3, 3, 3) | 1.801 log transformed (latency [ms]) | Standard Deviation 0.0374 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-LE, Wave III: Change at Week 22 (n= 3, 3, 3) | 0.032 log transformed (latency [ms]) | Standard Deviation 0.0657 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-RE, Wave V: Change at Week 22 (n= 3, 3, 3) | 0.093 log transformed (latency [ms]) | Standard Deviation 0.1529 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-LE, Wave I: Baseline (n= 3, 3, 4) | 0.57 log transformed (latency [ms]) | Standard Deviation 0.1268 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-LE, Wave I: Change at Week 22 (n= 3, 3, 3) | 0.019 log transformed (latency [ms]) | Standard Deviation 0.0927 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-LE, Wave V: Baseline (n= 3, 3, 4) | 1.831 log transformed (latency [ms]) | Standard Deviation 0.0955 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-RE, Wave I: Baseline (n= 3, 3, 3) | 0.567 log transformed (latency [ms]) | Standard Deviation 0.0066 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-RE, Wave I: Change at Week 22 (n= 3, 3, 3) | 0.179 log transformed (latency [ms]) | Standard Deviation 0.4104 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-LE, Wave III: Baseline (n= 3, 3, 4) | 1.424 log transformed (latency [ms]) | Standard Deviation 0.1213 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-RE, Wave III: Baseline (n= 3, 3, 3) | 1.409 log transformed (latency [ms]) | Standard Deviation 0.0439 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Log Transformed Latencies | LTL-LE, Wave V: Change at Week 22 (n= 3, 3, 4) | 0.036 log transformed (latency [ms]) | Standard Deviation 0.0338 |
Secondary
Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude
ABR assessments were to be conducted under anesthesia and measured the electrical response evoked by acoustic stimuli as sound is processed along the auditory pathway. Data for change from baseline in ABR square-root transformed amplitude (STA) by left and right ear were reported.
Time frame: Baseline, Week 22
Population: ITT population. Here, n = participants evaluable for specified category for each arm, respectively.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-RE, Wave I: Baseline (n= 2, 3, 3) | 0.7 square-root transformed(amplitude [mcV]) | Standard Deviation 0.0303 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-RE, Wave I: Week 22 (n= 2, 3, 3) | -0.193 square-root transformed(amplitude [mcV]) | Standard Deviation 0.0535 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-RE, Wave III: Baseline (n= 2, 3, 3) | 0.566 square-root transformed(amplitude [mcV]) | Standard Deviation 0.1374 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-RE, Wave III: Week 22 (n= 2, 3, 2) | -0.038 square-root transformed(amplitude [mcV]) | Standard Deviation 0.0838 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-RE, Wave V: Baseline (n= 2, 3, 3) | 0.775 square-root transformed(amplitude [mcV]) | Standard Deviation 0.2599 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-RE, Wave V: Week 22 (n= 2, 3, 3) | -0.18 square-root transformed(amplitude [mcV]) | Standard Deviation 0.2183 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-LE, Wave I: Baseline (n= 2, 3, 4) | 0.632 square-root transformed(amplitude [mcV]) | Standard Deviation 0.0224 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-LE, Wave I: Week 22 (n= 2, 3, 3) | -0.101 square-root transformed(amplitude [mcV]) | Standard Deviation 0.1638 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-LE, Wave III: Baseline (n= 2, 3, 4) | 0.456 square-root transformed(amplitude [mcV]) | Standard Deviation 0.1162 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-LE, Wave III: Week 22 (n= 2, 3, 3) | 0.057 square-root transformed(amplitude [mcV]) | Standard Deviation 0.068 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-LE, Wave V: Baseline (n= 2, 3, 4) | 0.709 square-root transformed(amplitude [mcV]) | Standard Deviation 0.1896 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-LE, Wave V: Week 22 (n= 2, 3, 4) | -0.084 square-root transformed(amplitude [mcV]) | Standard Deviation 0.167 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-LE, Wave V: Week 22 (n= 2, 3, 4) | -0.019 square-root transformed(amplitude [mcV]) | Standard Deviation 0.0956 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-RE, Wave I: Baseline (n= 2, 3, 3) | 0.644 square-root transformed(amplitude [mcV]) | Standard Deviation 0.0896 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-LE, Wave I: Baseline (n= 2, 3, 4) | 0.655 square-root transformed(amplitude [mcV]) | Standard Deviation 0.1437 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-LE, Wave III: Baseline (n= 2, 3, 4) | 0.601 square-root transformed(amplitude [mcV]) | Standard Deviation 0.1508 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-RE, Wave I: Week 22 (n= 2, 3, 3) | -0.172 square-root transformed(amplitude [mcV]) | Standard Deviation 0.1615 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-RE, Wave V: Week 22 (n= 2, 3, 3) | -0.069 square-root transformed(amplitude [mcV]) | Standard Deviation 0.1049 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-LE, Wave V: Baseline (n= 2, 3, 4) | 0.692 square-root transformed(amplitude [mcV]) | Standard Deviation 0.0873 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-RE, Wave III: Baseline (n= 2, 3, 3) | 0.575 square-root transformed(amplitude [mcV]) | Standard Deviation 0.171 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-LE, Wave I: Week 22 (n= 2, 3, 3) | -0.028 square-root transformed(amplitude [mcV]) | Standard Deviation 0.1961 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-RE, Wave V: Baseline (n= 2, 3, 3) | 0.678 square-root transformed(amplitude [mcV]) | Standard Deviation 0.1022 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-RE, Wave III: Week 22 (n= 2, 3, 2) | -0.122 square-root transformed(amplitude [mcV]) | Standard Deviation 0.0927 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-LE, Wave III: Week 22 (n= 2, 3, 3) | -0.085 square-root transformed(amplitude [mcV]) | Standard Deviation 0.1798 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-RE, Wave III: Week 22 (n= 2, 3, 2) | -0.211 square-root transformed(amplitude [mcV]) | Standard Deviation 0.2317 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-RE, Wave V: Baseline (n= 2, 3, 3) | 0.557 square-root transformed(amplitude [mcV]) | Standard Deviation 0.0954 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-LE, Wave III: Week 22 (n= 2, 3, 3) | 0.015 square-root transformed(amplitude [mcV]) | Standard Deviation 0.0422 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-RE, Wave V: Week 22 (n= 2, 3, 3) | -0.197 square-root transformed(amplitude [mcV]) | Standard Deviation 0.2547 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-LE, Wave I: Baseline (n= 2, 3, 4) | 0.499 square-root transformed(amplitude [mcV]) | Standard Deviation 0.133 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-LE, Wave I: Week 22 (n= 2, 3, 3) | 0.006 square-root transformed(amplitude [mcV]) | Standard Deviation 0.1365 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-LE, Wave V: Baseline (n= 2, 3, 4) | 0.518 square-root transformed(amplitude [mcV]) | Standard Deviation 0.1488 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-RE, Wave I: Baseline (n= 2, 3, 3) | 0.507 square-root transformed(amplitude [mcV]) | Standard Deviation 0.0933 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-RE, Wave I: Week 22 (n= 2, 3, 3) | -0.044 square-root transformed(amplitude [mcV]) | Standard Deviation 0.2298 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-LE, Wave III: Baseline (n= 2, 3, 4) | 0.45 square-root transformed(amplitude [mcV]) | Standard Deviation 0.1131 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-RE, Wave III: Baseline (n= 2, 3, 3) | 0.479 square-root transformed(amplitude [mcV]) | Standard Deviation 0.1274 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Amplitude | STA-LE, Wave V: Week 22 (n= 2, 3, 4) | -0.004 square-root transformed(amplitude [mcV]) | Standard Deviation 0.0832 |
Secondary
Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies
ABR assessments were to be conducted under anesthesia and measured the electrical response evoked by acoustic stimuli as sound is processed along the auditory pathway. Data for change from baseline in ABR square-root transformed latency (STL) by left and right ear were reported.
Time frame: Baseline, Week 22
Population: ITT population.Here, n = participants evaluable for specified category for each arm, respectively.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-RE, Wave III: Baseline (n= 3, 3, 3) | 2.003 square-root transformed (latency [ms]) | Standard Deviation 0.1123 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-RE, Wave III:Change at Week 22 (n= 3, 3, 2) | -0.021 square-root transformed (latency [ms]) | Standard Deviation 0.0884 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-RE, Wave V: Baseline (n= 3, 3, 3) | 2.462 square-root transformed (latency [ms]) | Standard Deviation 0.0571 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-RE, Wave V: Change at Week 22 (n= 3, 3, 3) | -0.035 square-root transformed (latency [ms]) | Standard Deviation 0.0974 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-LE, Wave I: Baseline (n= 3, 3, 4) | 1.315 square-root transformed (latency [ms]) | Standard Deviation 0.05 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-LE, Wave I: Change at Week 22 (n= 3, 3, 3) | -0.068 square-root transformed (latency [ms]) | Standard Deviation 0.0425 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-LE, Wave III: Baseline (n= 3, 3, 4) | 2.012 square-root transformed (latency [ms]) | Standard Deviation 0.0805 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-LE, Wave III: Change at Week 22 (n= 3, 3, 3) | -0.047 square-root transformed (latency [ms]) | Standard Deviation 0.0403 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-LE, Wave V: Baseline (n= 3, 3, 4) | 2.473 square-root transformed (latency [ms]) | Standard Deviation 0.0559 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-LE, Wave V: Change at Week 22 (n= 3, 3, 4) | -0.042 square-root transformed (latency [ms]) | Standard Deviation 0.0354 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-RE, Wave I: Baseline (n= 3, 3, 3) | 1.312 square-root transformed (latency [ms]) | Standard Deviation 0.0886 |
| HGT-1410 10 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-RE, Wave I: Change at Week 22 (n= 3, 3, 3) | -0.002 square-root transformed (latency [ms]) | Standard Deviation 0.1734 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-RE, Wave I: Change at Week 22 (n= 3, 3, 3) | 0.08 square-root transformed (latency [ms]) | Standard Deviation 0.1935 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-RE, Wave III: Baseline (n= 3, 3, 3) | 2.508 square-root transformed (latency [ms]) | Standard Deviation 0.0487 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-LE, Wave III: Baseline (n= 3, 3, 4) | 2.522 square-root transformed (latency [ms]) | Standard Deviation 0.0464 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-LE, Wave V: Baseline (n= 3, 3, 4) | 1.26 square-root transformed (latency [ms]) | Standard Deviation 0.0824 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-RE, Wave III:Change at Week 22 (n= 3, 3, 2) | 0.056 square-root transformed (latency [ms]) | Standard Deviation 0.1593 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-LE, Wave I: Change at Week 22 (n= 3, 3, 3) | -0.019 square-root transformed (latency [ms]) | Standard Deviation 0.1223 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-RE, Wave I: Baseline (n= 3, 3, 3) | 2.03 square-root transformed (latency [ms]) | Standard Deviation 0.061 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-RE, Wave V: Baseline (n= 3, 3, 3) | 1.297 square-root transformed (latency [ms]) | Standard Deviation 0.091 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-LE, Wave III: Change at Week 22 (n= 3, 3, 3) | -0.019 square-root transformed (latency [ms]) | Standard Deviation 0.0644 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-LE, Wave I: Baseline (n= 3, 3, 4) | 2.053 square-root transformed (latency [ms]) | Standard Deviation 0.055 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-RE, Wave V: Change at Week 22 (n= 3, 3, 3) | 0.059 square-root transformed (latency [ms]) | Standard Deviation 0.1233 |
| HGT-1410 45 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-LE, Wave V: Change at Week 22 (n= 3, 3, 4) | 0.166 square-root transformed (latency [ms]) | Standard Deviation 0.0633 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-RE, Wave V: Change at Week 22 (n= 3, 3, 3) | 0.125 square-root transformed (latency [ms]) | Standard Deviation 0.2057 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-LE, Wave I: Baseline (n= 3, 3, 4) | 1.332 square-root transformed (latency [ms]) | Standard Deviation 0.0858 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-LE, Wave V: Change at Week 22 (n= 3, 3, 4) | 0.047 square-root transformed (latency [ms]) | Standard Deviation 0.0438 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-LE, Wave I: Change at Week 22 (n= 3, 3, 3) | 0.014 square-root transformed (latency [ms]) | Standard Deviation 0.0608 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-LE, Wave III: Baseline (n= 3, 3, 4) | 2.04 square-root transformed (latency [ms]) | Standard Deviation 0.1236 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-LE, Wave III: Change at Week 22 (n= 3, 3, 3) | 0.035 square-root transformed (latency [ms]) | Standard Deviation 0.0709 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-RE, Wave I: Baseline (n= 3, 3, 3) | 1.328 square-root transformed (latency [ms]) | Standard Deviation 0.0044 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-RE, Wave III: Baseline (n= 3, 3, 3) | 2.023 square-root transformed (latency [ms]) | Standard Deviation 0.0446 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-RE, Wave III:Change at Week 22 (n= 3, 3, 2) | -0.008 square-root transformed (latency [ms]) | Standard Deviation 0.0107 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-LE, Wave V: Baseline (n= 3, 3, 4) | 2.5 square-root transformed (latency [ms]) | Standard Deviation 0.1208 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-RE, Wave V: Baseline (n= 3, 3, 3) | 2.461 square-root transformed (latency [ms]) | Standard Deviation 0.0461 |
| HGT-1410 90 mg | Change From Baseline in Auditory Brainstem Response (ABR) at Week 22: Square-root Transformed Latencies | STL-RE, Wave I: Change at Week 22 (n= 3, 3, 3) | 0.145 square-root transformed (latency [ms]) | Standard Deviation 0.3167 |
Secondary
Change From Baseline in Brain Magnetic Resonance Imaging (MRI) at Week 22
Brain MRI was measured for grey matter volume (GMV) , white matter volume (WMV) and Intracranial cerebrospinal fluid Volume (ICSFV) (Ventricles + Additional CSF Space).
Time frame: Baseline, Week 22
Population: ITT population. Here, n = participants evaluable for specified category for each arm, respectively.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| HGT-1410 10 mg | Change From Baseline in Brain Magnetic Resonance Imaging (MRI) at Week 22 | GMV: Baseline | 550.5 milliliter (mL) | Standard Deviation 111.043 |
| HGT-1410 10 mg | Change From Baseline in Brain Magnetic Resonance Imaging (MRI) at Week 22 | GMV: Change at Week 22 | -42.84 milliliter (mL) | Standard Deviation 36.793 |
| HGT-1410 10 mg | Change From Baseline in Brain Magnetic Resonance Imaging (MRI) at Week 22 | WMV: Baseline | 403.72 milliliter (mL) | Standard Deviation 105.575 |
| HGT-1410 10 mg | Change From Baseline in Brain Magnetic Resonance Imaging (MRI) at Week 22 | WMV: Change at Week 22 | -2.86 milliliter (mL) | Standard Deviation 13.997 |
| HGT-1410 10 mg | Change From Baseline in Brain Magnetic Resonance Imaging (MRI) at Week 22 | ICSFV: Baseline | 26.152 milliliter (mL) | Standard Deviation 9.2975 |
| HGT-1410 10 mg | Change From Baseline in Brain Magnetic Resonance Imaging (MRI) at Week 22 | ICSFV: Change at Week 22 | 4.739 milliliter (mL) | Standard Deviation 4.6455 |
| HGT-1410 45 mg | Change From Baseline in Brain Magnetic Resonance Imaging (MRI) at Week 22 | ICSFV: Change at Week 22 | 2.886 milliliter (mL) | Standard Deviation 3.9153 |
| HGT-1410 45 mg | Change From Baseline in Brain Magnetic Resonance Imaging (MRI) at Week 22 | GMV: Baseline | 534.25 milliliter (mL) | Standard Deviation 117.291 |
| HGT-1410 45 mg | Change From Baseline in Brain Magnetic Resonance Imaging (MRI) at Week 22 | WMV: Change at Week 22 | 3.33 milliliter (mL) | Standard Deviation 11.419 |
| HGT-1410 45 mg | Change From Baseline in Brain Magnetic Resonance Imaging (MRI) at Week 22 | ICSFV: Baseline | 22.904 milliliter (mL) | Standard Deviation 20.8459 |
| HGT-1410 45 mg | Change From Baseline in Brain Magnetic Resonance Imaging (MRI) at Week 22 | GMV: Change at Week 22 | -33.7 milliliter (mL) | Standard Deviation 24.161 |
| HGT-1410 45 mg | Change From Baseline in Brain Magnetic Resonance Imaging (MRI) at Week 22 | WMV: Baseline | 348.28 milliliter (mL) | Standard Deviation 76.854 |
| HGT-1410 90 mg | Change From Baseline in Brain Magnetic Resonance Imaging (MRI) at Week 22 | GMV: Change at Week 22 | -32.87 milliliter (mL) | Standard Deviation 36.862 |
| HGT-1410 90 mg | Change From Baseline in Brain Magnetic Resonance Imaging (MRI) at Week 22 | WMV: Baseline | 442.45 milliliter (mL) | Standard Deviation 79.814 |
| HGT-1410 90 mg | Change From Baseline in Brain Magnetic Resonance Imaging (MRI) at Week 22 | ICSFV: Change at Week 22 | 7.375 milliliter (mL) | Standard Deviation 6.6573 |
| HGT-1410 90 mg | Change From Baseline in Brain Magnetic Resonance Imaging (MRI) at Week 22 | WMV: Change at Week 22 | -0.44 milliliter (mL) | Standard Deviation 9.793 |
| HGT-1410 90 mg | Change From Baseline in Brain Magnetic Resonance Imaging (MRI) at Week 22 | GMV: Baseline | 600.28 milliliter (mL) | Standard Deviation 67.884 |
| HGT-1410 90 mg | Change From Baseline in Brain Magnetic Resonance Imaging (MRI) at Week 22 | ICSFV: Baseline | 20.925 milliliter (mL) | Standard Deviation 15.9681 |
Secondary
Change From Baseline in Concentration of Heparan Sulfate and Heparan Sulfate Derivatives in Cerebrospinal Fluid (CSF) at Week 6, 10, 14, 18, 22 and 26(EOS)
Levels of heparan sulfate and its derivatives were evaluated using the proprietary Sensi-Pro (SP) high-performance liquid chromatography (HPLC) based assay. Abbreviation: SP Total Heparan Sulfate (SPTHS), SP Non-Reducing End Assay (SPNREA),
Time frame: Baseline, Week 6, 10, 14, 18, 22 and 26(EOS)
Population: ITT population. Here, n = participants evaluable for specified category for each arm, respectively.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| HGT-1410 10 mg | Change From Baseline in Concentration of Heparan Sulfate and Heparan Sulfate Derivatives in Cerebrospinal Fluid (CSF) at Week 6, 10, 14, 18, 22 and 26(EOS) | SPTHS: Baseline (n= 4, 4, 4) | 90570.5 picomole per milliliter (pmol/mL) | Standard Deviation 38088.67 |
| HGT-1410 10 mg | Change From Baseline in Concentration of Heparan Sulfate and Heparan Sulfate Derivatives in Cerebrospinal Fluid (CSF) at Week 6, 10, 14, 18, 22 and 26(EOS) | SPTHS: Change at Week 6 (n= 4, 2, 2) | -34167.7 picomole per milliliter (pmol/mL) | Standard Deviation 20439.34 |
| HGT-1410 10 mg | Change From Baseline in Concentration of Heparan Sulfate and Heparan Sulfate Derivatives in Cerebrospinal Fluid (CSF) at Week 6, 10, 14, 18, 22 and 26(EOS) | SPTHS: Week 10 (n= 4, 4, 3) | -41825.4 picomole per milliliter (pmol/mL) | Standard Deviation 13482.5 |
| HGT-1410 10 mg | Change From Baseline in Concentration of Heparan Sulfate and Heparan Sulfate Derivatives in Cerebrospinal Fluid (CSF) at Week 6, 10, 14, 18, 22 and 26(EOS) | SPTHS: Change at Week 14 (n= 4, 3, 3) | -40309 picomole per milliliter (pmol/mL) | Standard Deviation 15107.94 |
| HGT-1410 10 mg | Change From Baseline in Concentration of Heparan Sulfate and Heparan Sulfate Derivatives in Cerebrospinal Fluid (CSF) at Week 6, 10, 14, 18, 22 and 26(EOS) | SPTHS: Change at Week 22 (n= 4, 4, 4) | -35469.5 picomole per milliliter (pmol/mL) | Standard Deviation 20922.54 |
| HGT-1410 10 mg | Change From Baseline in Concentration of Heparan Sulfate and Heparan Sulfate Derivatives in Cerebrospinal Fluid (CSF) at Week 6, 10, 14, 18, 22 and 26(EOS) | SPNREA: Baseline (n= 4, 4, 4) | 1255.93 picomole per milliliter (pmol/mL) | Standard Deviation 471.403 |
| HGT-1410 10 mg | Change From Baseline in Concentration of Heparan Sulfate and Heparan Sulfate Derivatives in Cerebrospinal Fluid (CSF) at Week 6, 10, 14, 18, 22 and 26(EOS) | SPNREA: Change at Week 6 (n= 4, 2, 2) | -213.25 picomole per milliliter (pmol/mL) | Standard Deviation 330.836 |
| HGT-1410 10 mg | Change From Baseline in Concentration of Heparan Sulfate and Heparan Sulfate Derivatives in Cerebrospinal Fluid (CSF) at Week 6, 10, 14, 18, 22 and 26(EOS) | SPNREA: Change at Week 10 (n= 4, 4, 3) | -411.77 picomole per milliliter (pmol/mL) | Standard Deviation 268.961 |
| HGT-1410 10 mg | Change From Baseline in Concentration of Heparan Sulfate and Heparan Sulfate Derivatives in Cerebrospinal Fluid (CSF) at Week 6, 10, 14, 18, 22 and 26(EOS) | SPNREA: Change at Week 14 (n= 4, 3, 3) | -359.15 picomole per milliliter (pmol/mL) | Standard Deviation 272.695 |
| HGT-1410 10 mg | Change From Baseline in Concentration of Heparan Sulfate and Heparan Sulfate Derivatives in Cerebrospinal Fluid (CSF) at Week 6, 10, 14, 18, 22 and 26(EOS) | SPNREA: Change at Week 22 (n= 4, 4, 4) | -254.07 picomole per milliliter (pmol/mL) | Standard Deviation 333.649 |
| HGT-1410 45 mg | Change From Baseline in Concentration of Heparan Sulfate and Heparan Sulfate Derivatives in Cerebrospinal Fluid (CSF) at Week 6, 10, 14, 18, 22 and 26(EOS) | SPNREA: Change at Week 14 (n= 4, 3, 3) | -491.24 picomole per milliliter (pmol/mL) | Standard Deviation 177.701 |
| HGT-1410 45 mg | Change From Baseline in Concentration of Heparan Sulfate and Heparan Sulfate Derivatives in Cerebrospinal Fluid (CSF) at Week 6, 10, 14, 18, 22 and 26(EOS) | SPTHS: Baseline (n= 4, 4, 4) | 69049.7 picomole per milliliter (pmol/mL) | Standard Deviation 42659.28 |
| HGT-1410 45 mg | Change From Baseline in Concentration of Heparan Sulfate and Heparan Sulfate Derivatives in Cerebrospinal Fluid (CSF) at Week 6, 10, 14, 18, 22 and 26(EOS) | SPNREA: Baseline (n= 4, 4, 4) | 1198.34 picomole per milliliter (pmol/mL) | Standard Deviation 627.436 |
| HGT-1410 45 mg | Change From Baseline in Concentration of Heparan Sulfate and Heparan Sulfate Derivatives in Cerebrospinal Fluid (CSF) at Week 6, 10, 14, 18, 22 and 26(EOS) | SPTHS: Change at Week 22 (n= 4, 4, 4) | -44669.5 picomole per milliliter (pmol/mL) | Standard Deviation 38360.07 |
| HGT-1410 45 mg | Change From Baseline in Concentration of Heparan Sulfate and Heparan Sulfate Derivatives in Cerebrospinal Fluid (CSF) at Week 6, 10, 14, 18, 22 and 26(EOS) | SPTHS: Change at Week 6 (n= 4, 2, 2) | -41331.4 picomole per milliliter (pmol/mL) | Standard Deviation 38967.66 |
| HGT-1410 45 mg | Change From Baseline in Concentration of Heparan Sulfate and Heparan Sulfate Derivatives in Cerebrospinal Fluid (CSF) at Week 6, 10, 14, 18, 22 and 26(EOS) | SPNREA: Change at Week 22 (n= 4, 4, 4) | -686.13 picomole per milliliter (pmol/mL) | Standard Deviation 517.558 |
| HGT-1410 45 mg | Change From Baseline in Concentration of Heparan Sulfate and Heparan Sulfate Derivatives in Cerebrospinal Fluid (CSF) at Week 6, 10, 14, 18, 22 and 26(EOS) | SPNREA: Change at Week 10 (n= 4, 4, 3) | -533.38 picomole per milliliter (pmol/mL) | Standard Deviation 476.259 |
| HGT-1410 45 mg | Change From Baseline in Concentration of Heparan Sulfate and Heparan Sulfate Derivatives in Cerebrospinal Fluid (CSF) at Week 6, 10, 14, 18, 22 and 26(EOS) | SPTHS: Week 10 (n= 4, 4, 3) | -39441.8 picomole per milliliter (pmol/mL) | Standard Deviation 29771.14 |
| HGT-1410 45 mg | Change From Baseline in Concentration of Heparan Sulfate and Heparan Sulfate Derivatives in Cerebrospinal Fluid (CSF) at Week 6, 10, 14, 18, 22 and 26(EOS) | SPNREA: Change at Week 6 (n= 4, 2, 2) | -677.79 picomole per milliliter (pmol/mL) | Standard Deviation 393.281 |
| HGT-1410 45 mg | Change From Baseline in Concentration of Heparan Sulfate and Heparan Sulfate Derivatives in Cerebrospinal Fluid (CSF) at Week 6, 10, 14, 18, 22 and 26(EOS) | SPTHS: Change at Week 14 (n= 4, 3, 3) | -32249.2 picomole per milliliter (pmol/mL) | Standard Deviation 21862.89 |
| HGT-1410 90 mg | Change From Baseline in Concentration of Heparan Sulfate and Heparan Sulfate Derivatives in Cerebrospinal Fluid (CSF) at Week 6, 10, 14, 18, 22 and 26(EOS) | SPNREA: Change at Week 10 (n= 4, 4, 3) | -788.65 picomole per milliliter (pmol/mL) | Standard Deviation 390.761 |
| HGT-1410 90 mg | Change From Baseline in Concentration of Heparan Sulfate and Heparan Sulfate Derivatives in Cerebrospinal Fluid (CSF) at Week 6, 10, 14, 18, 22 and 26(EOS) | SPTHS: Change at Week 14 (n= 4, 3, 3) | -64763.9 picomole per milliliter (pmol/mL) | Standard Deviation 25285.97 |
| HGT-1410 90 mg | Change From Baseline in Concentration of Heparan Sulfate and Heparan Sulfate Derivatives in Cerebrospinal Fluid (CSF) at Week 6, 10, 14, 18, 22 and 26(EOS) | SPTHS: Change at Week 22 (n= 4, 4, 4) | -60931.7 picomole per milliliter (pmol/mL) | Standard Deviation 20699.36 |
| HGT-1410 90 mg | Change From Baseline in Concentration of Heparan Sulfate and Heparan Sulfate Derivatives in Cerebrospinal Fluid (CSF) at Week 6, 10, 14, 18, 22 and 26(EOS) | SPNREA: Baseline (n= 4, 4, 4) | 1487.67 picomole per milliliter (pmol/mL) | Standard Deviation 329.79 |
| HGT-1410 90 mg | Change From Baseline in Concentration of Heparan Sulfate and Heparan Sulfate Derivatives in Cerebrospinal Fluid (CSF) at Week 6, 10, 14, 18, 22 and 26(EOS) | SPNREA: Change at Week 14 (n= 4, 3, 3) | -1033.58 picomole per milliliter (pmol/mL) | Standard Deviation 371.566 |
| HGT-1410 90 mg | Change From Baseline in Concentration of Heparan Sulfate and Heparan Sulfate Derivatives in Cerebrospinal Fluid (CSF) at Week 6, 10, 14, 18, 22 and 26(EOS) | SPNREA: Change at Week 6 (n= 4, 2, 2) | -1022.53 picomole per milliliter (pmol/mL) | Standard Deviation 163.905 |
| HGT-1410 90 mg | Change From Baseline in Concentration of Heparan Sulfate and Heparan Sulfate Derivatives in Cerebrospinal Fluid (CSF) at Week 6, 10, 14, 18, 22 and 26(EOS) | SPTHS: Baseline (n= 4, 4, 4) | 83287.0 picomole per milliliter (pmol/mL) | Standard Deviation 26012.16 |
| HGT-1410 90 mg | Change From Baseline in Concentration of Heparan Sulfate and Heparan Sulfate Derivatives in Cerebrospinal Fluid (CSF) at Week 6, 10, 14, 18, 22 and 26(EOS) | SPNREA: Change at Week 22 (n= 4, 4, 4) | -877.84 picomole per milliliter (pmol/mL) | Standard Deviation 435.044 |
| HGT-1410 90 mg | Change From Baseline in Concentration of Heparan Sulfate and Heparan Sulfate Derivatives in Cerebrospinal Fluid (CSF) at Week 6, 10, 14, 18, 22 and 26(EOS) | SPTHS: Change at Week 6 (n= 4, 2, 2) | -65862.6 picomole per milliliter (pmol/mL) | Standard Deviation 23010.52 |
| HGT-1410 90 mg | Change From Baseline in Concentration of Heparan Sulfate and Heparan Sulfate Derivatives in Cerebrospinal Fluid (CSF) at Week 6, 10, 14, 18, 22 and 26(EOS) | SPTHS: Week 10 (n= 4, 4, 3) | -54343.7 picomole per milliliter (pmol/mL) | Standard Deviation 25891.78 |
Secondary
Change From Baseline in Developmental Quotient (DQ) Using Bayley Scales of Infant Development Third Edition (BSID III) and Kaufman Assessment Battery for Children Second Edition (KABC II) at Week 22
BSID-III was used to assess the motor (fine and gross), language (receptive and expressive), and cognitive development of infants and toddlers. This measure consists of a series of developmental play tasks. Raw scores of successfully completed items are converted to scale scores and to composite scores. The mean composite score is 100 and the standard deviation (SD) is 15.Higher scores are indicative of decreased development. KABC-II was an individually administered measure of the processing and reasoning abilities of children and adolescents between the ages of 3 and 18 years and is an alternative to BSID-III. BSID-III DQ score was based on the Cognitive domain. The DQ score was calculated from the data obtained from either BSID-III/KABC-II mental age equivalent of the child in months divided by the calendar age in months (multiplied by 100 to give percentage points).
Time frame: Baseline, Week 22
Population: ITT population. Here, n = participants evaluable for specified category for each arm, respectively.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| HGT-1410 10 mg | Change From Baseline in Developmental Quotient (DQ) Using Bayley Scales of Infant Development Third Edition (BSID III) and Kaufman Assessment Battery for Children Second Edition (KABC II) at Week 22 | Baseline (n= 2, 4, 4) | 51.91 units on a scale | Standard Deviation 27.292 |
| HGT-1410 10 mg | Change From Baseline in Developmental Quotient (DQ) Using Bayley Scales of Infant Development Third Edition (BSID III) and Kaufman Assessment Battery for Children Second Edition (KABC II) at Week 22 | Change at Week 22 (n= 2, 4, 4) | -13.6 units on a scale | Standard Deviation 8.886 |
| HGT-1410 45 mg | Change From Baseline in Developmental Quotient (DQ) Using Bayley Scales of Infant Development Third Edition (BSID III) and Kaufman Assessment Battery for Children Second Edition (KABC II) at Week 22 | Baseline (n= 2, 4, 4) | 43.24 units on a scale | Standard Deviation 23.112 |
| HGT-1410 45 mg | Change From Baseline in Developmental Quotient (DQ) Using Bayley Scales of Infant Development Third Edition (BSID III) and Kaufman Assessment Battery for Children Second Edition (KABC II) at Week 22 | Change at Week 22 (n= 2, 4, 4) | -0.89 units on a scale | Standard Deviation 4.341 |
| HGT-1410 90 mg | Change From Baseline in Developmental Quotient (DQ) Using Bayley Scales of Infant Development Third Edition (BSID III) and Kaufman Assessment Battery for Children Second Edition (KABC II) at Week 22 | Baseline (n= 2, 4, 4) | 51.87 units on a scale | Standard Deviation 36.095 |
| HGT-1410 90 mg | Change From Baseline in Developmental Quotient (DQ) Using Bayley Scales of Infant Development Third Edition (BSID III) and Kaufman Assessment Battery for Children Second Edition (KABC II) at Week 22 | Change at Week 22 (n= 2, 4, 4) | -4.91 units on a scale | Standard Deviation 7.769 |
Secondary
Change From Baseline in Developmental Quotient (DQ) Using Vineland Adaptive Behavioral Scales Second Edition (VABS-II) at Week 22
VABS-II measures adaptive behaviors, including the ability to cope with environmental changes, to learn new everyday skills, and to demonstrate independence. It is an instrument that supports the diagnosis of intellectual and developmental disabilities in participants. This test measures 5 key domains: communication, daily living skills, socialization, motor skills, and the adaptive behavior composite (a composite of the other four domains). Scoring is 'Usually' = 2, 'Sometimes'/Partially' = 1 or 'Never' = 0. The raw scores will be converted to domain standard scores (mean 100, SD 15). Higher scores indicate undesirable behavior. The Overall DQ score was calculated from the mean age-equivalent score obtained by averaging out the age equivalent scores for all the sub-domains except for Gross and Fine motor skills.
Time frame: Baseline, Week 22
Population: ITT population. Here, n = participants evaluable for specified category for each arm, respectively.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| HGT-1410 10 mg | Change From Baseline in Developmental Quotient (DQ) Using Vineland Adaptive Behavioral Scales Second Edition (VABS-II) at Week 22 | Baseline (n= 4, 4, 4) | 44.78 units on a scale | Standard Deviation 24.747 |
| HGT-1410 10 mg | Change From Baseline in Developmental Quotient (DQ) Using Vineland Adaptive Behavioral Scales Second Edition (VABS-II) at Week 22 | Change at Week 22 (n= 2, 2, 2) | -11.38 units on a scale | Standard Deviation 12.478 |
| HGT-1410 45 mg | Change From Baseline in Developmental Quotient (DQ) Using Vineland Adaptive Behavioral Scales Second Edition (VABS-II) at Week 22 | Baseline (n= 4, 4, 4) | 47.24 units on a scale | Standard Deviation 26.612 |
| HGT-1410 45 mg | Change From Baseline in Developmental Quotient (DQ) Using Vineland Adaptive Behavioral Scales Second Edition (VABS-II) at Week 22 | Change at Week 22 (n= 2, 2, 2) | -23.96 units on a scale | Standard Deviation 10.014 |
| HGT-1410 90 mg | Change From Baseline in Developmental Quotient (DQ) Using Vineland Adaptive Behavioral Scales Second Edition (VABS-II) at Week 22 | Baseline (n= 4, 4, 4) | 47.71 units on a scale | Standard Deviation 33.687 |
| HGT-1410 90 mg | Change From Baseline in Developmental Quotient (DQ) Using Vineland Adaptive Behavioral Scales Second Edition (VABS-II) at Week 22 | Change at Week 22 (n= 2, 2, 2) | -10.18 units on a scale | Standard Deviation 13.333 |
Secondary
Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS)
FPSS is a sanfilippo-specific disability assessment which assesses motor function, expressive/speech language, and cognitive function on a 0 to 3 point scale. A score of 3 points is assigned for normal function, 2 points for beginning of regression, 1 point for severe level of regression, and 0 points for lost skills. The total disability score (TDS) is the average (0-3) of the motor skills (MS), speech abilities (SA), and cognitive function (CF) scores. Lower scores indicate developmental regression.
Time frame: Baseline, Week 22, Week 26
Population: ITT population. Here, n = participants evaluable for specified category for each arm, respectively.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| HGT-1410 10 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | MS: Baseline (n= 4, 3, 4) | 2.5 units on a scale | Standard Deviation 0.58 |
| HGT-1410 10 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | CF: Change at Week 26/EOS (n= 2, 2, 3) | 0 units on a scale | Standard Deviation 0 |
| HGT-1410 10 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | SA: Change at Week 26/EOS (n= 2, 2, 3) | -0.5 units on a scale | Standard Deviation 0.71 |
| HGT-1410 10 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | MS: Change at Week 26/EOS (n=2,2,3) | 0 units on a scale | Standard Deviation 0 |
| HGT-1410 10 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | CF: Change at Week 22 (n= 4, 3, 4) | 0 units on a scale | Standard Deviation 0 |
| HGT-1410 10 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | CF: Baseline (n= 4, 3, 4) | 2.3 units on a scale | Standard Deviation 0.5 |
| HGT-1410 10 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | MS: Change at Week 22 (n= 4, 3, 4) | 0.3 units on a scale | Standard Deviation 0.5 |
| HGT-1410 10 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | TDS: Change at Week 22 (n= 4, 3, 4) | -0.08 units on a scale | Standard Deviation 0.319 |
| HGT-1410 10 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | SA: Baseline (n= 4, 3, 4) | 2 units on a scale | Standard Deviation 0.82 |
| HGT-1410 10 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | TDS: Change at Week 26/EOS (n= 2, 2, 3) | -0.17 units on a scale | Standard Deviation 0.236 |
| HGT-1410 10 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | TDS: Baseline (n= 4, 3, 4) | 2.25 units on a scale | Standard Deviation 0.569 |
| HGT-1410 10 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | SA: Change at Week 22 (n= 4, 3, 4) | -0.5 units on a scale | Standard Deviation 0.58 |
| HGT-1410 45 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | CF: Baseline (n= 4, 3, 4) | 0.5 units on a scale | Standard Deviation 0.58 |
| HGT-1410 45 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | MS: Change at Week 22 (n= 4, 3, 4) | 0 units on a scale | Standard Deviation 0 |
| HGT-1410 45 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | MS: Change at Week 26/EOS (n=2,2,3) | 0 units on a scale | Standard Deviation 0 |
| HGT-1410 45 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | SA: Baseline (n= 4, 3, 4) | 2 units on a scale | Standard Deviation 0 |
| HGT-1410 45 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | SA: Change at Week 22 (n= 4, 3, 4) | 0.3 units on a scale | Standard Deviation 0.58 |
| HGT-1410 45 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | SA: Change at Week 26/EOS (n= 2, 2, 3) | 0 units on a scale | Standard Deviation 0 |
| HGT-1410 45 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | MS: Baseline (n= 4, 3, 4) | 3 units on a scale | Standard Deviation 0 |
| HGT-1410 45 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | CF: Change at Week 22 (n= 4, 3, 4) | 0 units on a scale | Standard Deviation 0 |
| HGT-1410 45 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | CF: Change at Week 26/EOS (n= 2, 2, 3) | 0 units on a scale | Standard Deviation 0 |
| HGT-1410 45 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | TDS: Baseline (n= 4, 3, 4) | 2.44 units on a scale | Standard Deviation 0.192 |
| HGT-1410 45 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | TDS: Change at Week 22 (n= 4, 3, 4) | 0.11 units on a scale | Standard Deviation 0.192 |
| HGT-1410 45 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | TDS: Change at Week 26/EOS (n= 2, 2, 3) | 0 units on a scale | Standard Deviation 0 |
| HGT-1410 90 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | MS: Baseline (n= 4, 3, 4) | 3 units on a scale | Standard Deviation 0 |
| HGT-1410 90 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | CF: Change at Week 26/EOS (n= 2, 2, 3) | 0 units on a scale | Standard Deviation 0 |
| HGT-1410 90 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | SA: Baseline (n= 4, 3, 4) | 2 units on a scale | Standard Deviation 0.82 |
| HGT-1410 90 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | TDS: Change at Week 26/EOS (n= 2, 2, 3) | 0.11 units on a scale | Standard Deviation 0.192 |
| HGT-1410 90 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | TDS: Baseline (n= 4, 3, 4) | 2.58 units on a scale | Standard Deviation 0.319 |
| HGT-1410 90 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | MS: Change at Week 26/EOS (n=2,2,3) | 0 units on a scale | Standard Deviation 0 |
| HGT-1410 90 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | MS: Change at Week 22 (n= 4, 3, 4) | 0 units on a scale | Standard Deviation 0 |
| HGT-1410 90 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | CF: Baseline (n= 4, 3, 4) | 2.8 units on a scale | Standard Deviation 0.5 |
| HGT-1410 90 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | SA: Change at Week 26/EOS (n= 2, 2, 3) | 0.3 units on a scale | Standard Deviation 0.58 |
| HGT-1410 90 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | TDS: Change at Week 22 (n= 4, 3, 4) | 0.08 units on a scale | Standard Deviation 0.167 |
| HGT-1410 90 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | CF: Change at Week 22 (n= 4, 3, 4) | 0 units on a scale | Standard Deviation 0 |
| HGT-1410 90 mg | Change From Baseline in Four Point Scoring System/Total Disability Score (FPSS/TDS) at Week 22 and Week 26 (EOS) | SA: Change at Week 22 (n= 4, 3, 4) | 0.3 units on a scale | Standard Deviation 0.5 |
Secondary
Change From Baseline in Mean Auditory Brainstem Response (ABR) at Week 22
ABR assessments were to be conducted under anesthesia and measured the electrical response evoked by acoustic stimuli as sound is processed along the auditory pathway. Mean ABR air and bone conduction threshold were assessed. Mean ABR bone conduction threshold was not possible to be reported as there was insufficient data to be analysed.
Time frame: Baseline, Week 22
Population: ITT population. Here, n = participants evaluable for specified category for each arm, respectively.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| HGT-1410 10 mg | Change From Baseline in Mean Auditory Brainstem Response (ABR) at Week 22 | Right Ear: Baseline (n= 1, 2, 3) | 62.5 Decibel Above Normal Adult Hearing Level | — |
| HGT-1410 10 mg | Change From Baseline in Mean Auditory Brainstem Response (ABR) at Week 22 | Right Ear: Change at Week 22 (n= 1, 2, 3) | -10.0 Decibel Above Normal Adult Hearing Level | — |
| HGT-1410 10 mg | Change From Baseline in Mean Auditory Brainstem Response (ABR) at Week 22 | Left Ear: Baseline (n= 2, 2, 4) | 52.5 Decibel Above Normal Adult Hearing Level | Standard Deviation 10.607 |
| HGT-1410 10 mg | Change From Baseline in Mean Auditory Brainstem Response (ABR) at Week 22 | Left Ear: Change at Week 22 (n= 2, 2, 4) | 5.0 Decibel Above Normal Adult Hearing Level | Standard Deviation 7.071 |
| HGT-1410 45 mg | Change From Baseline in Mean Auditory Brainstem Response (ABR) at Week 22 | Left Ear: Change at Week 22 (n= 2, 2, 4) | -5.0 Decibel Above Normal Adult Hearing Level | Standard Deviation 14.142 |
| HGT-1410 45 mg | Change From Baseline in Mean Auditory Brainstem Response (ABR) at Week 22 | Right Ear: Baseline (n= 1, 2, 3) | 56.25 Decibel Above Normal Adult Hearing Level | Standard Deviation 8.839 |
| HGT-1410 45 mg | Change From Baseline in Mean Auditory Brainstem Response (ABR) at Week 22 | Left Ear: Baseline (n= 2, 2, 4) | 55.0 Decibel Above Normal Adult Hearing Level | Standard Deviation 7.071 |
| HGT-1410 45 mg | Change From Baseline in Mean Auditory Brainstem Response (ABR) at Week 22 | Right Ear: Change at Week 22 (n= 1, 2, 3) | -3.75 Decibel Above Normal Adult Hearing Level | Standard Deviation 15.91 |
| HGT-1410 90 mg | Change From Baseline in Mean Auditory Brainstem Response (ABR) at Week 22 | Left Ear: Change at Week 22 (n= 2, 2, 4) | 3.13 Decibel Above Normal Adult Hearing Level | Standard Deviation 10.68 |
| HGT-1410 90 mg | Change From Baseline in Mean Auditory Brainstem Response (ABR) at Week 22 | Right Ear: Change at Week 22 (n= 1, 2, 3) | -0.83 Decibel Above Normal Adult Hearing Level | Standard Deviation 22.407 |
| HGT-1410 90 mg | Change From Baseline in Mean Auditory Brainstem Response (ABR) at Week 22 | Left Ear: Baseline (n= 2, 2, 4) | 44.38 Decibel Above Normal Adult Hearing Level | Standard Deviation 24.696 |
| HGT-1410 90 mg | Change From Baseline in Mean Auditory Brainstem Response (ABR) at Week 22 | Right Ear: Baseline (n= 1, 2, 3) | 49.17 Decibel Above Normal Adult Hearing Level | Standard Deviation 14.216 |
Secondary
Change From Baseline in Movement Assessment Battery for Children Second Edition (MABC-2) at Week 26 (EOS)
Movement Assessment Battery for Children, Second Edition (MABC-II) was to be used to identify, describe and guide the treatment of motor impairment in children from 3.0 to 16:11 years of age. As per statistical analysis plan, the outcome was to be assessed only if greater than (\>) 50 percent (%) of participants were available for the evaluation.
Time frame: Baseline, Week 26/EOS
Population: This outcome measure was not analyzed as it does not meet the pre-specified SAP criteria (outcome was to be assessed only if greater than (\>) 50 percent (%) of participants were available for the evaluation).
Secondary
Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Child Form 87 (CHQ-CF87) at Week 26 (EOS)
CHQ-CF87 form was designed to be a self-report for participants 10 years and older. It consists of 87 questions and contains the same scales as the PF-50, (with the omission of the parental impact scales and there are no psychosocial and physical summary scores derived). As per statistical analysis plan, the outcome was to be assessed only if greater than (\>) 50 percent (%) of participants were available for the evaluation.
Time frame: Baseline, Week 26/EOS
Population: This outcome measure was not analyzed as it does not meet the pre-specified SAP criteria (outcome was to be assessed only if greater than (\>) 50 percent (%) of participants were available for the evaluation).
Secondary
Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS)
CHQ-PF50 which was designed to measure the physical and psychosocial well-being of children 5 years to 18 years of age, consists of 13 health concepts including 11 multi-item and 2 single item scales: Physical Function (PF), Role/Social-Emotional/Behavioral (REB), Role/Social-Physical (RP), bodily pain (BP), General Behavior (BE), Mental Health (MH), Self Esteem (SE), General Health Perceptions (GH), Change in Health (CH), Parental Impact-Emotional (PE), Parental Impact-Time (PT), Family Activities (FA), and Family Cohesion (FC). Transformed scores for all subscales range from 0 to 100, with a higher score indicating better health. Physical and Psychosocial Summary measures (SM) were scored with the use of norm-based methods that standardize the scores to a mean (± Standard Deviation) of 50 ± 10 on the basis of an assessment of the general United States population. Higher values represent better health.
Time frame: Baseline, Week 22, Week 26/EOS
Population: ITT population. Here, n = participants evaluable for specified category for each arm, respectively.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | PF: Change at Week 26/EOS (n= 1, 2, 1) | 33.33 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | MH: Change at Week 22 (n= 2, 4, 2) | 0 units on a scale | Standard Deviation 21.21 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | PE: Change at Week 22 (n= 2, 4, 2) | -8.33 units on a scale | Standard Deviation 82.496 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | BP: Baseline (n= 2, 4, 2) | 90 units on a scale | Standard Deviation 14.14 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | SM Psychosocial: Baseline (n= 2, 4, 2) | 42.16 units on a scale | Standard Deviation 14.06 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | PE: Change at Week 26/EOS (n= 1, 2, 1) | 16.67 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | PF: Change at Week 22 (n= 2, 4, 2) | -8.33 units on a scale | Standard Deviation 51.069 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | SM Physical: Change at Week 26/EOS (n= 1, 2, 1) | 17.25 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | PT: Baseline (n= 2, 4, 2) | 50 units on a scale | Standard Deviation 70.711 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | MH: Baseline (n= 2, 4, 2) | 75 units on a scale | Standard Deviation 7.07 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | SM Physical: Change at Week 22 (n= 2, 4, 2) | -4.63 units on a scale | Standard Deviation 29.276 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | PT: Change at Week 22 (n= 2, 4, 2) | 11.11 units on a scale | Standard Deviation 62.854 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | BP: Change at Week 22 (n= 2, 4, 2) | -20 units on a scale | Standard Deviation 56.57 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | SM Physical: Baseline (n= 2, 4, 2) | 38.44 units on a scale | Standard Deviation 23.545 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | PT: Change at Week 26/EOS (n= 1, 2, 1) | 44.44 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | SE: Change at Week 22 (n= 2, 4, 2) | 0 units on a scale | Standard Deviation 11.785 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | FC: Change at Week 26/EOS (n= 1, 2, 1) | 55.0 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | FA: Baseline (n= 2, 4, 2) | 56.25 units on a scale | Standard Deviation 61.872 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | BE: Change at Week 26/EOS (n= 1, 2, 1) | 30.83 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | FC: Change at Week 22 (n= 2, 4, 2) | 27.5 units on a scale | Standard Deviation 38.89 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | FA: Change at Week 22 (n= 2, 4, 2) | 0 units on a scale | Standard Deviation 58.926 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | PF: Baseline (n= 2, 4, 2) | 66.67 units on a scale | Standard Deviation 47.14 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | FC: Baseline (n= 2, 4, 2) | 57.5 units on a scale | Standard Deviation 38.89 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | FA: Change at Week 26/EOS (n= 1, 2, 1) | 16.67 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | BP: Change at Week 26/EOS (n= 1, 2, 1) | 20 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | RP: Baseline (n= 2, 4, 2) | 66.67 units on a scale | Standard Deviation 47.14 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | BE: Change at Week 22 (n= 2, 4, 2) | 12.08 units on a scale | Standard Deviation 8.839 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | GH: Baseline (n= 2, 4, 2) | 45.42 units on a scale | Standard Deviation 2.946 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | BE: Baseline (n= 2, 4, 2) | 62.5 units on a scale | Standard Deviation 17.678 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | GH: Change at Week 22 (n= 2, 4, 2) | -7.75 units on a scale | Standard Deviation 38.537 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | PE: Baseline (n= 2, 4, 2) | 66.67 units on a scale | Standard Deviation 47.14 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | GH: Change at Week 26/EOS(n= 1, 2, 1) | 17.5 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | SE: Baseline (n= 2, 4, 2) | 62.5 units on a scale | Standard Deviation 5.893 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | RP: Change at Week 22 (n= 2, 4, 2) | 8.33 units on a scale | Standard Deviation 11.785 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | REB: Change at Week 26/EOS (n= 1, 2, 1) | 22.22 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | REB: Change at Week 22 (n= 2, 4, 2) | -38.89 units on a scale | Standard Deviation 86.424 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | SE: Change at Week 26/EOS (n= 1, 2, 1) | 16.67 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | REB: Baseline (n= 2, 4, 2) | 66.67 units on a scale | Standard Deviation 47.14 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | SM Psychosocial:Change at Week 26/EOS (n= 1, 2, 1) | 13.64 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | MH: Change at Week 26/EOS (n= 1, 2, 1) | 15 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | RP: Change at Week 26/EOS (n= 1, 2, 1) | 33.33 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | SM Psychosocial: Change at Week 22 (n= 2, 4, 2) | -1.18 units on a scale | Standard Deviation 23.341 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | FA: Change at Week 22 (n= 2, 4, 2) | -7.29 units on a scale | Standard Deviation 13.767 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | PF: Baseline (n= 2, 4, 2) | 80.28 units on a scale | Standard Deviation 35.141 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | PF: Change at Week 22 (n= 2, 4, 2) | -16.39 units on a scale | Standard Deviation 23.047 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | PF: Change at Week 26/EOS (n= 1, 2, 1) | -24.44 units on a scale | Standard Deviation 34.57 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | RP: Baseline (n= 2, 4, 2) | 50 units on a scale | Standard Deviation 57.735 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | RP: Change at Week 22 (n= 2, 4, 2) | 8.33 units on a scale | Standard Deviation 16.667 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | RP: Change at Week 26/EOS (n= 1, 2, 1) | 0 units on a scale | — |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | BP: Baseline (n= 2, 4, 2) | 67.5 units on a scale | Standard Deviation 29.86 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | BP: Change at Week 22 (n= 2, 4, 2) | 7.5 units on a scale | Standard Deviation 15 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | BP: Change at Week 26/EOS (n= 1, 2, 1) | 0 units on a scale | Standard Deviation 0 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | GH: Change at Week 22 (n= 2, 4, 2) | -5.21 units on a scale | Standard Deviation 10.417 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | GH: Change at Week 26/EOS(n= 1, 2, 1) | 0 units on a scale | Standard Deviation 0 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | GH: Baseline (n= 2, 4, 2) | 27.92 units on a scale | Standard Deviation 21.457 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | BE: Change at Week 26/EOS (n= 1, 2, 1) | -14.37 units on a scale | Standard Deviation 14.437 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | SE: Change at Week 26/EOS (n= 1, 2, 1) | 8.33 units on a scale | Standard Deviation 11.785 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | FC: Baseline (n= 2, 4, 2) | 72.5 units on a scale | Standard Deviation 14.43 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | SM Physical: Change at Week 22 (n= 2, 4, 2) | -1.82 units on a scale | Standard Deviation 6.112 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | SM Psychosocial: Baseline (n= 2, 4, 2) | 33.8 units on a scale | Standard Deviation 10.879 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | PE: Change at Week 22 (n= 2, 4, 2) | -2.08 units on a scale | Standard Deviation 14.232 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | PE: Change at Week 26/EOS (n= 1, 2, 1) | -4.17 units on a scale | Standard Deviation 5.893 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | PT: Baseline (n= 2, 4, 2) | 33.33 units on a scale | Standard Deviation 28.689 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | PT: Change at Week 22 (n= 2, 4, 2) | -5.56 units on a scale | Standard Deviation 26.45 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | PT: Change at Week 26/EOS (n= 1, 2, 1) | -5.56 units on a scale | Standard Deviation 23.57 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | FA: Baseline (n= 2, 4, 2) | 42.71 units on a scale | Standard Deviation 13.767 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | FA: Change at Week 26/EOS (n= 1, 2, 1) | -6.25 units on a scale | Standard Deviation 2.946 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | FC: Change at Week 22 (n= 2, 4, 2) | -6.3 units on a scale | Standard Deviation 12.5 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | FC: Change at Week 26/EOS (n= 1, 2, 1) | 0 units on a scale | Standard Deviation 0 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | SM Physical: Baseline (n= 2, 4, 2) | 31.62 units on a scale | Standard Deviation 23.071 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | SM Physical: Change at Week 26/EOS (n= 1, 2, 1) | -4.99 units on a scale | Standard Deviation 8.195 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | SM Psychosocial: Change at Week 22 (n= 2, 4, 2) | -0.26 units on a scale | Standard Deviation 14.004 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | SM Psychosocial:Change at Week 26/EOS (n= 1, 2, 1) | -1.58 units on a scale | Standard Deviation 2.584 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | REB: Baseline (n= 2, 4, 2) | 41.67 units on a scale | Standard Deviation 50 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | REB: Change at Week 22 (n= 2, 4, 2) | 11.11 units on a scale | Standard Deviation 64.788 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | REB: Change at Week 26/EOS (n= 1, 2, 1) | 0 units on a scale | Standard Deviation 0 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | BE: Baseline (n= 2, 4, 2) | 61.04 units on a scale | Standard Deviation 15.296 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | BE: Change at Week 22 (n= 2, 4, 2) | -2.92 units on a scale | Standard Deviation 15.716 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | MH: Baseline (n= 2, 4, 2) | 68.8 units on a scale | Standard Deviation 10.31 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | MH: Change at Week 22 (n= 2, 4, 2) | -2.5 units on a scale | Standard Deviation 16.58 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | MH: Change at Week 26/EOS (n= 1, 2, 1) | -5 units on a scale | Standard Deviation 7.07 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | SE: Baseline (n= 2, 4, 2) | 61.46 units on a scale | Standard Deviation 18.122 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | SE: Change at Week 22 (n= 2, 4, 2) | -3.13 units on a scale | Standard Deviation 7.116 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | PE: Baseline (n= 2, 4, 2) | 27.08 units on a scale | Standard Deviation 22.948 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | SM Psychosocial: Baseline (n= 2, 4, 2) | 17.57 units on a scale | Standard Deviation 2.418 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | FC: Change at Week 26/EOS (n= 1, 2, 1) | 30 units on a scale | — |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | MH: Change at Week 22 (n= 2, 4, 2) | 17.5 units on a scale | Standard Deviation 17.68 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | SM Psychosocial: Change at Week 22 (n= 2, 4, 2) | 2.27 units on a scale | Standard Deviation 4.972 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | SE: Baseline (n= 2, 4, 2) | 47.92 units on a scale | Standard Deviation 14.731 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | RP: Change at Week 22 (n= 2, 4, 2) | 0 units on a scale | Standard Deviation 0 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | SM Psychosocial:Change at Week 26/EOS (n= 1, 2, 1) | -2.29 units on a scale | — |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | GH: Change at Week 26/EOS(n= 1, 2, 1) | 0 units on a scale | — |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | MH: Change at Week 26/EOS (n= 1, 2, 1) | 5 units on a scale | — |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | SE: Change at Week 26/EOS (n= 1, 2, 1) | -8.33 units on a scale | — |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | REB: Baseline (n= 2, 4, 2) | 50 units on a scale | Standard Deviation 70.711 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | BE: Baseline (n= 2, 4, 2) | 45.83 units on a scale | Standard Deviation 11.785 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | RP: Baseline (n= 2, 4, 2) | 66.67 units on a scale | Standard Deviation 47.14 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | REB: Change at Week 22 (n= 2, 4, 2) | 0 units on a scale | Standard Deviation 0 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | GH: Change at Week 22 (n= 2, 4, 2) | 11.25 units on a scale | Standard Deviation 15.91 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | PF: Baseline (n= 2, 4, 2) | 66.67 units on a scale | Standard Deviation 23.57 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | REB: Change at Week 26/EOS (n= 1, 2, 1) | 0 units on a scale | — |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | GH: Baseline (n= 2, 4, 2) | 33.33 units on a scale | Standard Deviation 23.57 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | PF: Change at Week 26/EOS (n= 1, 2, 1) | -11.11 units on a scale | — |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | BP: Change at Week 26/EOS (n= 1, 2, 1) | 50 units on a scale | — |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | PE: Baseline (n= 2, 4, 2) | 20.83 units on a scale | Standard Deviation 17.678 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | BE: Change at Week 22 (n= 2, 4, 2) | -2.08 units on a scale | Standard Deviation 2.946 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | BP: Change at Week 22 (n= 2, 4, 2) | 45 units on a scale | Standard Deviation 7.07 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | BE: Change at Week 26/EOS (n= 1, 2, 1) | -8.33 units on a scale | — |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | BP: Baseline (n= 2, 4, 2) | 30 units on a scale | Standard Deviation 0 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | FA: Change at Week 26/EOS (n= 1, 2, 1) | 0 units on a scale | — |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | FA: Change at Week 22 (n= 2, 4, 2) | 0 units on a scale | Standard Deviation 0 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | FC: Baseline (n= 2, 4, 2) | 15.0 units on a scale | Standard Deviation 21.21 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | FA: Baseline (n= 2, 4, 2) | 10.42 units on a scale | Standard Deviation 14.731 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | SE: Change at Week 22 (n= 2, 4, 2) | -4.17 units on a scale | Standard Deviation 5.893 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | FC: Change at Week 22 (n= 2, 4, 2) | 15.0 units on a scale | Standard Deviation 21.21 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | PT: Change at Week 26/EOS (n= 1, 2, 1) | 0 units on a scale | — |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | MH: Baseline (n= 2, 4, 2) | 10 units on a scale | Standard Deviation 14.14 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | PT: Change at Week 22 (n= 2, 4, 2) | 16.67 units on a scale | Standard Deviation 23.57 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | RP: Change at Week 26/EOS (n= 1, 2, 1) | 0 units on a scale | — |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | SM Physical: Baseline (n= 2, 4, 2) | 31.27 units on a scale | Standard Deviation 18.193 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | PT: Baseline (n= 2, 4, 2) | 5.56 units on a scale | Standard Deviation 7.857 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | SM Physical: Change at Week 22 (n= 2, 4, 2) | 9.21 units on a scale | Standard Deviation 5.095 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | PE: Change at Week 26/EOS (n= 1, 2, 1) | 0 units on a scale | — |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | PF: Change at Week 22 (n= 2, 4, 2) | 5.56 units on a scale | Standard Deviation 15.713 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | SM Physical: Change at Week 26/EOS (n= 1, 2, 1) | 4.96 units on a scale | — |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Child Health Questionnaire™ Parent Form 50 (CHQ-PF50) Questions at Week 22 and Week 26 (EOS) | PE: Change at Week 22 (n= 2, 4, 2) | 4.17 units on a scale | Standard Deviation 5.893 |
Secondary
Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS)
Children's sleep habits rating scale consisting of 35 item parent questionnaire sleep screening tool. Items are scored on a 3-point scale from 1 (rarely) to 3 (usually) with a Total Sleep Disturbance score (TSDS) ranging from 35-105. Eight subscale scores are totaled to create the TSDS. The subscales are: Bedtime Resistance (BR)(6 items, score = 6-18), Sleep Duration (SD)(3 items, score = 3-9), Parasomnias (P)(7 items, score = 7-21), Sleep Disordered Breathing (SDB)(3 items, score = 3-9), Night Waking (NW)(3 items, score = 3-9), Daytime Sleepiness (DS)(8 items, score = 8-24), Sleep Anxiety (SA)(4 items, score = 4-12), and Sleep Onset Delay (SOD)(1 item, score = 1-3). The questionnaire was designed for children aged 4 through 12 years. A higher score is indicative of more disturbed sleep.
Time frame: Baseline, Week 22, Week 26/EOS
Population: ITT population. Here, n = participants evaluable for specified category for each arm, respectively.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SA: Baseline (n= 4, 4, 4) | 7 units on a scale | Standard Deviation 1.15 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | BR: Change at Week 22 (n= 4, 4, 4) | -1.3 units on a scale | Standard Deviation 1.89 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SDB: Baseline (n= 4, 4, 4) | 3.3 units on a scale | Standard Deviation 0.5 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SA: Change at Week 22 (n= 4, 4, 3) | -1.5 units on a scale | Standard Deviation 1.91 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SOD: Baseline (n= 4, 4, 4) | 1.5 units on a scale | Standard Deviation 1 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | P: Change at Week 26/EOS (n= 1, 2, 2) | -1 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SA: Change at Week 26/EOS (n= 3, 2, 2) | -0.7 units on a scale | Standard Deviation 1.15 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SOD: Change at Week 22 (n= 4, 4, 4) | 0 units on a scale | Standard Deviation 0.82 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | P: Change at Week 22 (n= 2, 4, 4) | -1 units on a scale | Standard Deviation 0 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | NW: Baseline (n= 4, 4, 4) | 4.3 units on a scale | Standard Deviation 1.5 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | BR: Baseline (n= 4, 4, 4) | 8.8 units on a scale | Standard Deviation 1.89 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | P: Baseline (n= 2, 4, 4) | 8.5 units on a scale | Standard Deviation 0.71 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | NW: Change at Week 22 (n= 4, 4, 4) | -0.3 units on a scale | Standard Deviation 2.06 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | DS: Change at Week 26/EOS (n= 3, 2, 2) | 0.7 units on a scale | Standard Deviation 1.15 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | NW: Change at Week 26/EOS (n= 3, 2, 2) | -1 units on a scale | Standard Deviation 1.73 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SOD: Change at Week 26/EOS (n= 3, 2, 2) | -0.3 units on a scale | Standard Deviation 0.58 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | TSDS: Change at Week 22 (n= 2, 4, 2) | 1.5 units on a scale | Standard Deviation 4.95 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | DS: Change at Week 22 (n= 4, 4, 4) | -0.3 units on a scale | Standard Deviation 0.5 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SD: Baseline (n= 4, 4, 4) | 5.3 units on a scale | Standard Deviation 2.22 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | BR: Change at Week 26/EOS (n= 3, 2, 2) | -1 units on a scale | Standard Deviation 1 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | DS: Baseline (n= 4, 4, 4) | 9.5 units on a scale | Standard Deviation 1.29 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SD: Change at Week 22 (n= 4, 4, 4) | -0.8 units on a scale | Standard Deviation 2.99 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | TSDS: Change at Week 26/EOS (n= 1, 2, 2) | -2 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SDB: Change at Week 26/EOS(n= 3, 2, 2) | 0.7 units on a scale | Standard Deviation 1.15 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SD: Change at Week 26/EOS (n= 3, 2, 2) | -1.7 units on a scale | Standard Deviation 2.89 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | TSDS: Baseline (n= 2, 4, 4) | 42.5 units on a scale | Standard Deviation 2.12 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SDB: Change at Week 22 (n= 4, 4, 3) | 0.8 units on a scale | Standard Deviation 0.96 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | DS: Change at Week 26/EOS (n= 3, 2, 2) | 1 units on a scale | Standard Deviation 0 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | BR: Baseline (n= 4, 4, 4) | 7.3 units on a scale | Standard Deviation 2.5 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | BR: Change at Week 22 (n= 4, 4, 4) | 1.3 units on a scale | Standard Deviation 0.96 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | BR: Change at Week 26/EOS (n= 3, 2, 2) | 1 units on a scale | Standard Deviation 1.41 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SOD: Baseline (n= 4, 4, 4) | 1.8 units on a scale | Standard Deviation 0.96 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SOD: Change at Week 22 (n= 4, 4, 4) | 0 units on a scale | Standard Deviation 0.82 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SOD: Change at Week 26/EOS (n= 3, 2, 2) | -0.5 units on a scale | Standard Deviation 0.71 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SD: Baseline (n= 4, 4, 4) | 6.5 units on a scale | Standard Deviation 2.65 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SD: Change at Week 22 (n= 4, 4, 4) | -0.5 units on a scale | Standard Deviation 1.29 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SD: Change at Week 26/EOS (n= 3, 2, 2) | -1 units on a scale | Standard Deviation 1.41 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SA: Baseline (n= 4, 4, 4) | 6 units on a scale | Standard Deviation 1.41 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SA: Change at Week 22 (n= 4, 4, 3) | 0.8 units on a scale | Standard Deviation 1.71 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SA: Change at Week 26/EOS (n= 3, 2, 2) | 0 units on a scale | Standard Deviation 1.41 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | NW: Baseline (n= 4, 4, 4) | 5.5 units on a scale | Standard Deviation 1.91 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | NW: Change at Week 22 (n= 4, 4, 4) | 0 units on a scale | Standard Deviation 1.63 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | NW: Change at Week 26/EOS (n= 3, 2, 2) | -0.5 units on a scale | Standard Deviation 0.71 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | P: Baseline (n= 2, 4, 4) | 10 units on a scale | Standard Deviation 2.31 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | P: Change at Week 22 (n= 2, 4, 4) | 0.3 units on a scale | Standard Deviation 3.4 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | P: Change at Week 26/EOS (n= 1, 2, 2) | -1 units on a scale | Standard Deviation 0 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SDB: Baseline (n= 4, 4, 4) | 4.3 units on a scale | Standard Deviation 0.5 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SDB: Change at Week 22 (n= 4, 4, 3) | 0.5 units on a scale | Standard Deviation 1.73 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SDB: Change at Week 26/EOS(n= 3, 2, 2) | -0.5 units on a scale | Standard Deviation 0.71 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | DS: Baseline (n= 4, 4, 4) | 11.5 units on a scale | Standard Deviation 3.87 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | DS: Change at Week 22 (n= 4, 4, 4) | 0.5 units on a scale | Standard Deviation 0.58 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | TSDS: Baseline (n= 2, 4, 4) | 50.3 units on a scale | Standard Deviation 12.28 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | TSDS: Change at Week 22 (n= 2, 4, 2) | 2 units on a scale | Standard Deviation 5.83 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | TSDS: Change at Week 26/EOS (n= 1, 2, 2) | -2 units on a scale | Standard Deviation 4.24 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SA: Baseline (n= 4, 4, 4) | 8.3 units on a scale | Standard Deviation 3.3 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | BR: Baseline (n= 4, 4, 4) | 11.5 units on a scale | Standard Deviation 4.65 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SDB: Baseline (n= 4, 4, 4) | 5.8 units on a scale | Standard Deviation 3.2 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SD: Change at Week 26/EOS (n= 3, 2, 2) | 1 units on a scale | Standard Deviation 1.41 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | TSDS: Baseline (n= 2, 4, 4) | 58.8 units on a scale | Standard Deviation 16.82 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SDB: Change at Week 22 (n= 4, 4, 3) | -1.7 units on a scale | Standard Deviation 2.89 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SD: Change at Week 22 (n= 4, 4, 4) | 1.3 units on a scale | Standard Deviation 1.5 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | BR: Change at Week 22 (n= 4, 4, 4) | -0.8 units on a scale | Standard Deviation 0.96 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SDB: Change at Week 26/EOS(n= 3, 2, 2) | 0 units on a scale | Standard Deviation 0 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SD: Baseline (n= 4, 4, 4) | 6 units on a scale | Standard Deviation 2.16 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | TSDS: Change at Week 26/EOS (n= 1, 2, 2) | 1 units on a scale | Standard Deviation 2.83 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | DS: Baseline (n= 4, 4, 4) | 11.3 units on a scale | Standard Deviation 3.4 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SOD: Change at Week 26/EOS (n= 3, 2, 2) | 0 units on a scale | Standard Deviation 1.41 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | TSDS: Change at Week 22 (n= 2, 4, 2) | 2 units on a scale | Standard Deviation 5.66 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | DS: Change at Week 22 (n= 4, 4, 4) | 0.5 units on a scale | Standard Deviation 2.65 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | NW: Change at Week 22 (n= 4, 4, 4) | -0.5 units on a scale | Standard Deviation 0.58 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SOD: Change at Week 22 (n= 4, 4, 4) | 0 units on a scale | Standard Deviation 0.82 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | NW: Change at Week 26/EOS (n= 3, 2, 2) | 0 units on a scale | Standard Deviation 0 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | NW: Baseline (n= 4, 4, 4) | 5.8 units on a scale | Standard Deviation 2.5 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SOD: Baseline (n= 4, 4, 4) | 2.3 units on a scale | Standard Deviation 0.96 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | P: Baseline (n= 2, 4, 4) | 12 units on a scale | Standard Deviation 2.94 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SA: Change at Week 26/EOS (n= 3, 2, 2) | -0.5 units on a scale | Standard Deviation 0.71 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | DS: Change at Week 26/EOS (n= 3, 2, 2) | 1 units on a scale | Standard Deviation 1.41 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | P: Change at Week 22 (n= 2, 4, 4) | -0.3 units on a scale | Standard Deviation 2.87 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | SA: Change at Week 22 (n= 4, 4, 3) | -0.3 units on a scale | Standard Deviation 0.58 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | BR: Change at Week 26/EOS (n= 3, 2, 2) | 0 units on a scale | Standard Deviation 0 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Children's Sleep Habits Rating Scale at Week 22 and Week 26 (EOS) | P: Change at Week 26/EOS (n= 1, 2, 2) | -0.5 units on a scale | Standard Deviation 0.71 |
Secondary
Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS)
ITQOL is a generic, validated health status measure for children aged 2 months up to 5 years, including items and scales to measure aspects of physical functioning, development, pain, mood, behavior, general health and impact on parents. The ITQOL consists of 97 items that are scored, summed, and transformed on a scale from 0 (worst health) to 100 (best health). The score range for all individual subscales is 0 (worst health) to 100 (best health). Abbreviation: Overall Health (OH), Physical Abilities (PA), Growth And Development (GAD), Bodily Pain (BP), Temperament And Moods (TAM), General Behavior (GEB), Global Behavior (GLB), Getting Along (GA), General Health Perceptions (GHP), PI-Emotion (PIE), PI-Time (PIT), Family Cohesion (FC).
Time frame: Baseline, Week 22, Week 26/EOS
Population: ITT population. Here, n = participants evaluable for specified category for each arm, respectively.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GAD: Change at Week 26/EOS (n= 1, 0, 0) | 3.1 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GHP: Change at Week 26/EOS (n= 1, 0, 0) | 0 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | BP: Baseline (n= 2, 3, 2) | 95.85 units on a scale | Standard Deviation 5.869 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | FC: Baseline (n= 2, 3, 2) | 85 units on a scale | Standard Deviation 0 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GHP: Change at Week 22 (n= 2, 2, 2) | -18.2 units on a scale | Standard Deviation 22.486 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | BP: Change at Week 22 (n= 2, 2, 2) | -29.2 units on a scale | Standard Deviation 17.678 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | OH: Change at Week 26/EOS (n= 1, 0, 0) | 0 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GHP: Baseline (n= 2, 3, 2) | 48.9 units on a scale | Standard Deviation 4.808 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | BP: Change at Week 26/EOS (n= 1, 0, 0) | -16.7 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | PIT: Change at Week 26/EOS (n= 1, 0, 0) | -28.6 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GA: Change at Week 26/EOS (n= 1, 0, 0) | -16.7 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | TAM: Baseline (n= 2, 3, 2) | 72.2 units on a scale | Standard Deviation 17.678 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GA: Change at Week 22 (n= 2, 2, 2) | -22.5 units on a scale | Standard Deviation 10.607 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | TAM: Change at Week 22 (n= 2, 2, 2) | -23.6 units on a scale | Standard Deviation 21.637 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | PA: Baseline (n= 2, 3, 2) | 83.35 units on a scale | Standard Deviation 9.405 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GA: Baseline (n= 2, 3, 2) | 62.5 units on a scale | Standard Deviation 17.678 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | TAM: Change at Week 26/EOS (n= 1, 0, 0) | -12.8 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GLB: Change at Week 26/EOS (n= 1, 0, 0) | 0 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GEB: Baseline (n= 2, 3, 2) | 58.35 units on a scale | Standard Deviation 32.456 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | OH: Baseline (n= 2, 3, 2) | 80 units on a scale | Standard Deviation 28.28 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GLB: Change at Week 22 (n= 2, 2, 2) | -15 units on a scale | Standard Deviation 21.21 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GEB: Change at Week 22 (n= 2, 2, 2) | -12.5 units on a scale | Standard Deviation 8.91 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GLB: Baseline (n= 2, 3, 2) | 57.5 units on a scale | Standard Deviation 38.89 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GEB: Change at Week 26/EOS (n= 1, 0, 0) | -8.3 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | PIT: Change at Week 22 (n= 2, 2, 2) | -33.35 units on a scale | Standard Deviation 6.718 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | PA: Change at Week 22 (n= 2, 2, 2) | 5 units on a scale | Standard Deviation 11.738 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | PIT: Baseline (n= 2, 3, 2) | 73.8 units on a scale | Standard Deviation 37.052 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | PA: Change at Week 26/EOS (n= 1, 0, 0) | -3.3 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | FC: Change at Week 22 (n= 2, 2, 2) | -12.5 units on a scale | Standard Deviation 17.68 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | PIE: Change at Week 26/EOS (n= 1, 0, 0) | -17.8 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GAD: Baseline (n= 2, 3, 2) | 57.2 units on a scale | Standard Deviation 18.102 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | OH: Change at Week 22 (n= 2, 2, 2) | 0 units on a scale | Standard Deviation 0 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | PIE: Change at Week 22 (n= 2, 2, 2) | -35.7 units on a scale | Standard Deviation 25.314 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GAD: Change at Week 22 (n= 2, 2, 2) | -14.7 units on a scale | Standard Deviation 28.709 |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | FC: Change at Week 26/EOS (n= 1, 0, 0) | -25 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | PIE: Baseline (n= 2, 3, 2) | 78.55 units on a scale | Standard Deviation 30.335 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GHP: Change at Week 22 (n= 2, 2, 2) | -5.7 units on a scale | Standard Deviation 1.556 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | OH: Change at Week 26/EOS (n= 1, 0, 0) | NA units on a scale | — |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | PA: Change at Week 22 (n= 2, 2, 2) | 1.65 units on a scale | Standard Deviation 11.809 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GAD: Change at Week 26/EOS (n= 1, 0, 0) | NA units on a scale | — |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | TAM: Baseline (n= 2, 3, 2) | 68.97 units on a scale | Standard Deviation 21.731 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | TAM: Change at Week 26/EOS (n= 1, 0, 0) | NA units on a scale | — |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GEB: Change at Week 22 (n= 2, 2, 2) | 7.25 units on a scale | Standard Deviation 25.102 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GLB: Baseline (n= 2, 3, 2) | 40 units on a scale | Standard Deviation 34.64 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GA: Change at Week 22 (n= 2, 2, 2) | -2.5 units on a scale | Standard Deviation 22.345 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GHP: Baseline (n= 2, 3, 2) | 31.8 units on a scale | Standard Deviation 20.178 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | PIE: Change at Week 22 (n= 2, 2, 2) | -3.6 units on a scale | Standard Deviation 25.314 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | PIT: Baseline (n= 2, 3, 2) | 49.2 units on a scale | Standard Deviation 26.264 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | FC: Baseline (n= 2, 3, 2) | 76.7 units on a scale | Standard Deviation 14.43 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | OH: Baseline (n= 2, 3, 2) | 56.7 units on a scale | Standard Deviation 49.07 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | OH: Change at Week 22 (n= 2, 2, 2) | -12.5 units on a scale | Standard Deviation 17.68 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | PA: Baseline (n= 2, 3, 2) | 75.57 units on a scale | Standard Deviation 39.496 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | PA: Change at Week 26/EOS (n= 1, 0, 0) | NA units on a scale | — |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GAD: Baseline (n= 2, 3, 2) | 60.83 units on a scale | Standard Deviation 25.658 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GAD: Change at Week 22 (n= 2, 2, 2) | -22.5 units on a scale | Standard Deviation 7.071 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | BP: Baseline (n= 2, 3, 2) | 58.33 units on a scale | Standard Deviation 36.294 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | BP: Change at Week 22 (n= 2, 2, 2) | 25 units on a scale | Standard Deviation 11.738 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | BP: Change at Week 26/EOS (n= 1, 0, 0) | NA units on a scale | — |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | TAM: Change at Week 22 (n= 2, 2, 2) | -2.75 units on a scale | Standard Deviation 33.446 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GEB: Baseline (n= 2, 3, 2) | 34.73 units on a scale | Standard Deviation 17.465 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GEB: Change at Week 26/EOS (n= 1, 0, 0) | NA units on a scale | — |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GLB: Change at Week 22 (n= 2, 2, 2) | 0 units on a scale | Standard Deviation 42.43 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GLB: Change at Week 26/EOS (n= 1, 0, 0) | NA units on a scale | — |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GA: Baseline (n= 2, 3, 2) | 55 units on a scale | Standard Deviation 15 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GA: Change at Week 26/EOS (n= 1, 0, 0) | NA units on a scale | — |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GHP: Change at Week 26/EOS (n= 1, 0, 0) | NA units on a scale | — |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | PIE: Baseline (n= 2, 3, 2) | 48.83 units on a scale | Standard Deviation 33.024 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | PIE: Change at Week 26/EOS (n= 1, 0, 0) | NA units on a scale | — |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | PIT: Change at Week 22 (n= 2, 2, 2) | 7.2 units on a scale | Standard Deviation 23.617 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | PIT: Change at Week 26/EOS (n= 1, 0, 0) | NA units on a scale | — |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | FC: Change at Week 22 (n= 2, 2, 2) | -12.5 units on a scale | Standard Deviation 17.68 |
| HGT-1410 45 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | FC: Change at Week 26/EOS (n= 1, 0, 0) | NA units on a scale | — |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GA: Baseline (n= 2, 3, 2) | 35.85 units on a scale | Standard Deviation 1.202 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | TAM: Baseline (n= 2, 3, 2) | 52.8 units on a scale | Standard Deviation 15.698 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GA: Change at Week 22 (n= 2, 2, 2) | -1.65 units on a scale | Standard Deviation 16.476 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | BP: Change at Week 26/EOS (n= 1, 0, 0) | NA units on a scale | — |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | PIT: Change at Week 26/EOS (n= 1, 0, 0) | NA units on a scale | — |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GA: Change at Week 26/EOS (n= 1, 0, 0) | NA units on a scale | — |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | BP: Change at Week 22 (n= 2, 2, 2) | 12.5 units on a scale | Standard Deviation 41.154 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GHP: Baseline (n= 2, 3, 2) | 57.95 units on a scale | Standard Deviation 36.982 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | BP: Baseline (n= 2, 3, 2) | 62.5 units on a scale | Standard Deviation 29.416 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | OH: Baseline (n= 2, 3, 2) | 65 units on a scale | Standard Deviation 49.5 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GHP: Change at Week 22 (n= 2, 2, 2) | -3.8 units on a scale | Standard Deviation 11.879 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GAD: Change at Week 26/EOS (n= 1, 0, 0) | NA units on a scale | — |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | OH: Change at Week 22 (n= 2, 2, 2) | 15 units on a scale | Standard Deviation 21.21 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GHP: Change at Week 26/EOS (n= 1, 0, 0) | NA units on a scale | — |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GAD: Change at Week 22 (n= 2, 2, 2) | 18.2 units on a scale | Standard Deviation 0.99 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | FC: Baseline (n= 2, 3, 2) | 42.5 units on a scale | Standard Deviation 60.1 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | PIE: Baseline (n= 2, 3, 2) | 41.65 units on a scale | Standard Deviation 23.547 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GAD: Baseline (n= 2, 3, 2) | 56.8 units on a scale | Standard Deviation 6.081 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | PIE: Change at Week 22 (n= 2, 2, 2) | 4.75 units on a scale | Standard Deviation 3.323 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | PA: Change at Week 26/EOS (n= 1, 0, 0) | NA units on a scale | — |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | PIT: Baseline (n= 2, 3, 2) | 45.25 units on a scale | Standard Deviation 63.993 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | PIE: Change at Week 26/EOS (n= 1, 0, 0) | NA units on a scale | — |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | PA: Change at Week 22 (n= 2, 2, 2) | -1.7 units on a scale | Standard Deviation 2.404 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | PA: Baseline (n= 2, 3, 2) | 93.35 units on a scale | Standard Deviation 4.738 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | FC: Change at Week 26/EOS (n= 1, 0, 0) | NA units on a scale | — |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GEB: Change at Week 26/EOS (n= 1, 0, 0) | NA units on a scale | — |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GEB: Change at Week 22 (n= 2, 2, 2) | 18.75 units on a scale | Standard Deviation 0.071 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GLB: Baseline (n= 2, 3, 2) | 0 units on a scale | Standard Deviation 0 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GEB: Baseline (n= 2, 3, 2) | 12.5 units on a scale | Standard Deviation 5.94 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | PIT: Change at Week 22 (n= 2, 2, 2) | 23.8 units on a scale | Standard Deviation 20.223 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GLB: Change at Week 22 (n= 2, 2, 2) | 0 units on a scale | Standard Deviation 0 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | TAM: Change at Week 26/EOS (n= 1, 0, 0) | NA units on a scale | — |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | OH: Change at Week 26/EOS (n= 1, 0, 0) | NA units on a scale | — |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | GLB: Change at Week 26/EOS (n= 1, 0, 0) | NA units on a scale | — |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | TAM: Change at Week 22 (n= 2, 2, 2) | -5.6 units on a scale | Standard Deviation 19.658 |
| HGT-1410 90 mg | Change From Baseline in Quality of Life (QoL) Using Infant Toddler Quality of Life Questionnaire™ (ITQOL) at Week 22 and Week 26 (EOS) | FC: Change at Week 22 (n= 2, 2, 2) | -27.5 units on a scale | Standard Deviation 38.89 |
Secondary
Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS)
SBRS a parent-scored behavioral inventory measuring: comprehensive language skills, expressive language skills, tantrums, mood and emotions, and other behaviors not otherwise classified. Subscale items are scored 0=Never, 1=Occasionally(5-10%), 2=Sometimes (25%), 3=About half the time, 4=Often(75%), 5=Almost always (90%), 6=Always. Summary scores are the sum of responses within a given domain. Higher values = undesirable behavior. Total score range listed below with the abbreviations. Current Communication (CC)(0-42), Past Communication (PC)(0-42), Orality (0-36), Body Movements (BM)(0-30), Interaction With Objects (IWO)(0-24), Activity And Routines (AAR)(0-36), Emotional Function (EF)(0-18), Safety-consciousness (SC)(0-18), Fearfulness (0-36), Social Interaction (SI)(0-36), Eye Contact (EC)(0-18), Emotional Engagement (EE)(0-18), Comfort Seeking (CS)(0-30), Attention (0-18), Self-control/Compliance (SCC)(0-18), Mood, Anger/Aggression (MAA)(0-42), Self-gratification (SG)(0-24).
Time frame: Baseline, Week 22, Week 26/EOS
Population: ITT population. Here, n = participants evaluable for specified category for each arm, respectively.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | EF: Baseline (n= 3, 4, 4) | 2.0 units on a scale | Standard Deviation 3.46 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | CC: Change at Week 22 (n= 4, 4, 2) | 5.0 units on a scale | Standard Deviation 13.44 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | EE: Change at Week 22 (n= 1, 4, 4) | 2.0 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | EF: Change at Week 22 (n= 3, 4, 4) | 2.0 units on a scale | Standard Deviation 3.46 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | MAA: Change at Week 22 (n= 2, 3, 4) | 5.5 units on a scale | Standard Deviation 7.78 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | EE: Baseline (n= 2, 4, 4) | 8.5 units on a scale | Standard Deviation 4.95 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | EF: Change at Week 26/EOS (n= 2, 2, 3) | 5.0 units on a scale | Standard Deviation 1.41 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | Orality: Change at Week 22 (n= 2, 3, 1) | 2.0 units on a scale | Standard Deviation 2.83 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | EC: Change at Week 26/EOS (n= 3, 2, 3) | 0.3 units on a scale | Standard Deviation 0.58 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SC: Baseline (n= 4, 4, 4) | 7.8 units on a scale | Standard Deviation 5.44 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SG: Change at Week 22 (n= 3, 3, 4) | -0.7 units on a scale | Standard Deviation 1.15 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | EC: Change at Week 22 (n= 4, 4, 3) | 1.5 units on a scale | Standard Deviation 1.73 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SC: Change at Week 22 (n= 4, 4, 4) | 0.5 units on a scale | Standard Deviation 1 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | MAA: Baseline (n= 4, 3, 4) | 5.3 units on a scale | Standard Deviation 4.11 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | EC: Baseline (n= 4, 4, 3) | 2.8 units on a scale | Standard Deviation 3.59 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SC: Change at Week 26/EOS (n= 3, 2, 3) | -1.0 units on a scale | Standard Deviation 2.65 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | Orality: Change at Week 26/EOS (n= 1, 1, 1) | 0 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SI: Change at Week 26/EOS (n= 3, 2, 3) | 0 units on a scale | Standard Deviation 8 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | Fearfulness: Baseline (n= 3, 3, 3) | 7.0 units on a scale | Standard Deviation 7.81 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | PC: Baseline (n= 3, 2, 4) | 7.3 units on a scale | Standard Deviation 6.03 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SI: Change at Week 22 (n= 4, 3, 4) | 3.0 units on a scale | Standard Deviation 4.76 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | Fearfulness: Change at Week 22 (n= 3, 3, 3) | 2.7 units on a scale | Standard Deviation 1.15 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SCC: Change at Week 26/EOS (n= 3, 2, 3) | 0.7 units on a scale | Standard Deviation 4.04 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SI: Baseline (n= 4, 4, 4) | 11.0 units on a scale | Standard Deviation 3.37 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | Fearfulness: Change at Week 26/EOS (n= 2, 1, 3) | 2.5 units on a scale | Standard Deviation 2.12 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | CC: Baseline (n= 4, 4, 2) | 8.0 units on a scale | Standard Deviation 5.48 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | BM: Baseline (n= 4, 4, 2) | 5.0 units on a scale | Standard Deviation 4.55 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | AAR: Change at Week 22 (n= 1, 3, 4) | 8.0 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SCC: Change at Week 22 (n= 3, 4, 4) | 0.3 units on a scale | Standard Deviation 4.51 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | BM: Change at Week 22 (n= 3, 4, 2) | 2.3 units on a scale | Standard Deviation 3.51 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | PC: Change at Week 22 (n= 1, 0, 1) | -8.0 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SCC: Baseline (n= 4, 4, 4) | 8.0 units on a scale | Standard Deviation 4.97 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | BM: Change at Week 26/EOS (n= 3, 2, 2) | 0.3 units on a scale | Standard Deviation 1.53 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SG: Change at Week 26/EOS (n= 3, 1, 3) | 0.3 units on a scale | Standard Deviation 0.58 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | Attention: Change at Week 26/EOS (n= 2, 2, 3) | 1.5 units on a scale | Standard Deviation 2.12 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | IWO: Baseline (n= 4, 3, 4) | 4.5 units on a scale | Standard Deviation 3.11 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SG: Baseline (n= 4, 3, 4) | 0.5 units on a scale | Standard Deviation 1 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | Attention: Change at Week 22 (n= 4, 4, 4) | 1.0 units on a scale | Standard Deviation 3.16 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | IWO: Change at Week 22 (n= 4, 3, 3) | 3.3 units on a scale | Standard Deviation 4.27 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | PC: Change at Week 26/EOS (n= 0, 0, 0) | NA units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | Attention: Baseline (n= 4, 4, 4) | 8.3 units on a scale | Standard Deviation 4.65 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | IWO: Change at Week 26/EOS (n= 2, 1, 3) | 1.0 units on a scale | Standard Deviation 1.41 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | CS: Change at Week 26/EOS (n= 2, 1, 3) | 0 units on a scale | Standard Deviation 1.41 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | AAR: Baseline (n= 1, 3, 4) | 6.0 units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | CC: Change at Week 26/EOS (n= 3, 2, 2) | 1.3 units on a scale | Standard Deviation 12.5 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | CS: Change at Week 22 (n= 3, 3, 3) | 1.7 units on a scale | Standard Deviation 4.73 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | MAA: Change at Week 26/EOS (n= 3, 1, 3) | 1.7 units on a scale | Standard Deviation 3.06 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | CS: Baseline (n= 4, 3, 4) | 10.3 units on a scale | Standard Deviation 5.32 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | AAR: Change at Week 26/EOS (n= 0, 1, 3) | NA units on a scale | — |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | Orality: Baseline (n= 2, 3, 3) | 7.0 units on a scale | Standard Deviation 9.9 |
| HGT-1410 10 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | EE: Change at Week 26/EOS (n= 0, 2, 3) | NA units on a scale | — |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | Fearfulness: Change at Week 26/EOS (n= 2, 1, 3) | -5 units on a scale | — |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | CC: Baseline (n= 4, 4, 2) | 6.3 units on a scale | Standard Deviation 6.85 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | CC: Change at Week 22 (n= 4, 4, 2) | -0.3 units on a scale | Standard Deviation 1.26 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | CC: Change at Week 26/EOS (n= 3, 2, 2) | -1.0 units on a scale | Standard Deviation 1.41 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | PC: Baseline (n= 3, 2, 4) | 8.5 units on a scale | Standard Deviation 12.02 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | PC: Change at Week 22 (n= 1, 0, 1) | NA units on a scale | — |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | PC: Change at Week 26/EOS (n= 0, 0, 0) | NA units on a scale | — |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | Orality: Baseline (n= 2, 3, 3) | 10.3 units on a scale | Standard Deviation 2.08 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | Orality: Change at Week 22 (n= 2, 3, 1) | 6.0 units on a scale | Standard Deviation 5.29 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | Orality: Change at Week 26/EOS (n= 1, 1, 1) | 5 units on a scale | — |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | BM: Baseline (n= 4, 4, 2) | 2.5 units on a scale | Standard Deviation 2.08 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | BM: Change at Week 22 (n= 3, 4, 2) | 2 units on a scale | Standard Deviation 2.45 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | BM: Change at Week 26/EOS (n= 3, 2, 2) | 0 units on a scale | Standard Deviation 0 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | IWO: Baseline (n= 4, 3, 4) | 10.3 units on a scale | Standard Deviation 2.08 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | IWO: Change at Week 22 (n= 4, 3, 3) | -2.0 units on a scale | Standard Deviation 4.58 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | AAR: Baseline (n= 1, 3, 4) | 14.3 units on a scale | Standard Deviation 3.06 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | AAR: Change at Week 22 (n= 1, 3, 4) | 0.7 units on a scale | Standard Deviation 0.58 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | AAR: Change at Week 26/EOS (n= 0, 1, 3) | 0 units on a scale | — |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | EF: Baseline (n= 3, 4, 4) | 2.5 units on a scale | Standard Deviation 3.11 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | EF: Change at Week 22 (n= 3, 4, 4) | 2.5 units on a scale | Standard Deviation 3.11 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | EF: Change at Week 26/EOS (n= 2, 2, 3) | 0.5 units on a scale | Standard Deviation 0.71 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SC: Baseline (n= 4, 4, 4) | 9.5 units on a scale | Standard Deviation 1.91 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SC: Change at Week 22 (n= 4, 4, 4) | 1.8 units on a scale | Standard Deviation 5.74 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SC: Change at Week 26/EOS (n= 3, 2, 3) | -3.0 units on a scale | Standard Deviation 2.83 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | Fearfulness: Baseline (n= 3, 3, 3) | 10.3 units on a scale | Standard Deviation 8.74 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | Fearfulness: Change at Week 22 (n= 3, 3, 3) | -1.0 units on a scale | Standard Deviation 5.2 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SI: Baseline (n= 4, 4, 4) | 12.5 units on a scale | Standard Deviation 2.38 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SI: Change at Week 22 (n= 4, 3, 4) | 0.3 units on a scale | Standard Deviation 4.04 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SI: Change at Week 26/EOS (n= 3, 2, 3) | -3.5 units on a scale | Standard Deviation 4.95 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | EC: Baseline (n= 4, 4, 3) | 5.5 units on a scale | Standard Deviation 3.7 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | EC: Change at Week 22 (n= 4, 4, 3) | 0 units on a scale | Standard Deviation 3.27 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | EC: Change at Week 26/EOS (n= 3, 2, 3) | -2.0 units on a scale | Standard Deviation 2.83 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | EE: Baseline (n= 2, 4, 4) | 7.3 units on a scale | Standard Deviation 1.71 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | EE: Change at Week 22 (n= 1, 4, 4) | -0.5 units on a scale | Standard Deviation 1.29 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | EE: Change at Week 26/EOS (n= 0, 2, 3) | -1.0 units on a scale | Standard Deviation 1.41 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | CS: Baseline (n= 4, 3, 4) | 10.3 units on a scale | Standard Deviation 3.06 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | CS: Change at Week 22 (n= 3, 3, 3) | -1.7 units on a scale | Standard Deviation 3.06 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | CS: Change at Week 26/EOS (n= 2, 1, 3) | -1.0 units on a scale | — |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | Attention: Baseline (n= 4, 4, 4) | 8.8 units on a scale | Standard Deviation 1.26 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | Attention: Change at Week 22 (n= 4, 4, 4) | 2.5 units on a scale | Standard Deviation 2.38 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | Attention: Change at Week 26/EOS (n= 2, 2, 3) | 0.5 units on a scale | Standard Deviation 0.71 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SCC: Baseline (n= 4, 4, 4) | 7.3 units on a scale | Standard Deviation 2.22 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SCC: Change at Week 22 (n= 3, 4, 4) | 3.3 units on a scale | Standard Deviation 3.2 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SCC: Change at Week 26/EOS (n= 3, 2, 3) | 0.5 units on a scale | Standard Deviation 0.71 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | MAA: Baseline (n= 4, 3, 4) | 9.7 units on a scale | Standard Deviation 3.51 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | MAA: Change at Week 22 (n= 2, 3, 4) | 3.3 units on a scale | Standard Deviation 5.13 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | MAA: Change at Week 26/EOS (n= 3, 1, 3) | 4.0 units on a scale | — |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SG: Baseline (n= 4, 3, 4) | 0.3 units on a scale | Standard Deviation 0.58 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SG: Change at Week 22 (n= 3, 3, 4) | 2.0 units on a scale | Standard Deviation 1 |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SG: Change at Week 26/EOS (n= 3, 1, 3) | 1.0 units on a scale | — |
| HGT-1410 45 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | IWO: Change at Week 26/EOS (n= 2, 1, 3) | -1.0 units on a scale | — |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | AAR: Change at Week 26/EOS (n= 0, 1, 3) | 1.7 units on a scale | Standard Deviation 2.08 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | MAA: Change at Week 22 (n= 2, 3, 4) | -0.3 units on a scale | Standard Deviation 2.5 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | EE: Change at Week 26/EOS (n= 0, 2, 3) | 0.7 units on a scale | Standard Deviation 1.15 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | AAR: Change at Week 22 (n= 1, 3, 4) | -1.3 units on a scale | Standard Deviation 6.08 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | PC: Change at Week 26/EOS (n= 0, 0, 0) | NA units on a scale | — |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | CS: Baseline (n= 4, 3, 4) | 16.5 units on a scale | Standard Deviation 6.56 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | AAR: Baseline (n= 1, 3, 4) | 12.3 units on a scale | Standard Deviation 9.46 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | PC: Baseline (n= 3, 2, 4) | 11.0 units on a scale | Standard Deviation 11.37 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | CS: Change at Week 22 (n= 3, 3, 3) | -2.3 units on a scale | Standard Deviation 3.21 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | IWO: Change at Week 26/EOS (n= 2, 1, 3) | -2.0 units on a scale | Standard Deviation 3.46 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | PC: Change at Week 22 (n= 1, 0, 1) | 0 units on a scale | — |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | CS: Change at Week 26/EOS (n= 2, 1, 3) | -2.3 units on a scale | Standard Deviation 3.21 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | IWO: Change at Week 22 (n= 4, 3, 3) | -2.0 units on a scale | Standard Deviation 3.46 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | MAA: Change at Week 26/EOS (n= 3, 1, 3) | -0.7 units on a scale | Standard Deviation 2.89 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | IWO: Baseline (n= 4, 3, 4) | 8.5 units on a scale | Standard Deviation 7.9 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SG: Baseline (n= 4, 3, 4) | 4.5 units on a scale | Standard Deviation 5.26 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | Attention: Change at Week 22 (n= 4, 4, 4) | 2.3 units on a scale | Standard Deviation 1.71 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | BM: Change at Week 26/EOS (n= 3, 2, 2) | -2.0 units on a scale | Standard Deviation 2.83 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | CC: Change at Week 26/EOS (n= 3, 2, 2) | 1.5 units on a scale | Standard Deviation 2.12 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | Attention: Change at Week 26/EOS (n= 2, 2, 3) | 3.3 units on a scale | Standard Deviation 1.15 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | BM: Change at Week 22 (n= 3, 4, 2) | -2.0 units on a scale | Standard Deviation 2.83 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | CC: Baseline (n= 4, 4, 2) | 10.0 units on a scale | Standard Deviation 5.66 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SCC: Baseline (n= 4, 4, 4) | 10.0 units on a scale | Standard Deviation 7.3 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | BM: Baseline (n= 4, 4, 2) | 12.0 units on a scale | Standard Deviation 2.83 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SG: Change at Week 22 (n= 3, 3, 4) | -1.0 units on a scale | Standard Deviation 4.08 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SCC: Change at Week 22 (n= 3, 4, 4) | 2.0 units on a scale | Standard Deviation 1.83 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | Fearfulness: Change at Week 26/EOS (n= 2, 1, 3) | 2.3 units on a scale | Standard Deviation 1.53 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | Fearfulness: Change at Week 22 (n= 3, 3, 3) | 2.0 units on a scale | Standard Deviation 1.73 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | Orality: Change at Week 26/EOS (n= 1, 1, 1) | 4 units on a scale | — |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SI: Baseline (n= 4, 4, 4) | 14.5 units on a scale | Standard Deviation 6.76 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | Fearfulness: Baseline (n= 3, 3, 3) | 11.0 units on a scale | Standard Deviation 6.08 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | CC: Change at Week 22 (n= 4, 4, 2) | 1.5 units on a scale | Standard Deviation 2.12 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SI: Change at Week 22 (n= 4, 3, 4) | -0.5 units on a scale | Standard Deviation 3.7 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SC: Change at Week 26/EOS (n= 3, 2, 3) | -1.0 units on a scale | Standard Deviation 3 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SCC: Change at Week 26/EOS (n= 3, 2, 3) | 2.7 units on a scale | Standard Deviation 1.53 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SI: Change at Week 26/EOS (n= 3, 2, 3) | -0.7 units on a scale | Standard Deviation 4.62 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SC: Change at Week 22 (n= 4, 4, 4) | -0.8 units on a scale | Standard Deviation 2.5 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | Orality: Change at Week 22 (n= 2, 3, 1) | 4.0 units on a scale | — |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | EC: Baseline (n= 4, 4, 3) | 6.7 units on a scale | Standard Deviation 5.77 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SC: Baseline (n= 4, 4, 4) | 11.5 units on a scale | Standard Deviation 7.9 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | Attention: Baseline (n= 4, 4, 4) | 10.5 units on a scale | Standard Deviation 7.72 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | EC: Change at Week 22 (n= 4, 4, 3) | -1.0 units on a scale | Standard Deviation 2.65 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | EF: Change at Week 26/EOS (n= 2, 2, 3) | -1.31 units on a scale | Standard Deviation 3.06 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | MAA: Baseline (n= 4, 3, 4) | 17.3 units on a scale | Standard Deviation 15.65 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | EC: Change at Week 26/EOS (n= 3, 2, 3) | -0.7 units on a scale | Standard Deviation 3.06 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | EF: Change at Week 22 (n= 3, 4, 4) | 7.5 units on a scale | Standard Deviation 5 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | Orality: Baseline (n= 2, 3, 3) | 17.3 units on a scale | Standard Deviation 10.26 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | EE: Baseline (n= 2, 4, 4) | 8.3 units on a scale | Standard Deviation 3.3 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | EF: Baseline (n= 3, 4, 4) | 7.5 units on a scale | Standard Deviation 5 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | SG: Change at Week 26/EOS (n= 3, 1, 3) | 1.0 units on a scale | Standard Deviation 1 |
| HGT-1410 90 mg | Change From Baseline in Sanfilippo Behavioral Rating Scale (SBRS) at Week 22 and Week 26 (EOS) | EE: Change at Week 22 (n= 1, 4, 4) | -0.8 units on a scale | Standard Deviation 2.87 |
Secondary
Number of Participants With Accumulation of Recombinant Human Heparan N-Sulfatase (rhHNS) in Cerebrospinal Fluid (CSF) at Week 22
Cerebrospinal fluid samples were collected from participants through an implanted IDDD or via lumbar puncture (LP) immediately prior to each administration of HGT-1410.
Time frame: Baseline, Week 22
Population: ITT population.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| HGT-1410 10 mg | Number of Participants With Accumulation of Recombinant Human Heparan N-Sulfatase (rhHNS) in Cerebrospinal Fluid (CSF) at Week 22 | 0 participants |
| HGT-1410 45 mg | Number of Participants With Accumulation of Recombinant Human Heparan N-Sulfatase (rhHNS) in Cerebrospinal Fluid (CSF) at Week 22 | 0 participants |
| HGT-1410 90 mg | Number of Participants With Accumulation of Recombinant Human Heparan N-Sulfatase (rhHNS) in Cerebrospinal Fluid (CSF) at Week 22 | 0 participants |