Metachromatic Leukodystrophy (MLD)
Conditions
Keywords
rhASA, MLD, Metazym
Brief summary
Objectives: The overall objective is to evaluate the safety, efficacy and pharmacokinetics (PK) of rhASA treatment in patients with late infantile MLD. Methodology: This is a single center, open-label study of patients with late infantile MLD. Twelve patients will be enrolled in this study receiving a total of thirteen intravenous infusions of Metazym. One infusion will be given every other week for a period of half a year. After the half year the subjects will continue treatment every other week until safety data is available. Safety (AE/SAE) will be monitored at every visit during this period.
Detailed description
Test product, dose, mode of administration, batch No.: The lowest dose level will be evaluated as a single dose of 25 U/kg. The three upper dose levels will be evaluated as repeated doses. Patients in each cohort will receive one dose of enzyme every other week for a period of eight weeks, a total of five doses. Dosing will be performed as follows: Cohort 1: 25 U/kg as a single dose - hereafter 50 U/kg; Cohort 2: 100 U/kg; Cohort 3: 200 U/kg. Patients receiving the lowest dose as a single dose will receive the next dose level as a repeated dose. After twenty six weeks the subjects will continue treatment every other week until safety data is available. Safety (AE/SAE) will be monitored at every visit during this period. The dose will be adjusted monthly to account for changes in body weight. The infusion length will be dependent on the dose. Doses of 25 U/kg, 50 U/kg and 100 U/kg will be diluted in 50 ml isotonic sodium chloride and infused over 30 minutes. Infusion of 200 U/kg will be administered in the same manner except for an infusion time of 60 minutes. Duration of treatment: Half a year (26 weeks)
Interventions
BIOLOGICALrhASA - Dose Level 1
Intravenous infusion 25 U/kg as a single dose - hereafter 50 U/kg every other week for 26 weeks
BIOLOGICALrhASA - Dose Level 2
Intravenous infusion 100 U/kg every other week for 26 weeks
BIOLOGICALrhASA - Dose Level 3
Intravenous infusion 200 U/kg every other week for 26 weeks
Sponsors
Shire
Study design
Allocation
NON_RANDOMIZED
Intervention model
FACTORIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Years to 5 Years
Healthy volunteers
No
Inclusion criteria
1. Subject's legally authorized guardian(s) must provide signed, informed consent prior to performing any study-related activities (Trial-related activities are any procedures that would not have been performed during normal management of the subject). 2. The patient must have a confirmed diagnosis of MLD as defined by: ASA activity \< 10 nmol/h/mg in leukocytes Presence of elevated sulfatide in urine 3. The patient must have a confirmed nerve conduction velocity \< 2 standard deviations (from the appropriate age level) 4. The patient must have a residual level of voluntary function (as judged by the investigator), including presence of residual cognitive function (attention, executive and visual functions) as well as the presence of residual voluntary motor function in one upper or lower limb as a minimum. 5. The patient must have an age at the time of screening ≥ 1 year and \< 6 years 6. The patient must have had onset of symptoms before the age of 4 years 7. The subject and his/her guardian(s) must have the ability to comply with the clinical protocol 8. The patients' medical record must document that the legal guardian(s) has had independent counselling or a consultation regarding stem cell transplantation in order to assure that the guardian(s) is fully informed regarding the risks and benefits of this alternative
Exclusion criteria
Patients will be excluded from this study if they do not meet the specific inclusion criteria, or if any of the following criteria apply: 1. Lack of voluntary function 2. Presence of severe pseudo-bulbar signs (weakness and disco-ordination of tongue and swallowing muscles leading to severe difficulty with swallowing) 3. Spasticity so severe to inhibit transportation 4. Known multiple sulfatase deficiency 5. Presence of major congenital abnormality 6. Presence of known chromosomal abnormality and syndromes affecting psychomotor development 7. History of stem cell transplantation 8. Presence of known clinically significant cardiovascular, hepatic, pulmonary or renal disease or other medical condition 9. Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial 10. Use of any investigational product within 30 days prior to study enrolment or currently enrolled in another study which involves clinical investigations 11. Received ERT with rhASA from any source 12. Planned or anticipated initiation of antispastic treatment after trial initiation
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | From study drug administration up to Week 28 | An adverse event (AE) is any untoward, undesired, unplanned clinical event in the form of signs, symptoms, disease, or laboratory or physiological observations occurring in a participant, participating in a clinical study with study drug, regardless of causal relationship. TEAEs were AEs occurred after study drug administration that were absent before treatment or that worsened relative to pre-treatment state, up to Week 28 until evaluation (when last cohort had 26-week evaluation and data management performed within 4 weeks) completed. |
| Arylsulfatase A (ASA) Activity in Leukocytes | Pre-dose and post-dose at 24 hours on Day 0 and at Weeks 8 and 26 | — |
| Maximum Plasma Drug Concentration (Cmax) of Recombinant Human Arylsulphatase A (rhASA) | Pre-dose and post-dose at 20, 40, 90 minutes, 3, 6 and 8 hours on Day 0, 40 minutes post-dose at Week 4, Pre-dose and post-dose at 20, 40, 90 minutes, 3, 6 and 8 hours at Week 8 | — |
| Change From Baseline in Mullen's Scales of Early Learning at Week 26 | Baseline, Week 26 | Mullen's Scales of Early Learning is used to assess performance and learning ability in young children. The scale consisted of 144 items that had specific scoring criteria for each item. The scores were converted to T-scores with a decrease in score indicating worsening of disease. Relative change from baseline at Week 26 was calculated as percentage change from baseline divided by the age-difference in months between first and last visit. Adjusted mean and 95 percent (%) confidence intervals were reported. |
| Number of Participants With Shift From Baseline to Week 26 in Sulfatide Levels in Urine | Baseline up to Week 26 | Number of participants with shifts between negative (value=0) and positive (value=1) values in urine sulfatide levels from baseline at Week 26 is reported. |
| Change From Baseline in Cerebrospinal Fluid (CSF) Sulfatide at Week 26 | Baseline, Week 26 | Relative change from baseline at Week 26 was calculated as percentage change from baseline divided by the age-difference in months between first and last visit. Adjusted mean and 95 percent (%) confidence intervals were reported. |
| Change From Baseline in Gross Motor Function Measure (GMFM) at Week 26 | Baseline, Week 26 | GMFM was measured using GMFM-88 item scores and summed to calculate a total GMFM-88 score. For each GMFM-88 item, the score was between 0 (minimal) to 3 (maximum). The total GMFM-88 score was between 0 (minimal) and 264 (maximum). The decrease in GMFM score over time indicates worsening of disease over time. Relative change from baseline at Week 26 was calculated as percentage change from baseline divided by the age-difference in months between first and last visit. Adjusted mean and 95 percent (%) confidence intervals were reported. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Nerve Conduction Velocity at Week 26 | Baseline, Week 26 | An electrophysiological evaluation using standard electrophysiological and electromyography to measure the speed and extent of nerve conduction and units are expressed in meters per second. Abbreviations: MN=Median Nerve; PN=Peroneal Nerve; SN=Sural Nerve; Dig.=Digit; FH=fibular hemimelia; L LM=left lateral medial; R LM=right lateral medial; MC=medial collateral. |
| Number of Participants Who Had Undergone Nerve Biopsy and Had a Normal Nerve at Both Baseline and Week 26 | Baseline, Week 26 | — |
| Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Baseline up to Week 26 | Loes scoring system is used to grade the demyelinating abnormalities on brain MRI. A total of 17 locations of the brain were scored from 0 (normal appearance) to 2 (dense appearance). The total score ranged from 0 to 34 with a score of 14 or greater being considered severe. Number of participants with any shift of score between 0 to 2 for each of the 17 locations (Parieto Occipital \[PO\]-Periventricular \[P\], Central \[C\], Subcortical \[Sc\]; Anterior Temporal \[AT\]-P, C, Sc; Frontal \[F\]-P, C, Sc; Corpus Callosum \[CC\]-Splenium \[S\], Genus \[G\]; Projection Fibers \[PF\]-Capsular interna \[CI\] ant, CI post, Brainstem \[B\]; Cerebellum \[Cb\]-Cortex, Atrophy; Basal Ganglia \[BG\]-BG, Thalamus \[T\]; Cerebral Atrophy \[CA\]-CA), are only reported. |
| Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Baseline, Week 26 | PEDI is used for the clinical evaluation of functional capabilities, performance and changes in functional skills in children with disabilities. It consisted of 20 items scored on a scale from 0 (total assistance) to 5 (independent). Total score ranged from 0-100 with higher scores indicating better functioning. None, child, rehab, extensive are items in 3 domains (self-care, mobility and social functioning). |
Other
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Baseline up to Week 26 | Number of participants with at least 1 shift from baseline to Week 26 are reported. Abbreviations: Abs=Absolute count; ERCS=Erythrocytes; MCHC=Mean corpuscular hemoglobin concentration; MCH=Mean cell hemoglobin. |
| Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Baseline up to Week 26 | Number of participants with at least 1 shift from baseline to Week 26 are reported. Abbreviations: CSF=Cerebrospinal fluid; NFP=Neurofilament proteins. |
| Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Coagulation | Baseline up to Week 26 | Number of participants with at least 1 shift from baseline to Week 26 are reported. The shift reported below for Cohort 1 was from low level at baseline to low level at Week 26. |
| Change From Baseline in Neurofilament Proteins (NFP), Glial Fibrillary Acidic Protein (GFAP) and Tauprotein in Cerebrospinal Fluid (CSF) at Week 26 | Baseline, Week 26 | — |
| Change From Baseline in Chitotriosidase at Week 26 | Baseline, Week 26 | — |
| Number of Participants With Clinically Significant Abnormal Electrocardiogram (ECG) Findings | Baseline up to Week 26 | Abnormal ECG findings were considered as clinically significant at the discretion of investigator. |
| Number of Participants With Abnormal Findings in Urine Analysis | Baseline up to Week 26 | The parameters analyzed in urine were albumin/protein, glucose, leucocytes, acetoacetate/ketones, nitrite and pH. Urine analysis findings were considered abnormal as judged by the investigator. |
| Change From Baseline in Amplitude at Week 26 | Baseline, Week 26 | Abbreviations: MN=Median Nerve; PN=Peroneal Nerve; SN=Sural Nerve; Dig.=Digit; APB=abductor pollicis brevis; EDB=extensor digitorum brevis. |
| Physical Examination Results | Baseline up to Week 26 | Physical examination included general appearance, skin, head, ears, eyes, nose and throat, lymph nodes, heart, lungs, abdomen, extremities/joints, hip, neurological, mental status and, if appropriate, breasts, external genitalia, pelvic and rectal, and in addition weight, height and head circumference were recorded. |
| Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Baseline up to Week 26 | Number of participants with at least 1 shift from baseline to Week 26, are reported. Abbreviations: ALT=Alanine transaminase; CK=Creatine kinase; AP=Amyloid P component; LDH=Lactate dehydrogenase. |
Countries
Denmark
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Cohort 1 Single dose of 25 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), thereafter, received repeated doses of 50 U/kg recombinant human Arylsulphatase A, once in every 2 weeks for a period of 26 weeks, as an IV infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight. | 4 |
| Cohort 2 Repeated doses of 100 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight. | 5 |
| Cohort 3 Repeated doses of 200 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 60 minutes. Dosage adjustment was done monthly to account for changes in body weight. | 4 |
| Total | 13 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 |
|---|---|---|---|---|
| Overall Study | Participant's (guardian's) decision | 0 | 1 | 0 |
Baseline characteristics
| Characteristic | Cohort 1 | Cohort 2 | Cohort 3 | Total |
|---|---|---|---|---|
| Age, Continuous | 36.25 months STANDARD_DEVIATION 9.32 | 41.80 months STANDARD_DEVIATION 10.13 | 30.75 months STANDARD_DEVIATION 7.27 | 36.69 months STANDARD_DEVIATION 9.59 |
| Sex: Female, Male Female | 2 Participants | 3 Participants | 3 Participants | 8 Participants |
| Sex: Female, Male Male | 2 Participants | 2 Participants | 1 Participants | 5 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 | 5 / 5 | 4 / 4 |
| serious Total, serious adverse events | 1 / 4 | 2 / 5 | 2 / 4 |
Outcome results
Primary
Arylsulfatase A (ASA) Activity in Leukocytes
Time frame: Pre-dose and post-dose at 24 hours on Day 0 and at Weeks 8 and 26
Population: Data were not available to report as ASA activity in leukocytes was presented graphically, as per planned analysis.
Primary
Change From Baseline in Cerebrospinal Fluid (CSF) Sulfatide at Week 26
Relative change from baseline at Week 26 was calculated as percentage change from baseline divided by the age-difference in months between first and last visit. Adjusted mean and 95 percent (%) confidence intervals were reported.
Time frame: Baseline, Week 26
Population: ITT
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Cohort 1 | Change From Baseline in Cerebrospinal Fluid (CSF) Sulfatide at Week 26 | 24.55 percent (%) change |
| Cohort 2 | Change From Baseline in Cerebrospinal Fluid (CSF) Sulfatide at Week 26 | -3.77 percent (%) change |
| Cohort 3 | Change From Baseline in Cerebrospinal Fluid (CSF) Sulfatide at Week 26 | -4.32 percent (%) change |
Comparison: The comparisons of the three doses were done using ANOVA model including the baseline measurement as a covariate.p-value: 0.1115ANOVA
Primary
Change From Baseline in Gross Motor Function Measure (GMFM) at Week 26
GMFM was measured using GMFM-88 item scores and summed to calculate a total GMFM-88 score. For each GMFM-88 item, the score was between 0 (minimal) to 3 (maximum). The total GMFM-88 score was between 0 (minimal) and 264 (maximum). The decrease in GMFM score over time indicates worsening of disease over time. Relative change from baseline at Week 26 was calculated as percentage change from baseline divided by the age-difference in months between first and last visit. Adjusted mean and 95 percent (%) confidence intervals were reported.
Time frame: Baseline, Week 26
Population: ITT
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Cohort 1 | Change From Baseline in Gross Motor Function Measure (GMFM) at Week 26 | -2.36 percent (%) change |
| Cohort 2 | Change From Baseline in Gross Motor Function Measure (GMFM) at Week 26 | -8.29 percent (%) change |
| Cohort 3 | Change From Baseline in Gross Motor Function Measure (GMFM) at Week 26 | -10.90 percent (%) change |
Comparison: The comparisons of the three doses were done using analysis of variance (ANOVA) model including the baseline measurement as a covariate.p-value: 0.0737ANOVA
Primary
Change From Baseline in Mullen's Scales of Early Learning at Week 26
Mullen's Scales of Early Learning is used to assess performance and learning ability in young children. The scale consisted of 144 items that had specific scoring criteria for each item. The scores were converted to T-scores with a decrease in score indicating worsening of disease. Relative change from baseline at Week 26 was calculated as percentage change from baseline divided by the age-difference in months between first and last visit. Adjusted mean and 95 percent (%) confidence intervals were reported.
Time frame: Baseline, Week 26
Population: ITT
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Cohort 1 | Change From Baseline in Mullen's Scales of Early Learning at Week 26 | 16.28 percent (%) change |
| Cohort 2 | Change From Baseline in Mullen's Scales of Early Learning at Week 26 | 0.26 percent (%) change |
| Cohort 3 | Change From Baseline in Mullen's Scales of Early Learning at Week 26 | -4.92 percent (%) change |
Comparison: The comparisons of the three doses were done using ANOVA model including the baseline measurement as a covariate.p-value: 0.1268ANOVA
Primary
Maximum Plasma Drug Concentration (Cmax) of Recombinant Human Arylsulphatase A (rhASA)
Time frame: Pre-dose and post-dose at 20, 40, 90 minutes, 3, 6 and 8 hours on Day 0, 40 minutes post-dose at Week 4, Pre-dose and post-dose at 20, 40, 90 minutes, 3, 6 and 8 hours at Week 8
Population: Due to quick disappearance of rhASA from plasma, rhASA levels were not possible to report.
Primary
Number of Participants With Shift From Baseline to Week 26 in Sulfatide Levels in Urine
Number of participants with shifts between negative (value=0) and positive (value=1) values in urine sulfatide levels from baseline at Week 26 is reported.
Time frame: Baseline up to Week 26
Population: ITT. Here, the number of participants analyzed are the participants evaluable for this outcome.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Sulfatide Levels in Urine | Negative(0) to negative(0) | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Sulfatide Levels in Urine | Negative(0) to positive(1) | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Sulfatide Levels in Urine | Positive(1) to negative(0) | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Sulfatide Levels in Urine | Positive(1) to positive(1) | 3 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Sulfatide Levels in Urine | Positive(1) to positive(1) | 2 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Sulfatide Levels in Urine | Negative(0) to negative(0) | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Sulfatide Levels in Urine | Positive(1) to negative(0) | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Sulfatide Levels in Urine | Negative(0) to positive(1) | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Sulfatide Levels in Urine | Positive(1) to positive(1) | 2 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Sulfatide Levels in Urine | Negative(0) to positive(1) | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Sulfatide Levels in Urine | Positive(1) to negative(0) | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Sulfatide Levels in Urine | Negative(0) to negative(0) | 0 participants |
Primary
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
An adverse event (AE) is any untoward, undesired, unplanned clinical event in the form of signs, symptoms, disease, or laboratory or physiological observations occurring in a participant, participating in a clinical study with study drug, regardless of causal relationship. TEAEs were AEs occurred after study drug administration that were absent before treatment or that worsened relative to pre-treatment state, up to Week 28 until evaluation (when last cohort had 26-week evaluation and data management performed within 4 weeks) completed.
Time frame: From study drug administration up to Week 28
Population: Intent-to-treat (ITT) population included all participants who received at least 1 dose of study drug.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Cohort 1 | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | 4 participants |
| Cohort 2 | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | 5 participants |
| Cohort 3 | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | 4 participants |
Secondary
Change From Baseline in Nerve Conduction Velocity at Week 26
An electrophysiological evaluation using standard electrophysiological and electromyography to measure the speed and extent of nerve conduction and units are expressed in meters per second. Abbreviations: MN=Median Nerve; PN=Peroneal Nerve; SN=Sural Nerve; Dig.=Digit; FH=fibular hemimelia; L LM=left lateral medial; R LM=right lateral medial; MC=medial collateral.
Time frame: Baseline, Week 26
Population: ITT. Here, N signifies the number of participants who were evaluable for the respective category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Cohort 1 | Change From Baseline in Nerve Conduction Velocity at Week 26 | MN, Elbow Wrist: Baseline (N=4,5,4) | 20.38 meters per second | Standard Deviation 6.99 |
| Cohort 1 | Change From Baseline in Nerve Conduction Velocity at Week 26 | MN, Elbow Wrist: Change at Week 26 (N=4,4,4) | -4.00 meters per second | Standard Deviation 1.1 |
| Cohort 1 | Change From Baseline in Nerve Conduction Velocity at Week 26 | MN, Dig. II Wrist: Baseline (N=3,5,4) | 39.83 meters per second | Standard Deviation 7.22 |
| Cohort 1 | Change From Baseline in Nerve Conduction Velocity at Week 26 | MN, Dig. II Wrist: Change at Week 26 (N=3,4,4) | -11.7 meters per second | Standard Deviation 5.78 |
| Cohort 1 | Change From Baseline in Nerve Conduction Velocity at Week 26 | PN, Dig. Ankle FH: Baseline (N=4,4,4) | 20.70 meters per second | Standard Deviation 9.08 |
| Cohort 1 | Change From Baseline in Nerve Conduction Velocity at Week 26 | PN, Dig. Ankle FH: Change at Week 26 (N=4,3,4) | -7.85 meters per second | Standard Deviation 3.92 |
| Cohort 1 | Change From Baseline in Nerve Conduction Velocity at Week 26 | SN, Sensory L LM - MC: Baseline (N=4,4,4) | 26.88 meters per second | Standard Deviation 9.29 |
| Cohort 1 | Change From Baseline in Nerve Conduction Velocity at Week 26 | SN, Sensory L LM - MC: Change at Week 26 (N=3,3,4) | -5.57 meters per second | Standard Deviation 4.18 |
| Cohort 1 | Change From Baseline in Nerve Conduction Velocity at Week 26 | SN, Sensory R LM - MC: Baseline (N=4,4,4) | 31.35 meters per second | Standard Deviation 6.46 |
| Cohort 1 | Change From Baseline in Nerve Conduction Velocity at Week 26 | SN, Sensory R LM - MC: Change at Week 26 (N=0,0,0) | NA meters per second | — |
| Cohort 2 | Change From Baseline in Nerve Conduction Velocity at Week 26 | SN, Sensory R LM - MC: Baseline (N=4,4,4) | 34.58 meters per second | Standard Deviation 17.74 |
| Cohort 2 | Change From Baseline in Nerve Conduction Velocity at Week 26 | MN, Elbow Wrist: Baseline (N=4,5,4) | 25.78 meters per second | Standard Deviation 17.72 |
| Cohort 2 | Change From Baseline in Nerve Conduction Velocity at Week 26 | PN, Dig. Ankle FH: Change at Week 26 (N=4,3,4) | -2.43 meters per second | Standard Deviation 1.59 |
| Cohort 2 | Change From Baseline in Nerve Conduction Velocity at Week 26 | PN, Dig. Ankle FH: Baseline (N=4,4,4) | 32.23 meters per second | Standard Deviation 21.41 |
| Cohort 2 | Change From Baseline in Nerve Conduction Velocity at Week 26 | MN, Elbow Wrist: Change at Week 26 (N=4,4,4) | 2.62 meters per second | Standard Deviation 4.42 |
| Cohort 2 | Change From Baseline in Nerve Conduction Velocity at Week 26 | SN, Sensory R LM - MC: Change at Week 26 (N=0,0,0) | NA meters per second | — |
| Cohort 2 | Change From Baseline in Nerve Conduction Velocity at Week 26 | SN, Sensory L LM - MC: Change at Week 26 (N=3,3,4) | 3.70 meters per second | Standard Deviation 9.79 |
| Cohort 2 | Change From Baseline in Nerve Conduction Velocity at Week 26 | MN, Dig. II Wrist: Baseline (N=3,5,4) | 36.82 meters per second | Standard Deviation 16.64 |
| Cohort 2 | Change From Baseline in Nerve Conduction Velocity at Week 26 | SN, Sensory L LM - MC: Baseline (N=4,4,4) | 36.13 meters per second | Standard Deviation 20.61 |
| Cohort 2 | Change From Baseline in Nerve Conduction Velocity at Week 26 | MN, Dig. II Wrist: Change at Week 26 (N=3,4,4) | -0.25 meters per second | Standard Deviation 4.39 |
| Cohort 3 | Change From Baseline in Nerve Conduction Velocity at Week 26 | SN, Sensory L LM - MC: Change at Week 26 (N=3,3,4) | -9.33 meters per second | Standard Deviation 10.64 |
| Cohort 3 | Change From Baseline in Nerve Conduction Velocity at Week 26 | MN, Dig. II Wrist: Change at Week 26 (N=3,4,4) | -1.33 meters per second | Standard Deviation 4.72 |
| Cohort 3 | Change From Baseline in Nerve Conduction Velocity at Week 26 | PN, Dig. Ankle FH: Baseline (N=4,4,4) | 14.13 meters per second | Standard Deviation 5.92 |
| Cohort 3 | Change From Baseline in Nerve Conduction Velocity at Week 26 | PN, Dig. Ankle FH: Change at Week 26 (N=4,3,4) | -2.65 meters per second | Standard Deviation 3.61 |
| Cohort 3 | Change From Baseline in Nerve Conduction Velocity at Week 26 | SN, Sensory R LM - MC: Baseline (N=4,4,4) | 27.48 meters per second | Standard Deviation 13.91 |
| Cohort 3 | Change From Baseline in Nerve Conduction Velocity at Week 26 | SN, Sensory L LM - MC: Baseline (N=4,4,4) | 29.18 meters per second | Standard Deviation 15.93 |
| Cohort 3 | Change From Baseline in Nerve Conduction Velocity at Week 26 | MN, Elbow Wrist: Baseline (N=4,5,4) | 15.95 meters per second | Standard Deviation 5.68 |
| Cohort 3 | Change From Baseline in Nerve Conduction Velocity at Week 26 | SN, Sensory R LM - MC: Change at Week 26 (N=0,0,0) | NA meters per second | — |
| Cohort 3 | Change From Baseline in Nerve Conduction Velocity at Week 26 | MN, Elbow Wrist: Change at Week 26 (N=4,4,4) | -3.65 meters per second | Standard Deviation 3.33 |
| Cohort 3 | Change From Baseline in Nerve Conduction Velocity at Week 26 | MN, Dig. II Wrist: Baseline (N=3,5,4) | 23.30 meters per second | Standard Deviation 11.99 |
Secondary
Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26
PEDI is used for the clinical evaluation of functional capabilities, performance and changes in functional skills in children with disabilities. It consisted of 20 items scored on a scale from 0 (total assistance) to 5 (independent). Total score ranged from 0-100 with higher scores indicating better functioning. None, child, rehab, extensive are items in 3 domains (self-care, mobility and social functioning).
Time frame: Baseline, Week 26
Population: ITT. Here, N signifies the number of participants who were evaluable for the respective category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Cohort 1 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Self-care, Extensive: Baseline (N=4,5,4) | 0 units on a scale | Standard Deviation 0 |
| Cohort 1 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Self-care, None: Change at Week 26 (N=4,4,4) | -0.75 units on a scale | Standard Deviation 1.5 |
| Cohort 1 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Self-care, Child: Baseline (N=4,5,4) | 1.75 units on a scale | Standard Deviation 0.96 |
| Cohort 1 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Self-care, Child: Change at Week 26 (N=4,4,4) | 0.75 units on a scale | Standard Deviation 1.5 |
| Cohort 1 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Self-care, Rehab: Baseline (N=4,5,4) | 0 units on a scale | Standard Deviation 0 |
| Cohort 1 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Self-care, Rehab: Change at Week 26 (N=4,4,4) | 0 units on a scale | Standard Deviation 0 |
| Cohort 1 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Self-care, None: Baseline (N=4,5,4) | 6.25 units on a scale | Standard Deviation 0.96 |
| Cohort 1 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Self-care, Extensive: Change at Week 26 (N=4,4,4) | 0 units on a scale | Standard Deviation 0 |
| Cohort 1 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Mobility, None: Baseline (N=4,5,4) | 4.25 units on a scale | Standard Deviation 1.89 |
| Cohort 1 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Mobility, None: Change at Week 26 (N=4,4,4) | -0.75 units on a scale | Standard Deviation 1.71 |
| Cohort 1 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Mobility, Child: Baseline (N=4,5,4) | 2.50 units on a scale | Standard Deviation 1.91 |
| Cohort 1 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Mobility, Child: Change at Week 26 (N=4,4,4) | 0.00 units on a scale | Standard Deviation 1.41 |
| Cohort 1 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Mobility, Rehab: Baseline (N=4,5,4) | 0.00 units on a scale | Standard Deviation 0 |
| Cohort 1 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Mobility, Rehab: Change at Week 26 (N=4,4,4) | 1.00 units on a scale | Standard Deviation 0.82 |
| Cohort 1 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Mobility, Extensive: Baseline (N=4,5,4) | 0.00 units on a scale | Standard Deviation 0 |
| Cohort 1 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Mobility, Extensive: Change at Week 26 (N=4,4,4) | 0.00 units on a scale | Standard Deviation 0 |
| Cohort 1 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Social, None: Baseline (N=4,5,4) | 4.25 units on a scale | Standard Deviation 0.96 |
| Cohort 1 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Social, None: Change at Week 26 (N=4,4,4) | 0.75 units on a scale | Standard Deviation 0.96 |
| Cohort 1 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Social, Child: Baseline (N=4,5,4) | 1.50 units on a scale | Standard Deviation 2.38 |
| Cohort 1 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Social, Child: Change at Week 26 (N=4,4,4) | -1.50 units on a scale | Standard Deviation 2.38 |
| Cohort 1 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Social, Rehab: Baseline (N=4,5,4) | 0.00 units on a scale | Standard Deviation 0 |
| Cohort 1 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Social, Rehab: Change at Week 26 (N=4,4,4) | 0.00 units on a scale | Standard Deviation 0 |
| Cohort 1 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Social, Extensive: Baseline (N=4,5,4) | 0.00 units on a scale | Standard Deviation 0 |
| Cohort 1 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Social, Extensive: Change at Week 26 (N=4,4,4) | 0.00 units on a scale | Standard Deviation 0 |
| Cohort 2 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Social, Rehab: Change at Week 26 (N=4,4,4) | 0.00 units on a scale | Standard Deviation 0 |
| Cohort 2 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Self-care, None: Baseline (N=4,5,4) | 6.00 units on a scale | Standard Deviation 1.41 |
| Cohort 2 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Mobility, Rehab: Baseline (N=4,5,4) | 1.00 units on a scale | Standard Deviation 1.41 |
| Cohort 2 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Social, None: Baseline (N=4,5,4) | 4.60 units on a scale | Standard Deviation 0.55 |
| Cohort 2 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Self-care, None: Change at Week 26 (N=4,4,4) | -1.50 units on a scale | Standard Deviation 1.73 |
| Cohort 2 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Mobility, Child: Baseline (N=4,5,4) | 2.40 units on a scale | Standard Deviation 1.67 |
| Cohort 2 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Social, Rehab: Baseline (N=4,5,4) | 0.40 units on a scale | Standard Deviation 0.55 |
| Cohort 2 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Self-care, Child: Baseline (N=4,5,4) | 2.00 units on a scale | Standard Deviation 1.41 |
| Cohort 2 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Mobility, Rehab: Change at Week 26 (N=4,4,4) | 0.25 units on a scale | Standard Deviation 0.5 |
| Cohort 2 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Social, Extensive: Baseline (N=4,5,4) | 0.00 units on a scale | Standard Deviation 0 |
| Cohort 2 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Self-care, Child: Change at Week 26 (N=4,4,4) | 1.25 units on a scale | Standard Deviation 1.5 |
| Cohort 2 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Mobility, None: Change at Week 26 (N=4,4,4) | -0.50 units on a scale | Standard Deviation 1.29 |
| Cohort 2 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Social, None: Change at Week 26 (N=4,4,4) | -0.25 units on a scale | Standard Deviation 0.5 |
| Cohort 2 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Self-care, Rehab: Baseline (N=4,5,4) | 0 units on a scale | Standard Deviation 0 |
| Cohort 2 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Mobility, Extensive: Baseline (N=4,5,4) | 0.00 units on a scale | Standard Deviation 0 |
| Cohort 2 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Mobility, Child: Change at Week 26 (N=4,4,4) | 0.25 units on a scale | Standard Deviation 1.26 |
| Cohort 2 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Self-care, Rehab: Change at Week 26 (N=4,4,4) | 0 units on a scale | Standard Deviation 0 |
| Cohort 2 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Mobility, None: Baseline (N=4,5,4) | 3.60 units on a scale | Standard Deviation 1.14 |
| Cohort 2 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Social, Extensive: Change at Week 26 (N=4,4,4) | 0.25 units on a scale | Standard Deviation 0.5 |
| Cohort 2 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Self-care, Extensive: Baseline (N=4,5,4) | 0.20 units on a scale | Standard Deviation 0.45 |
| Cohort 2 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Mobility, Extensive: Change at Week 26 (N=4,4,4) | 0.00 units on a scale | Standard Deviation 0 |
| Cohort 2 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Social, Child: Baseline (N=4,5,4) | 0.00 units on a scale | Standard Deviation 0 |
| Cohort 2 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Self-care, Extensive: Change at Week 26 (N=4,4,4) | 0.25 units on a scale | Standard Deviation 0.5 |
| Cohort 2 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Social, Child: Change at Week 26 (N=4,4,4) | 0.00 units on a scale | Standard Deviation 0 |
| Cohort 3 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Self-care, Extensive: Change at Week 26 (N=4,4,4) | 0.50 units on a scale | Standard Deviation 0.58 |
| Cohort 3 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Mobility, None: Baseline (N=4,5,4) | 5.00 units on a scale | Standard Deviation 0.82 |
| Cohort 3 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Mobility, None: Change at Week 26 (N=4,4,4) | -1.25 units on a scale | Standard Deviation 1.71 |
| Cohort 3 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Mobility, Child: Baseline (N=4,5,4) | 1.25 units on a scale | Standard Deviation 1.26 |
| Cohort 3 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Social, Child: Change at Week 26 (N=4,4,4) | -0.25 units on a scale | Standard Deviation 0.5 |
| Cohort 3 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Mobility, Child: Change at Week 26 (N=4,4,4) | -0.50 units on a scale | Standard Deviation 0.58 |
| Cohort 3 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Social, Extensive: Baseline (N=4,5,4) | 0.00 units on a scale | Standard Deviation 0 |
| Cohort 3 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Mobility, Rehab: Baseline (N=4,5,4) | 0.75 units on a scale | Standard Deviation 0.96 |
| Cohort 3 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Mobility, Rehab: Change at Week 26 (N=4,4,4) | 0.50 units on a scale | Standard Deviation 1.73 |
| Cohort 3 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Social, Rehab: Baseline (N=4,5,4) | 0.00 units on a scale | Standard Deviation 0 |
| Cohort 3 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Mobility, Extensive: Baseline (N=4,5,4) | 0.00 units on a scale | Standard Deviation 0 |
| Cohort 3 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Social, Extensive: Change at Week 26 (N=4,4,4) | 0.00 units on a scale | Standard Deviation 0 |
| Cohort 3 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Mobility, Extensive: Change at Week 26 (N=4,4,4) | 1.25 units on a scale | Standard Deviation 1.5 |
| Cohort 3 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Self-care, None: Baseline (N=4,5,4) | 4.75 units on a scale | Standard Deviation 2.06 |
| Cohort 3 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Self-care, None: Change at Week 26 (N=4,4,4) | -1.50 units on a scale | Standard Deviation 1.91 |
| Cohort 3 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Social, None: Baseline (N=4,5,4) | 4.75 units on a scale | Standard Deviation 0.5 |
| Cohort 3 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Self-care, Child: Baseline (N=4,5,4) | 3.25 units on a scale | Standard Deviation 2.06 |
| Cohort 3 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Social, Rehab: Change at Week 26 (N=4,4,4) | 0.00 units on a scale | Standard Deviation 0 |
| Cohort 3 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Self-care, Child: Change at Week 26 (N=4,4,4) | 0.75 units on a scale | Standard Deviation 2.22 |
| Cohort 3 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Self-care, Rehab: Baseline (N=4,5,4) | 0 units on a scale | Standard Deviation 0 |
| Cohort 3 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Social, None: Change at Week 26 (N=4,4,4) | 0.25 units on a scale | Standard Deviation 0.5 |
| Cohort 3 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Self-care, Rehab: Change at Week 26 (N=4,4,4) | 0 units on a scale | Standard Deviation 0 |
| Cohort 3 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Self-care, Extensive: Baseline (N=4,5,4) | 0 units on a scale | Standard Deviation 0 |
| Cohort 3 | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Social, Child: Baseline (N=4,5,4) | 0.25 units on a scale | Standard Deviation 0.5 |
Secondary
Number of Participants Who Had Undergone Nerve Biopsy and Had a Normal Nerve at Both Baseline and Week 26
Time frame: Baseline, Week 26
Population: ITT.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Cohort 1 | Number of Participants Who Had Undergone Nerve Biopsy and Had a Normal Nerve at Both Baseline and Week 26 | Baseline | 0 participants |
| Cohort 1 | Number of Participants Who Had Undergone Nerve Biopsy and Had a Normal Nerve at Both Baseline and Week 26 | Week 26 | 0 participants |
| Cohort 2 | Number of Participants Who Had Undergone Nerve Biopsy and Had a Normal Nerve at Both Baseline and Week 26 | Baseline | 2 participants |
| Cohort 2 | Number of Participants Who Had Undergone Nerve Biopsy and Had a Normal Nerve at Both Baseline and Week 26 | Week 26 | 2 participants |
| Cohort 3 | Number of Participants Who Had Undergone Nerve Biopsy and Had a Normal Nerve at Both Baseline and Week 26 | Baseline | 0 participants |
| Cohort 3 | Number of Participants Who Had Undergone Nerve Biopsy and Had a Normal Nerve at Both Baseline and Week 26 | Week 26 | 0 participants |
Secondary
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores
Loes scoring system is used to grade the demyelinating abnormalities on brain MRI. A total of 17 locations of the brain were scored from 0 (normal appearance) to 2 (dense appearance). The total score ranged from 0 to 34 with a score of 14 or greater being considered severe. Number of participants with any shift of score between 0 to 2 for each of the 17 locations (Parieto Occipital \[PO\]-Periventricular \[P\], Central \[C\], Subcortical \[Sc\]; Anterior Temporal \[AT\]-P, C, Sc; Frontal \[F\]-P, C, Sc; Corpus Callosum \[CC\]-Splenium \[S\], Genus \[G\]; Projection Fibers \[PF\]-Capsular interna \[CI\] ant, CI post, Brainstem \[B\]; Cerebellum \[Cb\]-Cortex, Atrophy; Basal Ganglia \[BG\]-BG, Thalamus \[T\]; Cerebral Atrophy \[CA\]-CA), are only reported.
Time frame: Baseline up to Week 26
Population: ITT. Here, number of participants analyzed in the Cohort 2 are the participants evaluable for this outcome.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | F, C: 1 to 2 | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Bg, Bg: 1 to 1 | 2 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, CI post: 2 to 2 | 2 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | F, C: 2 to 2 | 3 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PO, Sc: 2 to 2 | 3 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, CI post: 2 to 1 | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | F, Sc: 0 to 0 | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CA, CA: 0 to 1 | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, CI post: 1 to 2 | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | F, Sc: 0 to 2 | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Bg, Bg: 1 to 0 | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, CI post: 1 to 1 | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | F, Sc: 1 to 2 | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, P: 0 to 2 | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, CI post: 0 to 1 | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | F, Sc: 2 to 2 | 2 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PO, Sc: 0 to 0 | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, CI post: 0 to 0 | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CC, S: 1 to 0 | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Bg, Bg: 0 to 1 | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, CI ant: 1 to 1 | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CC, S: 1 to 2 | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, P: 1 to 2 | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, CI ant: 0 to 1 | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CC, S: 2 to 0 | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PO, P: 2 to 2 | 3 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, CI ant: 0 to 0 | 2 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CC, S: 2 to 1 | 2 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Bg, Bg: 0 to 0 | 2 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CC, G: 2 to 2 | 2 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CC, S: 2 to 2 | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, P: 2 to 2 | 3 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CC, G: 2 to 1 | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CC, G: 0 to 2 | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CA, CA: 0 to 0 | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CC, G: 1 to 2 | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CC, G: 1 to 1 | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Cb, Atrophy: 1 to 1 | 2 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, C: 0 to 2 | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PO, Sc: 0 to 1 | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Cb, Atrophy: 0 to 1 | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, C: 1 to 2 | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PO, P: 1 to 2 | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Cb, Atrophy: 0 to 0 | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, C: 2 to 2 | 3 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Bg, T: 1 to 1 | 2 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Cb, Cortex: 1 to 1 | 2 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, Sc: 0 to 0 | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PO, Sc: 0 to 2 | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Cb, Cortex: 0 to 1 | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, Sc: 0 to 1 | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CA, CA: 1 to 1 | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Cb, Cortex: 0 to 0 | 2 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, Sc: 0 to 2 | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | F, P: 1 to 2 | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, B: 2 to 2 | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, Sc: 1 to 1 | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Bg, T: 1 to 0 | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, B: 1 to 2 | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, Sc: 1 to 2 | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PO, Sc: 1 to 1 | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, B: 1 to 1 | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, Sc: 2 to 2 | 2 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PO, C: 2 to 2 | 4 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, B: 1 to 0 | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | F, P: 0 to 2 | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Bg, T: 0 to 0 | 2 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, B: 0 to 2 | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PO, Sc: 1 to 2 | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, B: 0 to 1 | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | F, P: 2 to 2 | 3 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CA, CA: 1 to 2 | 2 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, B: 0 to 0 | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, Sc: 0 to 2 | 2 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, CI ant: 0 to 0 | 3 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PO, P: 1 to 2 | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PO, P: 2 to 2 | 4 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PO, C: 2 to 2 | 4 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PO, Sc: 0 to 0 | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PO, Sc: 0 to 1 | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PO, Sc: 0 to 2 | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PO, Sc: 1 to 1 | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PO, Sc: 1 to 2 | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PO, Sc: 2 to 2 | 2 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, P: 0 to 2 | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, P: 1 to 2 | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, P: 2 to 2 | 3 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, C: 0 to 2 | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, C: 1 to 2 | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, C: 2 to 2 | 4 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, Sc: 0 to 0 | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, Sc: 0 to 1 | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, Sc: 1 to 1 | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, Sc: 1 to 2 | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, Sc: 2 to 2 | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | F, P: 0 to 2 | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | F, P: 1 to 2 | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | F, P: 2 to 2 | 3 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | F, C: 1 to 2 | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | F, C: 2 to 2 | 4 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | F, Sc: 0 to 0 | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | F, Sc: 0 to 2 | 2 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | F, Sc: 1 to 2 | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | F, Sc: 2 to 2 | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CC, S: 1 to 0 | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CC, S: 1 to 2 | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CC, S: 2 to 0 | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CC, S: 2 to 1 | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CC, S: 2 to 2 | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CC, G: 0 to 2 | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CC, G: 1 to 1 | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CC, G: 1 to 2 | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CC, G: 2 to 1 | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CC, G: 2 to 2 | 2 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, CI ant: 0 to 1 | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, CI ant: 1 to 1 | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, CI post: 0 to 0 | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, CI post: 0 to 1 | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, CI post: 1 to 1 | 2 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, CI post: 1 to 2 | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, CI post: 2 to 1 | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, CI post: 2 to 2 | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, B: 0 to 0 | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, B: 0 to 1 | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, B: 0 to 2 | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, B: 1 to 0 | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, B: 1 to 1 | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, B: 1 to 2 | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, B: 2 to 2 | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Cb, Cortex: 0 to 0 | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Cb, Cortex: 0 to 1 | 2 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Cb, Cortex: 1 to 1 | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Cb, Atrophy: 0 to 0 | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Cb, Atrophy: 0 to 1 | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Cb, Atrophy: 1 to 1 | 2 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Bg, Bg: 0 to 0 | 3 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Bg, Bg: 0 to 1 | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Bg, Bg: 1 to 0 | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Bg, Bg: 1 to 1 | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Bg, T: 0 to 0 | 3 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Bg, T: 1 to 0 | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Bg, T: 1 to 1 | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CA, CA: 0 to 0 | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CA, CA: 0 to 1 | 2 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CA, CA: 1 to 1 | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CA, CA: 1 to 2 | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, CI post: 2 to 2 | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | F, P: 2 to 2 | 2 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PO, Sc: 1 to 2 | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, B: 0 to 0 | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | F, P: 1 to 2 | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PO, P: 1 to 2 | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, B: 0 to 1 | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | F, P: 0 to 2 | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Bg, Bg: 1 to 1 | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, B: 0 to 2 | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, Sc: 2 to 2 | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PO, Sc: 1 to 1 | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, B: 1 to 0 | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, Sc: 1 to 2 | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CA, CA: 0 to 1 | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, B: 1 to 1 | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, Sc: 1 to 1 | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Bg, T: 0 to 0 | 3 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, B: 1 to 2 | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, Sc: 0 to 2 | 2 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PO, Sc: 0 to 2 | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, B: 2 to 2 | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, Sc: 0 to 1 | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PO, P: 2 to 2 | 3 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Cb, Cortex: 0 to 0 | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, Sc: 0 to 0 | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Bg, T: 1 to 0 | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Cb, Cortex: 0 to 1 | 2 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, C: 2 to 2 | 3 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PO, Sc: 0 to 1 | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Cb, Cortex: 1 to 1 | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, C: 1 to 2 | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CA, CA: 1 to 2 | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Cb, Atrophy: 0 to 0 | 3 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, C: 0 to 2 | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Bg, T: 1 to 1 | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Cb, Atrophy: 0 to 1 | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, P: 2 to 2 | 2 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CC, G: 1 to 1 | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CC, G: 0 to 2 | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PO, Sc: 0 to 0 | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, CI post: 1 to 2 | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CC, G: 1 to 2 | 2 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CC, S: 2 to 2 | 2 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Cb, Atrophy: 1 to 1 | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CC, G: 2 to 1 | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CC, S: 2 to 1 | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, P: 1 to 2 | 2 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CC, G: 2 to 2 | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CC, S: 2 to 0 | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, CI ant: 0 to 0 | 4 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CC, S: 1 to 2 | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CA, CA: 1 to 1 | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, CI ant: 0 to 1 | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CC, S: 1 to 0 | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Bg, Bg: 0 to 0 | 2 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, CI ant: 1 to 1 | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | F, Sc: 2 to 2 | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | AT, P: 0 to 2 | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, CI post: 0 to 0 | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | F, Sc: 1 to 2 | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | CA, CA: 0 to 0 | 2 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, CI post: 0 to 1 | 2 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | F, Sc: 0 to 2 | 2 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Bg, Bg: 0 to 1 | 2 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, CI post: 1 to 1 | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | F, Sc: 0 to 0 | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PO, Sc: 2 to 2 | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | F, C: 2 to 2 | 2 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PO, C: 2 to 2 | 4 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | PF, CI post: 2 to 1 | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | F, C: 1 to 2 | 2 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Bg, Bg: 1 to 0 | 0 participants |
Other Pre-specified
Change From Baseline in Amplitude at Week 26
Abbreviations: MN=Median Nerve; PN=Peroneal Nerve; SN=Sural Nerve; Dig.=Digit; APB=abductor pollicis brevis; EDB=extensor digitorum brevis.
Time frame: Baseline, Week 26
Population: ITT. Here, N signifies the number of participants who were evaluable for the respective category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Cohort 1 | Change From Baseline in Amplitude at Week 26 | SN, Sensory L LM - MC: Baseline (N=4,4,4) | 5.25 millivolts | Standard Deviation 6.31 |
| Cohort 1 | Change From Baseline in Amplitude at Week 26 | MN, Dig. II Wrist: Change at Week 26 (N=4,4,4) | -0.32 millivolts | Standard Deviation 1 |
| Cohort 1 | Change From Baseline in Amplitude at Week 26 | MN, Elbow-APB: Baseline (N=4,5,4) | 3.85 millivolts | Standard Deviation 1.23 |
| Cohort 1 | Change From Baseline in Amplitude at Week 26 | PN, FH EDB: Change at Week 26 (N=4,3,4) | -1.68 millivolts | Standard Deviation 2.06 |
| Cohort 1 | Change From Baseline in Amplitude at Week 26 | PN, Ankle EDB: Baseline (N=4,4,4) | 1.85 millivolts | Standard Deviation 0.91 |
| Cohort 1 | Change From Baseline in Amplitude at Week 26 | MN, Wrist-APB: Change at Week 26 (N=4,4,4) | 0.00 millivolts | Standard Deviation 2.84 |
| Cohort 1 | Change From Baseline in Amplitude at Week 26 | PN, FH EDB: Baseline (N=4,4,4) | 2.43 millivolts | Standard Deviation 1.8 |
| Cohort 1 | Change From Baseline in Amplitude at Week 26 | PN, Ankle EDB: Change at Week 26 (N=4,3,4) | -0.72 millivolts | Standard Deviation 1.66 |
| Cohort 1 | Change From Baseline in Amplitude at Week 26 | SN, Sensory L LM - MC: Change at Week 26 (N=4,3,4) | -2.45 millivolts | Standard Deviation 4.18 |
| Cohort 1 | Change From Baseline in Amplitude at Week 26 | MN, Elbow-APB: Change at Week 26 (N=4,4,4) | 0.15 millivolts | Standard Deviation 2.05 |
| Cohort 1 | Change From Baseline in Amplitude at Week 26 | SN, Sensory R LM - MC: Change at Week 26 (N=0,0,0) | NA millivolts | — |
| Cohort 1 | Change From Baseline in Amplitude at Week 26 | MN, Wrist-APB: Baseline (N=4,5,4) | 5.63 millivolts | Standard Deviation 1.73 |
| Cohort 1 | Change From Baseline in Amplitude at Week 26 | MN, Dig. II Wrist: Baseline (N=4,5,4) | 2.68 millivolts | Standard Deviation 1.3 |
| Cohort 1 | Change From Baseline in Amplitude at Week 26 | SN, Sensory R LM - MC: Baseline (N=4,4,4) | 5.35 millivolts | Standard Deviation 5.45 |
| Cohort 2 | Change From Baseline in Amplitude at Week 26 | SN, Sensory L LM - MC: Baseline (N=4,4,4) | 41.18 millivolts | Standard Deviation 51.5 |
| Cohort 2 | Change From Baseline in Amplitude at Week 26 | MN, Wrist-APB: Baseline (N=4,5,4) | 7.88 millivolts | Standard Deviation 2.2 |
| Cohort 2 | Change From Baseline in Amplitude at Week 26 | MN, Wrist-APB: Change at Week 26 (N=4,4,4) | 0.65 millivolts | Standard Deviation 0.74 |
| Cohort 2 | Change From Baseline in Amplitude at Week 26 | MN, Elbow-APB: Baseline (N=4,5,4) | 6.34 millivolts | Standard Deviation 2.71 |
| Cohort 2 | Change From Baseline in Amplitude at Week 26 | MN, Elbow-APB: Change at Week 26 (N=4,4,4) | 0.33 millivolts | Standard Deviation 0.57 |
| Cohort 2 | Change From Baseline in Amplitude at Week 26 | MN, Dig. II Wrist: Baseline (N=4,5,4) | 11.64 millivolts | Standard Deviation 14.89 |
| Cohort 2 | Change From Baseline in Amplitude at Week 26 | MN, Dig. II Wrist: Change at Week 26 (N=4,4,4) | 3.53 millivolts | Standard Deviation 6.6 |
| Cohort 2 | Change From Baseline in Amplitude at Week 26 | PN, Ankle EDB: Baseline (N=4,4,4) | 5.40 millivolts | Standard Deviation 2.55 |
| Cohort 2 | Change From Baseline in Amplitude at Week 26 | PN, Ankle EDB: Change at Week 26 (N=4,3,4) | -0.43 millivolts | Standard Deviation 1.56 |
| Cohort 2 | Change From Baseline in Amplitude at Week 26 | PN, FH EDB: Baseline (N=4,4,4) | 4.98 millivolts | Standard Deviation 2.95 |
| Cohort 2 | Change From Baseline in Amplitude at Week 26 | PN, FH EDB: Change at Week 26 (N=4,3,4) | -0.63 millivolts | Standard Deviation 1.46 |
| Cohort 2 | Change From Baseline in Amplitude at Week 26 | SN, Sensory L LM - MC: Change at Week 26 (N=4,3,4) | 0.53 millivolts | Standard Deviation 21.75 |
| Cohort 2 | Change From Baseline in Amplitude at Week 26 | SN, Sensory R LM - MC: Baseline (N=4,4,4) | 50.05 millivolts | Standard Deviation 56.13 |
| Cohort 2 | Change From Baseline in Amplitude at Week 26 | SN, Sensory R LM - MC: Change at Week 26 (N=0,0,0) | NA millivolts | — |
| Cohort 3 | Change From Baseline in Amplitude at Week 26 | SN, Sensory R LM - MC: Baseline (N=4,4,4) | 1.73 millivolts | Standard Deviation 2.01 |
| Cohort 3 | Change From Baseline in Amplitude at Week 26 | PN, FH EDB: Change at Week 26 (N=4,3,4) | 0.16 millivolts | Standard Deviation 0.75 |
| Cohort 3 | Change From Baseline in Amplitude at Week 26 | MN, Dig. II Wrist: Baseline (N=4,5,4) | 1.78 millivolts | Standard Deviation 0.83 |
| Cohort 3 | Change From Baseline in Amplitude at Week 26 | MN, Elbow-APB: Change at Week 26 (N=4,4,4) | -0.55 millivolts | Standard Deviation 0.65 |
| Cohort 3 | Change From Baseline in Amplitude at Week 26 | SN, Sensory L LM - MC: Baseline (N=4,4,4) | 1.65 millivolts | Standard Deviation 2.28 |
| Cohort 3 | Change From Baseline in Amplitude at Week 26 | MN, Elbow-APB: Baseline (N=4,5,4) | 2.65 millivolts | Standard Deviation 0.6 |
| Cohort 3 | Change From Baseline in Amplitude at Week 26 | MN, Wrist-APB: Baseline (N=4,5,4) | 4.30 millivolts | Standard Deviation 1.05 |
| Cohort 3 | Change From Baseline in Amplitude at Week 26 | SN, Sensory L LM - MC: Change at Week 26 (N=4,3,4) | -0.78 millivolts | Standard Deviation 2.34 |
| Cohort 3 | Change From Baseline in Amplitude at Week 26 | MN, Wrist-APB: Change at Week 26 (N=4,4,4) | -0.35 millivolts | Standard Deviation 1.66 |
| Cohort 3 | Change From Baseline in Amplitude at Week 26 | PN, Ankle EDB: Change at Week 26 (N=4,3,4) | 0.08 millivolts | Standard Deviation 0.96 |
| Cohort 3 | Change From Baseline in Amplitude at Week 26 | PN, Ankle EDB: Baseline (N=4,4,4) | 1.30 millivolts | Standard Deviation 0.68 |
| Cohort 3 | Change From Baseline in Amplitude at Week 26 | SN, Sensory R LM - MC: Change at Week 26 (N=0,0,0) | NA millivolts | — |
| Cohort 3 | Change From Baseline in Amplitude at Week 26 | PN, FH EDB: Baseline (N=4,4,4) | 0.95 millivolts | Standard Deviation 0.74 |
| Cohort 3 | Change From Baseline in Amplitude at Week 26 | MN, Dig. II Wrist: Change at Week 26 (N=4,4,4) | -0.93 millivolts | Standard Deviation 0.85 |
Other Pre-specified
Change From Baseline in Chitotriosidase at Week 26
Time frame: Baseline, Week 26
Population: ITT. Here, N signifies the number of participants who were evaluable for the respective category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Cohort 1 | Change From Baseline in Chitotriosidase at Week 26 | Baseline (N=4, 5, 4) | 924.0 nanomole/hour/milliliter | Standard Deviation 1431 |
| Cohort 1 | Change From Baseline in Chitotriosidase at Week 26 | Change at Week 26 (N=4, 4, 4) | -76.0 nanomole/hour/milliliter | Standard Deviation 393.9 |
| Cohort 2 | Change From Baseline in Chitotriosidase at Week 26 | Baseline (N=4, 5, 4) | 1481 nanomole/hour/milliliter | Standard Deviation 1165 |
| Cohort 2 | Change From Baseline in Chitotriosidase at Week 26 | Change at Week 26 (N=4, 4, 4) | -228 nanomole/hour/milliliter | Standard Deviation 692.7 |
| Cohort 3 | Change From Baseline in Chitotriosidase at Week 26 | Baseline (N=4, 5, 4) | 367.0 nanomole/hour/milliliter | Standard Deviation 179 |
| Cohort 3 | Change From Baseline in Chitotriosidase at Week 26 | Change at Week 26 (N=4, 4, 4) | 94.75 nanomole/hour/milliliter | Standard Deviation 103.6 |
Other Pre-specified
Change From Baseline in Neurofilament Proteins (NFP), Glial Fibrillary Acidic Protein (GFAP) and Tauprotein in Cerebrospinal Fluid (CSF) at Week 26
Time frame: Baseline, Week 26
Population: ITT. Here, N signifies the number of participants who were evaluable for the respective category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Cohort 1 | Change From Baseline in Neurofilament Proteins (NFP), Glial Fibrillary Acidic Protein (GFAP) and Tauprotein in Cerebrospinal Fluid (CSF) at Week 26 | NFP: Baseline (N=4, 5, 4) | 5436 nanogram/milliliter | Standard Deviation 3698 |
| Cohort 1 | Change From Baseline in Neurofilament Proteins (NFP), Glial Fibrillary Acidic Protein (GFAP) and Tauprotein in Cerebrospinal Fluid (CSF) at Week 26 | NFP: Change at Week 26 (N=4, 4, 4) | 1134 nanogram/milliliter | Standard Deviation 8098 |
| Cohort 1 | Change From Baseline in Neurofilament Proteins (NFP), Glial Fibrillary Acidic Protein (GFAP) and Tauprotein in Cerebrospinal Fluid (CSF) at Week 26 | GFAP: Baseline (N=4, 5, 4) | 1758 nanogram/milliliter | Standard Deviation 397.4 |
| Cohort 1 | Change From Baseline in Neurofilament Proteins (NFP), Glial Fibrillary Acidic Protein (GFAP) and Tauprotein in Cerebrospinal Fluid (CSF) at Week 26 | GFAP: Change at Week 26 (N=4, 4, 4) | 330.0 nanogram/milliliter | Standard Deviation 380.3 |
| Cohort 1 | Change From Baseline in Neurofilament Proteins (NFP), Glial Fibrillary Acidic Protein (GFAP) and Tauprotein in Cerebrospinal Fluid (CSF) at Week 26 | Tauprotein: Baseline (N=4, 5, 4) | 1148 nanogram/milliliter | Standard Deviation 143.4 |
| Cohort 1 | Change From Baseline in Neurofilament Proteins (NFP), Glial Fibrillary Acidic Protein (GFAP) and Tauprotein in Cerebrospinal Fluid (CSF) at Week 26 | Tauprotein: Change at Week 26 (N=4, 4, 4) | -288 nanogram/milliliter | Standard Deviation 692.3 |
| Cohort 2 | Change From Baseline in Neurofilament Proteins (NFP), Glial Fibrillary Acidic Protein (GFAP) and Tauprotein in Cerebrospinal Fluid (CSF) at Week 26 | Tauprotein: Change at Week 26 (N=4, 4, 4) | -273 nanogram/milliliter | Standard Deviation 228.7 |
| Cohort 2 | Change From Baseline in Neurofilament Proteins (NFP), Glial Fibrillary Acidic Protein (GFAP) and Tauprotein in Cerebrospinal Fluid (CSF) at Week 26 | NFP: Baseline (N=4, 5, 4) | 7848 nanogram/milliliter | Standard Deviation 4510 |
| Cohort 2 | Change From Baseline in Neurofilament Proteins (NFP), Glial Fibrillary Acidic Protein (GFAP) and Tauprotein in Cerebrospinal Fluid (CSF) at Week 26 | GFAP: Change at Week 26 (N=4, 4, 4) | 402.5 nanogram/milliliter | Standard Deviation 460.5 |
| Cohort 2 | Change From Baseline in Neurofilament Proteins (NFP), Glial Fibrillary Acidic Protein (GFAP) and Tauprotein in Cerebrospinal Fluid (CSF) at Week 26 | Tauprotein: Baseline (N=4, 5, 4) | 1014 nanogram/milliliter | Standard Deviation 530.9 |
| Cohort 2 | Change From Baseline in Neurofilament Proteins (NFP), Glial Fibrillary Acidic Protein (GFAP) and Tauprotein in Cerebrospinal Fluid (CSF) at Week 26 | NFP: Change at Week 26 (N=4, 4, 4) | -3505 nanogram/milliliter | Standard Deviation 2553 |
| Cohort 2 | Change From Baseline in Neurofilament Proteins (NFP), Glial Fibrillary Acidic Protein (GFAP) and Tauprotein in Cerebrospinal Fluid (CSF) at Week 26 | GFAP: Baseline (N=4, 5, 4) | 1014 nanogram/milliliter | Standard Deviation 524.3 |
| Cohort 3 | Change From Baseline in Neurofilament Proteins (NFP), Glial Fibrillary Acidic Protein (GFAP) and Tauprotein in Cerebrospinal Fluid (CSF) at Week 26 | NFP: Change at Week 26 (N=4, 4, 4) | -4020 nanogram/milliliter | Standard Deviation 9061 |
| Cohort 3 | Change From Baseline in Neurofilament Proteins (NFP), Glial Fibrillary Acidic Protein (GFAP) and Tauprotein in Cerebrospinal Fluid (CSF) at Week 26 | GFAP: Baseline (N=4, 5, 4) | 1415 nanogram/milliliter | Standard Deviation 625.7 |
| Cohort 3 | Change From Baseline in Neurofilament Proteins (NFP), Glial Fibrillary Acidic Protein (GFAP) and Tauprotein in Cerebrospinal Fluid (CSF) at Week 26 | Tauprotein: Change at Week 26 (N=4, 4, 4) | -118 nanogram/milliliter | Standard Deviation 907.6 |
| Cohort 3 | Change From Baseline in Neurofilament Proteins (NFP), Glial Fibrillary Acidic Protein (GFAP) and Tauprotein in Cerebrospinal Fluid (CSF) at Week 26 | GFAP: Change at Week 26 (N=4, 4, 4) | 502.5 nanogram/milliliter | Standard Deviation 285.4 |
| Cohort 3 | Change From Baseline in Neurofilament Proteins (NFP), Glial Fibrillary Acidic Protein (GFAP) and Tauprotein in Cerebrospinal Fluid (CSF) at Week 26 | NFP: Baseline (N=4, 5, 4) | 12558 nanogram/milliliter | Standard Deviation 3529 |
| Cohort 3 | Change From Baseline in Neurofilament Proteins (NFP), Glial Fibrillary Acidic Protein (GFAP) and Tauprotein in Cerebrospinal Fluid (CSF) at Week 26 | Tauprotein: Baseline (N=4, 5, 4) | 1610 nanogram/milliliter | Standard Deviation 543.4 |
Other Pre-specified
Number of Participants With Abnormal Findings in Urine Analysis
The parameters analyzed in urine were albumin/protein, glucose, leucocytes, acetoacetate/ketones, nitrite and pH. Urine analysis findings were considered abnormal as judged by the investigator.
Time frame: Baseline up to Week 26
Population: ITT
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Cohort 1 | Number of Participants With Abnormal Findings in Urine Analysis | 0 participants |
| Cohort 2 | Number of Participants With Abnormal Findings in Urine Analysis | 0 participants |
| Cohort 3 | Number of Participants With Abnormal Findings in Urine Analysis | 0 participants |
Other Pre-specified
Number of Participants With Clinically Significant Abnormal Electrocardiogram (ECG) Findings
Abnormal ECG findings were considered as clinically significant at the discretion of investigator.
Time frame: Baseline up to Week 26
Population: ITT
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Cohort 1 | Number of Participants With Clinically Significant Abnormal Electrocardiogram (ECG) Findings | 0 participants |
| Cohort 2 | Number of Participants With Clinically Significant Abnormal Electrocardiogram (ECG) Findings | 0 participants |
| Cohort 3 | Number of Participants With Clinically Significant Abnormal Electrocardiogram (ECG) Findings | 0 participants |
Other Pre-specified
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry
Number of participants with at least 1 shift from baseline to Week 26, are reported. Abbreviations: ALT=Alanine transaminase; CK=Creatine kinase; AP=Amyloid P component; LDH=Lactate dehydrogenase.
Time frame: Baseline up to Week 26
Population: ITT. Here, number of participants analyzed in the Cohort 2 are the participants evaluable for this outcome.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Magnesium-Serum: Normal to normal | 4 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | CK-Serum: Normal to high | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | ALT-Serum: Low to low | 2 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | LDH-Serum: High to normal | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | CK-Serum: High to normal | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | ALT-Serum: High to low | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | LDH-Serum: Normal to normal | 3 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | CK-Serum: High to high | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | T Bilirubin-Serum: Normal to normal | 4 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Iron-Serum: Normal to high | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Creatinine-Serum: Normal to normal | 4 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Sodium-Serum: Normal to normal | 4 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Iron-Serum: Normal to normal | 3 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Iron-Serum: Low to normal | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | ALT-Serum: High to normal | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Iron-Serum: Normal to low | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | ALT-Serum: Normal to low | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Potassium-Serum: Normal to normal | 4 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Amylase-Serum: Normal to normal | 4 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | ALT-Serum: Low to normal | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Phosphate-Serum: High to normal | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | AP-Serum: Normal to normal | 4 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Sodium-Serum: High to normal | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Phosphate-Serum: Normal to high | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Calcium-Serum: Normal to normal | 4 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | ALT-Serum: Normal to normal | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Phosphate-Serum: Normal to normal | 2 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | CK-Serum: Normal to normal | 4 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | T Bilirubin-Serum: High to normal | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Sodium-Serum: Normal to normal | 3 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | ALT-Serum: Low to low | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | ALT-Serum: Low to normal | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | ALT-Serum: Normal to low | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | ALT-Serum: Normal to normal | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | ALT-Serum: High to low | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | ALT-Serum: High to normal | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Amylase-Serum: Normal to normal | 4 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | AP-Serum: Normal to normal | 4 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Calcium-Serum: Normal to normal | 3 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | CK-Serum: Normal to normal | 3 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | CK-Serum: Normal to high | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | CK-Serum: High to normal | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | CK-Serum: High to high | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Creatinine-Serum: Normal to normal | 4 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Iron-Serum: Low to normal | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Iron-Serum: Normal to low | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Iron-Serum: Normal to normal | 3 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Iron-Serum: Normal to high | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | LDH-Serum: Normal to normal | 4 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | LDH-Serum: High to normal | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Magnesium-Serum: Normal to normal | 4 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Phosphate-Serum: Normal to normal | 3 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Phosphate-Serum: Normal to high | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Phosphate-Serum: High to normal | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Potassium-Serum: Normal to normal | 4 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Sodium-Serum: High to normal | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | T Bilirubin-Serum: Normal to normal | 3 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | T Bilirubin-Serum: High to normal | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | ALT-Serum: Normal to low | 2 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | LDH-Serum: High to normal | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | CK-Serum: Normal to normal | 2 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | ALT-Serum: Low to low | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Magnesium-Serum: Normal to normal | 4 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Calcium-Serum: Normal to normal | 4 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Sodium-Serum: High to normal | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Phosphate-Serum: Normal to normal | 3 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | AP-Serum: Normal to normal | 4 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | ALT-Serum: Low to normal | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Phosphate-Serum: Normal to high | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Amylase-Serum: Normal to normal | 4 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | T Bilirubin-Serum: High to normal | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Phosphate-Serum: High to normal | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | ALT-Serum: High to normal | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | T Bilirubin-Serum: Normal to normal | 4 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Iron-Serum: Low to normal | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Potassium-Serum: Normal to normal | 4 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Iron-Serum: Normal to low | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Creatinine-Serum: Normal to normal | 4 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | ALT-Serum: High to low | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Iron-Serum: Normal to normal | 3 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | CK-Serum: High to high | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | ALT-Serum: Normal to normal | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Iron-Serum: Normal to high | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | CK-Serum: High to normal | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Sodium-Serum: Normal to normal | 4 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | LDH-Serum: Normal to normal | 4 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | CK-Serum: Normal to high | 0 participants |
Other Pre-specified
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Coagulation
Number of participants with at least 1 shift from baseline to Week 26 are reported. The shift reported below for Cohort 1 was from low level at baseline to low level at Week 26.
Time frame: Baseline up to Week 26
Population: ITT. Data for Cohorts 2 and 3 were not reported since there were no participants with shift from baseline to Week 26 in coagulation evaluations.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Coagulation | 4 participants |
Other Pre-specified
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping
Number of participants with at least 1 shift from baseline to Week 26 are reported. Abbreviations: CSF=Cerebrospinal fluid; NFP=Neurofilament proteins.
Time frame: Baseline up to Week 26
Population: ITT. The number of participants analyzed in the Cohort 2 are the participants evaluable for this outcome. Here, N signifies the number of participants who were evaluable for the respective category.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Albumin index: High to high (N=3,4,4) | 3 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Albumin CSF: Normal to high (N=3,4,4) | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Albumin CSF: High to high (N=3,4,4) | 2 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Albumin index: Normal to normal (N=3,4,4) | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Albumin CSF: Normal to normal (N=3,4,4) | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Albumin Serum: Low to low (N=3,4,4) | 2 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Albumin Serum: Low to normal (N=3,4,4) | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Albumin Serum: Normal to low (N=3,4,4) | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Albumin Serum: Normal to normal (N=3,4,4) | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Chitotriosidase CSF: Low to low (N=4,4,4) | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Chitotriosidase CSF: High to high (N=4,4,4) | 3 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | NFP CSF: Normal to high (N=4,4,4) | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | NFP CSF: High to high (N=4,4,4) | 3 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Sulfatide CSF: High to high (N=3,4,4) | 3 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Tauprotein CSF: High to low (N=4,4,4) | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Tauprotein CSF: High to high (N=4,4,4) | 4 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Albumin Serum: Low to low (N=3,4,4) | 2 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Albumin Serum: Low to normal (N=3,4,4) | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Albumin Serum: Normal to low (N=3,4,4) | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Sulfatide CSF: High to high (N=3,4,4) | 4 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Albumin Serum: Normal to normal (N=3,4,4) | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Chitotriosidase CSF: Low to low (N=4,4,4) | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Tauprotein CSF: High to high (N=4,4,4) | 3 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Chitotriosidase CSF: High to high (N=4,4,4) | 4 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Albumin CSF: Normal to normal (N=3,4,4) | 2 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Tauprotein CSF: High to low (N=4,4,4) | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Albumin CSF: Normal to high (N=3,4,4) | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | NFP CSF: Normal to high (N=4,4,4) | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Albumin CSF: High to high (N=3,4,4) | 2 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Albumin index: Normal to normal (N=3,4,4) | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Albumin index: High to high (N=3,4,4) | 3 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | NFP CSF: High to high (N=4,4,4) | 4 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Chitotriosidase CSF: High to high (N=4,4,4) | 4 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Albumin Serum: Low to low (N=3,4,4) | 2 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | NFP CSF: High to high (N=4,4,4) | 4 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Albumin CSF: High to high (N=3,4,4) | 4 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Albumin Serum: Low to normal (N=3,4,4) | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Tauprotein CSF: High to high (N=4,4,4) | 4 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Albumin CSF: Normal to normal (N=3,4,4) | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Albumin Serum: Normal to low (N=3,4,4) | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | NFP CSF: Normal to high (N=4,4,4) | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Albumin index: High to high (N=3,4,4) | 4 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Albumin Serum: Normal to normal (N=3,4,4) | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Sulfatide CSF: High to high (N=3,4,4) | 4 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Albumin CSF: Normal to high (N=3,4,4) | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Chitotriosidase CSF: Low to low (N=4,4,4) | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Albumin index: Normal to normal (N=3,4,4) | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Tauprotein CSF: High to low (N=4,4,4) | 0 participants |
Other Pre-specified
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology
Number of participants with at least 1 shift from baseline to Week 26 are reported. Abbreviations: Abs=Absolute count; ERCS=Erythrocytes; MCHC=Mean corpuscular hemoglobin concentration; MCH=Mean cell hemoglobin.
Time frame: Baseline up to Week 26
Population: ITT. The number of participants analyzed in the Cohort 2 are the participants evaluable for this outcome. Here, N signifies the number of participants who were evaluable for the respective category.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | ERCS - Blood: Normal to normal (N=4,4,4) | 3 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Monocytes Abs - Blood: High to normal (N=4,4,4) | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Lymphocyte Abs - Blood: Normal to low (N=4,4,4) | 2 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | T Leucocytes - Blood: Normal to normal (N=4,4,4) | 2 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Basophils Abs - Blood: Normal to normal (N=4,4,4) | 4 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Lymphocyte Abs - Blood: Normal to normal (N=4,4,4) | 2 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Thrombocytes - Blood: High to normal (N=4,3,4) | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Monocytes Abs - Blood: Normal to normal (N=4,4,4) | 4 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | MCHC - Blood: Normal to normal (N=4,4,4) | 3 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Haemoglobin - Blood: Low to low (N=4,4,4) | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | MCH - Blood: Normal to normal (N=4,4,4) | 4 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | MCHC - Blood: High to normal (N=4,4,4) | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | T Leucocytes - Blood: Normal to low (N=4,4,4) | 2 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | MCH - Blood: Low to low (N=4,4,4) | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Thrombocytes - Blood: Normal to normal (N=4,3,4) | 2 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Haemoglobin - Blood: Low to normal (N=4,4,4) | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | ERCS - Blood: Low to low (N=4,4,4) | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Thrombocytes - Blood: Normal to low (N=4,3,4) | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Haemoglobin - Blood: Normal to low (N=4,4,4) | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Eosinophil Abs - Blood: Normal to normal (N=4,4,4) | 4 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Neutropil Abs - Blood: Normal to high (N=4,4,4) | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Haemoglobin - Blood: Normal to normal (N=4,4,4) | 1 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | T Leucocytes - Blood: Low to normal (N=4,4,4) | 0 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Neutropil Abs - Blood: Normal to normal (N=4,4,4) | 4 participants |
| Cohort 1 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Lymphocyte Abs - Blood: Low to low (N=4,4,4) | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Monocytes Abs - Blood: Normal to normal (N=4,4,4) | 4 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Basophils Abs - Blood: Normal to normal (N=4,4,4) | 4 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Eosinophil Abs - Blood: Normal to normal (N=4,4,4) | 4 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | ERCS - Blood: Low to low (N=4,4,4) | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | ERCS - Blood: Normal to normal (N=4,4,4) | 4 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Haemoglobin - Blood: Low to low (N=4,4,4) | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Haemoglobin - Blood: Low to normal (N=4,4,4) | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Haemoglobin - Blood: Normal to low (N=4,4,4) | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Haemoglobin - Blood: Normal to normal (N=4,4,4) | 3 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Lymphocyte Abs - Blood: Low to low (N=4,4,4) | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Lymphocyte Abs - Blood: Normal to low (N=4,4,4) | 2 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Lymphocyte Abs - Blood: Normal to normal (N=4,4,4) | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | MCHC - Blood: Normal to normal (N=4,4,4) | 3 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | MCHC - Blood: High to normal (N=4,4,4) | 1 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | MCH - Blood: Low to low (N=4,4,4) | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | MCH - Blood: Normal to normal (N=4,4,4) | 4 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Monocytes Abs - Blood: High to normal (N=4,4,4) | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Neutropil Abs - Blood: Normal to normal (N=4,4,4) | 4 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Neutropil Abs - Blood: Normal to high (N=4,4,4) | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Thrombocytes - Blood: Normal to low (N=4,3,4) | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Thrombocytes - Blood: Normal to normal (N=4,3,4) | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Thrombocytes - Blood: High to normal (N=4,3,4) | 3 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | T Leucocytes - Blood: Low to normal (N=4,4,4) | 0 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | T Leucocytes - Blood: Normal to low (N=4,4,4) | 2 participants |
| Cohort 2 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | T Leucocytes - Blood: Normal to normal (N=4,4,4) | 2 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Monocytes Abs - Blood: High to normal (N=4,4,4) | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Haemoglobin - Blood: Normal to normal (N=4,4,4) | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Basophils Abs - Blood: Normal to normal (N=4,4,4) | 4 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Neutropil Abs - Blood: Normal to normal (N=4,4,4) | 3 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Haemoglobin - Blood: Normal to low (N=4,4,4) | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | T Leucocytes - Blood: Low to normal (N=4,4,4) | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Neutropil Abs - Blood: Normal to high (N=4,4,4) | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Haemoglobin - Blood: Low to normal (N=4,4,4) | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Eosinophil Abs - Blood: Normal to normal (N=4,4,4) | 4 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Thrombocytes - Blood: Normal to low (N=4,3,4) | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Haemoglobin - Blood: Low to low (N=4,4,4) | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | T Leucocytes - Blood: Normal to normal (N=4,4,4) | 3 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Thrombocytes - Blood: Normal to normal (N=4,3,4) | 2 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | ERCS - Blood: Normal to normal (N=4,4,4) | 4 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | MCHC - Blood: High to normal (N=4,4,4) | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | T Leucocytes - Blood: Normal to low (N=4,4,4) | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | MCH - Blood: Low to low (N=4,4,4) | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | MCHC - Blood: Normal to normal (N=4,4,4) | 4 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Thrombocytes - Blood: High to normal (N=4,3,4) | 1 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | MCH - Blood: Normal to normal (N=4,4,4) | 3 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Lymphocyte Abs - Blood: Normal to normal (N=4,4,4) | 2 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Lymphocyte Abs - Blood: Normal to low (N=4,4,4) | 2 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Monocytes Abs - Blood: Normal to normal (N=4,4,4) | 3 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | Lymphocyte Abs - Blood: Low to low (N=4,4,4) | 0 participants |
| Cohort 3 | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | ERCS - Blood: Low to low (N=4,4,4) | 0 participants |
Other Pre-specified
Physical Examination Results
Physical examination included general appearance, skin, head, ears, eyes, nose and throat, lymph nodes, heart, lungs, abdomen, extremities/joints, hip, neurological, mental status and, if appropriate, breasts, external genitalia, pelvic and rectal, and in addition weight, height and head circumference were recorded.
Time frame: Baseline up to Week 26
Population: Physical examination results were not summarized since data were collected in participant's listing only as planned.