Spinal Muscular Atrophy
Conditions
Keywords
Zolgensma, OAV101, AVXS 101, gene therapy, Muscle atrophy, SBMA, spinal and bulbar muscular atrophy, spinal muscular atrophy, bulbar muscular atrophy, muscle function, myopathy, muscle wasting, atrophied muscle, loss of muscle strength
Brief summary
To evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene weighing ≥ 8.5 kg and ≤ 21 kg, over a 12 month period.
Detailed description
This was an open-label, single arm, multi-center study designed to evaluate the safety, tolerability and efficacy of OAV101 in participants with SMA who weigh ≥ 8.5 kg and ≤ 21 kg. The study aimed to enroll approximately 24 to 30 participants, with approximately 6 to 10 participants across each of 3 weight brackets (8.5 to 13 kg, \>13 to 17 kg, \>17 to 21 kg). Eligible participants received a single administration of OAV101 at the approved dose of 1.1e14 vg/kg on Day 1 (Treatment period), and were followed for a period of 12 months. Participants were admitted to the hospital on Day -1 for pre-treatment baseline procedures. After receiving OAV101 on Day 1, participants underwent in-patient safety monitoring over the next 48 hours, after which the participant could be discharged, based on Investigator judgment. After study completion, eligible participants could enroll into a Long Term follow-up study to collect additional safety and efficacy data. (COAV101A12308 (NCT05335876) https://classic.clinicaltrials.gov/ct2/show/NCT05335876?term=COAV101A12308&draw=2&rank=1))
Interventions
GENETICOAV101
Gene Therapy - 1.1e14 vector genome (vg)/kg as a one-time IV infusion was administered over approximately 60 minutes.
Sponsors
Novartis Pharmaceuticals
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Participants will receive a single administration of OAV101
Eligibility
Sex/Gender
ALL
Age
No minimum to 17 Years
Healthy volunteers
No
Inclusion criteria
Inclusion * Symptomatic SMA diagnosis based on gene mutation analysis with bi-allelic survival motor neuron 1 (SMN1) mutations (deletion or point mutations) and any copy of the survival motor neuron 2 (SMN2) gene. * Weight ≥ 8.5 kg and ≤ 21 kg at the time of Screening Visit 2 * Naive to treatment or have discontinued an approved drug/therapy Exclusion: * Previous OAV101 use or previous use of any adeno-associated virus serotype 9 (AAV9) gene therapy * BMI \< 3rd percentile * Participant with history of aspiration pneumonia or signs of aspiration * Elevated anti-AAV9 antibody * History of gene therapy, hematopoietic transplantation, or solid organ transplantation * Inability to take corticosteroids * Concomitant use of immunosuppressive therapy * Requiring invasive ventilation, tracheostomy or awake non-invasive ventilation 9. Administration of vaccines 2 weeks prior to infusion of OAV101 * Awake hypoxemia or awake oxygen saturation level decrease * Hepatic dysfunction * Presence of a confirmed or suspected infection * If previously treated with disease modifying therapy, specified washout times apply * Documented any parental consanguinity.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52 | Change from baseline in vital signs measurements - oxygen saturation level (%). Oxygen saturation is the fraction of oxygen-saturated hemoglobin relative to total hemoglobin (unsaturated+saturated) in the blood and then multiplied by 100. |
| Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52 | Change from baseline in vital signs measurements - Pulse Rate (beats/min |
| Summary of Participants Meeting Criteria for Potentially Clinically Significant Vital Sign Values by Weight Bracket - Temperature | 12 months | Change from baseline in vital signs measurements - temperature (degrees Celsius) Temperature-Low:\<=35ºC(Any Age),High:\>=38.4ºC(\<18 yrs). |
| Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52 | — |
| Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Up to Month 12 | An AE is any untoward medical occurrence (e.g. any unfavorable and unintended sign \[including abnormal laboratory findings\], symptom or disease) in a clinical investigation participant after providing written informed consent for participation in the study. |
| Number of Participants With Important Identified and Important Potential Risks (Adverse Events of Special Interest (AESI)) by Risk Name and Weight Bracket | Up to Month 12 | Important identified and important potential risks included the following AESIs: Hepatotoxicity, Thrombocytopenia, Cardiac adverse events, Dorsal root ganglia toxicity and Thrombotic microangiopathy. These were assessed by the investigator. |
| Summary of Participants Meeting Criteria for Potentially Clinically Significant Vital Sign Values by Weight Bracket - Systolic and Diastolic Blood Pressure | 12 months | Change from baseline in vital signs measurements - systolic and diastolic blood pressure (mmHg). Systolic Blood Pressure-Low:\<=5th percentile of the age(Any Age), High:\>=90th percentile of the age, gender, and height group (\<18 yrs). Diastolic Blood Pressure-High:\>=90th percentile of the age, gender, and height group(\<18 yrs). |
| Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52 | — |
| Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52 | — |
| Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52 | Change from baseline in vital signs measurements - Respiratory Rate (breaths/min) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE), as Appropriate According to Participant Age | Baseline, Week 4, Week 13, Week 26, Week 39 and Week 52 | The HFMSE was devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE is formed of 33 assessments. Each motor skill item is scored on a 3 point Likert scale from 0 (no response) to 2 (full response), with a total score range of 0 to 66. A higher score indicates a higher level of ability. |
| Change From Baseline in Revised Upper Limb Module (RULM), as Appropriate According to Participant Age. | Baseline, Week 4, Week 13, Week 26, Week 39 and Week 52 | The RULM assesses motor performance in the upper limbs from childhood through adulthood in ambulatory and non-ambulatory individuals with SMA. 'The scale consists of an entry item to establish functional levels and 19 items covering distal to proximal movements. The entry item is a modified version of the Brooke scale, including activities ranging from no functional use of hands (score 0) to full bilateral shoulder abduction (score 6). The entry item does not contribute to the total score but serves as a functional classification of overall upper limb functional ability. Of the remaining 19 items, 18 are scored on a 3 point scoring system and 1 item is scored on a 2 point scoring system. The test is performed unilaterally using the limb preferred by the participant. The total score ranges from 0, if all the items cannot be performed, to 37, if all the activities are achieved fully without any compensation. ' Higher scores indicate higher levels of motor ability. |
| Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline, Week 26 and Week 52 | The World Health Organization-Multicentre Growth Reference Study (WHO-MGRS) and Bayley scale of Infant and Toddler Development was modified and combined into a single scale expressly for this study, to measure developmental motor milestones. These were assessed via the milestone checklist, formed of 10 yes/no questions with optional video documentation. The developmental milestones are: head control, sitting with support, sitting without support, sitting without support for 30 seconds, hands-and-knees crawling, pulls to stand, standing with assistance, walking with assistance, standing alone and walking alone. A yes response indicates that the patient reached a particular development milestone. |
Countries
Australia, Belgium, Canada, France, Italy, Portugal, Taiwan, United Kingdom, United States
Participant flow
Recruitment details
Participants took part in 13 investigative sites across 9 countries.
Pre-assignment details
The screening period began after signature of the study informed consent. The study included a screening period of up to 45 days. On Day -1, participants were admitted to the hospital for pre-treatment baseline procedures. On Day 1, participants received the study treatment.
Participants by arm
| Arm | Count |
|---|---|
| OAV101 1.1e14 vg/kg 8.5-13 kg 8.5-13 kg | 7 |
| OAV101 1.1e14 vg/kg >13-17 kg \>13-17 kg | 8 |
| OAV101 1.1e14 vg/kg >17-21 kg \>17-21 kg | 9 |
| Total | 24 |
Baseline characteristics
| Characteristic | Total | OAV101 1.1e14 vg/kg 8.5-13 kg | OAV101 1.1e14 vg/kg >17-21 kg | OAV101 1.1e14 vg/kg >13-17 kg |
|---|---|---|---|---|
| Age, Continuous | 4.690 years STANDARD_DEVIATION 1.824 | 3.027 years STANDARD_DEVIATION 1.1458 | 6.137 years STANDARD_DEVIATION 1.6018 | 4.519 years STANDARD_DEVIATION 1.1769 |
| Age, Customized 0 < 28 days | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Age, Customized 12 years - <18 years | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Age, Customized >= 18 years | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Age, Customized 28 days - < 2 years | 1 Participants | 1 Participants | 0 Participants | 0 Participants |
| Age, Customized 2 years - < 12 years | 23 Participants | 6 Participants | 9 Participants | 8 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 9 Participants | 1 Participants | 3 Participants | 5 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 2 Participants | 0 Participants | 0 Participants | 2 Participants |
| Race (NIH/OMB) White | 13 Participants | 6 Participants | 6 Participants | 1 Participants |
| Sex: Female, Male Female | 12 Participants | 3 Participants | 4 Participants | 5 Participants |
| Sex: Female, Male Male | 12 Participants | 4 Participants | 5 Participants | 3 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk |
|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 7 | 0 / 8 | 0 / 9 | 0 / 24 |
| other Total, other adverse events | 7 / 7 | 8 / 8 | 9 / 9 | 24 / 24 |
| serious Total, serious adverse events | 3 / 7 | 7 / 8 | 5 / 9 | 15 / 24 |
Outcome results
Primary
Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg)
Time frame: Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52
Population: Safety set
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 6 (n=6,8,8,22) | 10.3 mmHg | Standard Deviation 10.91 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Day 2 (n=6,8,9,23) | 1.7 mmHg | Standard Deviation 12.42 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 2 | 7.1 mmHg | Standard Deviation 16.07 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 13 (n=6,8,9,23) | 9.7 mmHg | Standard Deviation 15.37 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 39 (n=7,7,9,23) | 2.4 mmHg | Standard Deviation 15.73 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 10 (n=6,8,9,23) | 6.7 mmHg | Standard Deviation 15.55 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 3 (n=6,8,9,23) | 12.0 mmHg | Standard Deviation 15.09 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 52 | 0.3 mmHg | Standard Deviation 11.74 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 1 | 7.4 mmHg | Standard Deviation 20.07 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 26 | -0.7 mmHg | Standard Deviation 9.05 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 4 (n=6,8,8,22) | 3.8 mmHg | Standard Deviation 19.27 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 8 (n=6,7,9,22) | 10.5 mmHg | Standard Deviation 14.57 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Day 3 (n=6,8,9,23) | -3.7 mmHg | Standard Deviation 12.29 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Day 2 (n=6,8,9,23) | 4.8 mmHg | Standard Deviation 13.22 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 6 (n=6,8,8,22) | -0.5 mmHg | Standard Deviation 17.87 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Day 3 (n=6,8,9,23) | -5.1 mmHg | Standard Deviation 13.91 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 8 (n=6,7,9,22) | 3.0 mmHg | Standard Deviation 17.68 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 10 (n=6,8,9,23) | -2.0 mmHg | Standard Deviation 8.88 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 1 | 1.8 mmHg | Standard Deviation 13.56 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 13 (n=6,8,9,23) | 0.4 mmHg | Standard Deviation 13.54 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 39 (n=7,7,9,23) | -4.4 mmHg | Standard Deviation 13.01 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 2 | 7.3 mmHg | Standard Deviation 11.3 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 26 | -9.1 mmHg | Standard Deviation 10.26 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 3 (n=6,8,9,23) | -1.5 mmHg | Standard Deviation 14.01 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 52 | -2.0 mmHg | Standard Deviation 11.89 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 4 (n=6,8,8,22) | 1.8 mmHg | Standard Deviation 9.97 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 26 | -1.2 mmHg | Standard Deviation 7.55 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 52 | 1.7 mmHg | Standard Deviation 5.52 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Day 2 (n=6,8,9,23) | -2.1 mmHg | Standard Deviation 13.04 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Day 3 (n=6,8,9,23) | 4.2 mmHg | Standard Deviation 5.45 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 1 | 3.8 mmHg | Standard Deviation 13.41 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 2 | 4.4 mmHg | Standard Deviation 10.33 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 3 (n=6,8,9,23) | 5.0 mmHg | Standard Deviation 9.6 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 4 (n=6,8,8,22) | 1.3 mmHg | Standard Deviation 8.53 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 6 (n=6,8,8,22) | 6.5 mmHg | Standard Deviation 10.14 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 8 (n=6,7,9,22) | 5.9 mmHg | Standard Deviation 11.72 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 10 (n=6,8,9,23) | 4.8 mmHg | Standard Deviation 11.74 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 13 (n=6,8,9,23) | 0.1 mmHg | Standard Deviation 11.03 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 39 (n=7,7,9,23) | -0.4 mmHg | Standard Deviation 9.62 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 6 (n=6,8,8,22) | 5.0 mmHg | Standard Deviation 13.77 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 4 (n=6,8,8,22) | 2.1 mmHg | Standard Deviation 12.12 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Day 2 (n=6,8,9,23) | 1.3 mmHg | Standard Deviation 12.71 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 13 (n=6,8,9,23) | 2.7 mmHg | Standard Deviation 13.2 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 3 (n=6,8,9,23) | 4.6 mmHg | Standard Deviation 13.28 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 2 | 6.2 mmHg | Standard Deviation 12.05 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 26 | -3.7 mmHg | Standard Deviation 9.41 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 1 | 4.2 mmHg | Standard Deviation 15.13 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 52 | 0.0 mmHg | Standard Deviation 9.6 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 39 (n=7,7,9,23) | -0.8 mmHg | Standard Deviation 12.44 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 8 (n=6,7,9,22) | 6.2 mmHg | Standard Deviation 14.18 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Day 3 (n=6,8,9,23) | -1.1 mmHg | Standard Deviation 11.22 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg) | Change from baseline at Week 10 (n=6,8,9,23) | 2.9 mmHg | Standard Deviation 12.01 |
Primary
Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level
Change from baseline in vital signs measurements - oxygen saturation level (%). Oxygen saturation is the fraction of oxygen-saturated hemoglobin relative to total hemoglobin (unsaturated+saturated) in the blood and then multiplied by 100.
Time frame: Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52
Population: Safety set
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 39 (n=7,7,8,22) | -1.0 % Oxygen Saturated | Standard Deviation 1.41 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 26 | -0.6 % Oxygen Saturated | Standard Deviation 1.51 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 8 (n=7,7,9,23) | -0.7 % Oxygen Saturated | Standard Deviation 1.89 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 3 (n=6,8,9,23) | -1.0 % Oxygen Saturated | Standard Deviation 1.9 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 13 | 0.1 % Oxygen Saturated | Standard Deviation 1.57 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 10 | 0.3 % Oxygen Saturated | Standard Deviation 1.89 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 1 | 0.1 % Oxygen Saturated | Standard Deviation 1.07 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 4 (n=7,8,8,23) | 0.0 % Oxygen Saturated | Standard Deviation 1.91 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 2 | -0.7 % Oxygen Saturated | Standard Deviation 1.89 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Day 3 | 0.3 % Oxygen Saturated | Standard Deviation 1.8 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 52 | -1.0 % Oxygen Saturated | Standard Deviation 2 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 6 (n=7,8,8,23) | -0.1 % Oxygen Saturated | Standard Deviation 1.21 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Day 2 | 0.0 % Oxygen Saturated | Standard Deviation 0.82 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 1 | -0.4 % Oxygen Saturated | Standard Deviation 2 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Day 2 | -0.3 % Oxygen Saturated | Standard Deviation 1.04 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 3 (n=6,8,9,23) | -0.3 % Oxygen Saturated | Standard Deviation 1.28 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 4 (n=7,8,8,23) | -0.5 % Oxygen Saturated | Standard Deviation 1.31 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 6 (n=7,8,8,23) | -1.0 % Oxygen Saturated | Standard Deviation 1.51 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 8 (n=7,7,9,23) | 0.0 % Oxygen Saturated | Standard Deviation 1.15 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 10 | -1.0 % Oxygen Saturated | Standard Deviation 1.93 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 13 | -0.5 % Oxygen Saturated | Standard Deviation 1.07 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 26 | -0.1 % Oxygen Saturated | Standard Deviation 1.36 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 39 (n=7,7,8,22) | 0.1 % Oxygen Saturated | Standard Deviation 0.9 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 52 | -0.6 % Oxygen Saturated | Standard Deviation 1.51 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Day 3 | -0.1 % Oxygen Saturated | Standard Deviation 0.83 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 2 | -1.0 % Oxygen Saturated | Standard Deviation 1.51 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 13 | -0.4 % Oxygen Saturated | Standard Deviation 1.67 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 26 | -0.2 % Oxygen Saturated | Standard Deviation 1.99 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 6 (n=7,8,8,23) | -0.6 % Oxygen Saturated | Standard Deviation 1.19 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Day 2 | -0.4 % Oxygen Saturated | Standard Deviation 1.13 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 39 (n=7,7,8,22) | -0.4 % Oxygen Saturated | Standard Deviation 1.19 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 52 | -0.1 % Oxygen Saturated | Standard Deviation 1.54 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 4 (n=7,8,8,23) | -0.8 % Oxygen Saturated | Standard Deviation 0.89 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 2 | -0.8 % Oxygen Saturated | Standard Deviation 2.11 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Day 3 | -1.3 % Oxygen Saturated | Standard Deviation 1.12 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 3 (n=6,8,9,23) | -1.1 % Oxygen Saturated | Standard Deviation 1.05 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 10 | -0.7 % Oxygen Saturated | Standard Deviation 1.32 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 8 (n=7,7,9,23) | -0.4 % Oxygen Saturated | Standard Deviation 0.88 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 1 | -0.8 % Oxygen Saturated | Standard Deviation 1.2 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 8 (n=7,7,9,23) | -0.4 % Oxygen Saturated | Standard Deviation 1.31 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 6 (n=7,8,8,23) | -0.6 % Oxygen Saturated | Standard Deviation 1.31 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 1 | -0.4 % Oxygen Saturated | Standard Deviation 1.47 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Day 2 | -0.3 % Oxygen Saturated | Standard Deviation 0.99 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 26 | -0.3 % Oxygen Saturated | Standard Deviation 1.6 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 10 | -0.5 % Oxygen Saturated | Standard Deviation 1.72 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 13 | -0.3 % Oxygen Saturated | Standard Deviation 1.43 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Day 3 | -0.5 % Oxygen Saturated | Standard Deviation 1.41 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 39 (n=7,7,8,22) | -0.4 % Oxygen Saturated | Standard Deviation 1.22 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 4 (n=7,8,8,23) | -0.4 % Oxygen Saturated | Standard Deviation 1.38 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 2 | -0.8 % Oxygen Saturated | Standard Deviation 1.79 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 3 (n=6,8,9,23) | -0.8 % Oxygen Saturated | Standard Deviation 1.38 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level | Change from baseline at Week 52 | -0.5 % Oxygen Saturated | Standard Deviation 1.64 |
Primary
Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min)
Change from baseline in vital signs measurements - Pulse Rate (beats/min
Time frame: Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52
Population: Safety set
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Day 2 | 3.3 beats/min | Standard Deviation 18.54 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Day 3 | -1.4 beats/min | Standard Deviation 19.29 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 1 | 1.4 beats/min | Standard Deviation 14.27 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 2 | -13.4 beats/min | Standard Deviation 21.25 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 3 (n=6,8,9,23) | 21.0 beats/min | Standard Deviation 21.83 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 4 (n=7,8,8,23) | -4.7 beats/min | Standard Deviation 17.27 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 6 (n=7,8,8,23) | 12.9 beats/min | Standard Deviation 20.7 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 8 (n=7,7,9,23) | -3.6 beats/min | Standard Deviation 15.75 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 10 | -3.9 beats/min | Standard Deviation 25.14 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 13 | -6.4 beats/min | Standard Deviation 12.23 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 26 | -6.9 beats/min | Standard Deviation 12.85 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 39 (n=7,7,9,23) | -4.7 beats/min | Standard Deviation 21.85 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 52 | -8.9 beats/min | Standard Deviation 10.32 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 2 | -8.8 beats/min | Standard Deviation 15.21 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 52 | -16.4 beats/min | Standard Deviation 14.38 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 26 | -6.3 beats/min | Standard Deviation 17 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 8 (n=7,7,9,23) | -6.3 beats/min | Standard Deviation 22.69 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 1 | -9.6 beats/min | Standard Deviation 18.04 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Day 2 | 6.3 beats/min | Standard Deviation 4.43 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 13 | 2.6 beats/min | Standard Deviation 15.65 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 10 | -4.3 beats/min | Standard Deviation 16.57 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 4 (n=7,8,8,23) | -3.5 beats/min | Standard Deviation 22.82 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 3 (n=6,8,9,23) | -3.8 beats/min | Standard Deviation 12.46 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Day 3 | -3.1 beats/min | Standard Deviation 14.21 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 39 (n=7,7,9,23) | -7.7 beats/min | Standard Deviation 13.23 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 6 (n=7,8,8,23) | 0.0 beats/min | Standard Deviation 9.59 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 26 | 3.8 beats/min | Standard Deviation 14.34 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 2 | 4.7 beats/min | Standard Deviation 12.12 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 3 (n=6,8,9,23) | 13.1 beats/min | Standard Deviation 15.12 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 52 | 4.4 beats/min | Standard Deviation 16.46 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 4 (n=7,8,8,23) | 12.8 beats/min | Standard Deviation 16.23 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 6 (n=7,8,8,23) | 8.8 beats/min | Standard Deviation 17.64 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 39 (n=7,7,9,23) | 3.1 beats/min | Standard Deviation 17.12 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 8 (n=7,7,9,23) | 6.4 beats/min | Standard Deviation 17.63 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 10 | 9.7 beats/min | Standard Deviation 14.13 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 13 | 8.4 beats/min | Standard Deviation 18.06 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Day 2 | 16.3 beats/min | Standard Deviation 26.54 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Day 3 | 10.2 beats/min | Standard Deviation 14.19 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 1 | -3.9 beats/min | Standard Deviation 18.84 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 13 | 2.2 beats/min | Standard Deviation 16.28 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 8 (n=7,7,9,23) | -0.5 beats/min | Standard Deviation 18.83 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 2 | -5.1 beats/min | Standard Deviation 17.39 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Day 2 | 9.2 beats/min | Standard Deviation 19.35 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 6 (n=7,8,8,23) | 7.0 beats/min | Standard Deviation 16.58 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 4 (n=7,8,8,23) | 1.8 beats/min | Standard Deviation 19.95 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 1 | -4.3 beats/min | Standard Deviation 17.19 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Day 3 | 2.4 beats/min | Standard Deviation 16.36 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 3 (n=6,8,9,23) | 9.3 beats/min | Standard Deviation 18.6 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 10 | 1.1 beats/min | Standard Deviation 19.08 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 52 | -6.4 beats/min | Standard Deviation 16.35 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 39 (n=7,7,9,23) | -2.6 beats/min | Standard Deviation 17.54 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min) | Change from baseline at Week 26 | -2.7 beats/min | Standard Deviation 15.12 |
Primary
Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min)
Change from baseline in vital signs measurements - Respiratory Rate (breaths/min)
Time frame: Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52
Population: Safety set
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Day 2 | -1.1 breaths/min) | Standard Deviation 3.08 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Day 3 | -0.1 breaths/min) | Standard Deviation 5.87 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 1 | 2.7 breaths/min) | Standard Deviation 6.78 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 2 | 2.4 breaths/min) | Standard Deviation 5.44 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 3 (n=6,8,9,23) | 5.5 breaths/min) | Standard Deviation 8.96 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 4 (n=7,8,8,23) | 2.6 breaths/min) | Standard Deviation 4.54 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 6 (n=7,8,8,23) | 5.1 breaths/min) | Standard Deviation 9.56 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 8 (n=7,7,9,23) | 3.3 breaths/min) | Standard Deviation 7.45 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 10 | 3.3 breaths/min) | Standard Deviation 6.58 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 13 | 1.7 breaths/min) | Standard Deviation 6.75 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 26 | 4.1 breaths/min) | Standard Deviation 8.69 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 39 (n=7,7,9,23) | 1.4 breaths/min) | Standard Deviation 5.03 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 52 | 1.7 breaths/min) | Standard Deviation 7.34 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 2 | -0.8 breaths/min) | Standard Deviation 5.01 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 52 | -0.5 breaths/min) | Standard Deviation 4.66 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 26 | 0.8 breaths/min) | Standard Deviation 3.69 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 8 (n=7,7,9,23) | -0.6 breaths/min) | Standard Deviation 4.47 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 1 | -0.1 breaths/min) | Standard Deviation 4.22 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Day 2 | -1.6 breaths/min) | Standard Deviation 3.74 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 13 | -0.5 breaths/min) | Standard Deviation 3.51 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 10 | -1.0 breaths/min) | Standard Deviation 4.31 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 4 (n=7,8,8,23) | 1.4 breaths/min) | Standard Deviation 7.35 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 3 (n=6,8,9,23) | -1.8 breaths/min) | Standard Deviation 5.09 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Day 3 | -0.1 breaths/min) | Standard Deviation 4.52 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 39 (n=7,7,9,23) | -1.7 breaths/min) | Standard Deviation 4.35 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 6 (n=7,8,8,23) | -0.6 breaths/min) | Standard Deviation 4.75 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 26 | 0.7 breaths/min) | Standard Deviation 4.24 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 2 | 1.4 breaths/min) | Standard Deviation 4.16 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 3 (n=6,8,9,23) | 1.8 breaths/min) | Standard Deviation 5.02 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 52 | -2.2 breaths/min) | Standard Deviation 3.19 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 4 (n=7,8,8,23) | 1.4 breaths/min) | Standard Deviation 4 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 6 (n=7,8,8,23) | 0.3 breaths/min) | Standard Deviation 6.43 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 39 (n=7,7,9,23) | -0.6 breaths/min) | Standard Deviation 4.56 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 8 (n=7,7,9,23) | 0.0 breaths/min) | Standard Deviation 4.42 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 10 | -0.3 breaths/min) | Standard Deviation 3.61 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 13 | -0.6 breaths/min) | Standard Deviation 2.92 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Day 2 | 0.8 breaths/min) | Standard Deviation 3.96 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Day 3 | 0.4 breaths/min) | Standard Deviation 4.3 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 1 | 1.6 breaths/min) | Standard Deviation 5.61 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 13 | 0.1 breaths/min) | Standard Deviation 4.44 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 8 (n=7,7,9,23) | 0.8 breaths/min) | Standard Deviation 5.52 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 2 | 1.0 breaths/min) | Standard Deviation 4.81 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Day 2 | -0.6 breaths/min) | Standard Deviation 3.66 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 6 (n=7,8,8,23) | 1.4 breaths/min) | Standard Deviation 7.19 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 4 (n=7,8,8,23) | 1.7 breaths/min) | Standard Deviation 5.31 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 1 | 1.3 breaths/min) | Standard Deviation 5.45 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Day 3 | 0.1 breaths/min) | Standard Deviation 4.66 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 3 (n=6,8,9,23) | 1.5 breaths/min) | Standard Deviation 6.63 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 10 | 0.5 breaths/min) | Standard Deviation 4.99 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 52 | -0.5 breaths/min) | Standard Deviation 5.18 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 39 (n=7,7,9,23) | -0.3 breaths/min) | Standard Deviation 4.61 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min) | Change from baseline at Week 26 | 1.7 breaths/min) | Standard Deviation 5.71 |
Primary
Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg)
Time frame: Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52
Population: Safety set
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 3 (n=6,8,9,23) | 15.8 mmHg | Standard Deviation 8.04 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 4 (n=6,8,8,22) | 12.3 mmHg | Standard Deviation 16.5 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 6 (n=7,8,8,23) | 8.1 mmHg | Standard Deviation 12.92 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 8 (n=7,7,9,23) | 3.9 mmHg | Standard Deviation 15.49 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 10 | 10.0 mmHg | Standard Deviation 16.99 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 13 | 6.1 mmHg | Standard Deviation 10.78 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 26 | -5.7 mmHg | Standard Deviation 9.05 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 39 (n=7,7,9,23) | 16.4 mmHg | Standard Deviation 29.7 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 52 | 5.0 mmHg | Standard Deviation 21.6 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Day 2 | 5.3 mmHg | Standard Deviation 12.65 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Day 3 | -2.3 mmHg | Standard Deviation 15.01 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 1 | 13.4 mmHg | Standard Deviation 10.53 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 2 | 11.7 mmHg | Standard Deviation 20.33 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 8 (n=7,7,9,23) | -0.7 mmHg | Standard Deviation 14.57 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 2 | -3.8 mmHg | Standard Deviation 17.6 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Day 3 | -5.6 mmHg | Standard Deviation 11.16 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 39 (n=7,7,9,23) | -10.3 mmHg | Standard Deviation 13.59 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 6 (n=7,8,8,23) | -2.8 mmHg | Standard Deviation 24.15 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 3 (n=6,8,9,23) | -3.5 mmHg | Standard Deviation 12.87 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Day 2 | -1.3 mmHg | Standard Deviation 13.01 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 52 | -7.8 mmHg | Standard Deviation 13.54 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 13 | -4.5 mmHg | Standard Deviation 17.5 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 10 | -5.6 mmHg | Standard Deviation 13.19 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 4 (n=6,8,8,22) | -3.5 mmHg | Standard Deviation 15.89 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 1 | -7.4 mmHg | Standard Deviation 15.59 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 26 | -12.9 mmHg | Standard Deviation 15.34 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Day 3 | 3.0 mmHg | Standard Deviation 8.38 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 8 (n=7,7,9,23) | 3.6 mmHg | Standard Deviation 12.83 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 10 | 4.6 mmHg | Standard Deviation 10.06 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 2 | 1.8 mmHg | Standard Deviation 9.15 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 13 | 1.4 mmHg | Standard Deviation 9.93 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 26 | -6.9 mmHg | Standard Deviation 14.99 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 1 | 2.0 mmHg | Standard Deviation 12.84 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 39 (n=7,7,9,23) | -3.9 mmHg | Standard Deviation 3.56 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 52 | 0.1 mmHg | Standard Deviation 10.81 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Day 2 | 0.4 mmHg | Standard Deviation 10.36 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 3 (n=6,8,9,23) | -0.7 mmHg | Standard Deviation 7.25 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 4 (n=6,8,8,22) | 6.8 mmHg | Standard Deviation 10.02 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 6 (n=7,8,8,23) | 5.4 mmHg | Standard Deviation 15.39 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Day 2 | 1.3 mmHg | Standard Deviation 11.75 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 39 (n=7,7,9,23) | 0.3 mmHg | Standard Deviation 21.98 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 8 (n=7,7,9,23) | 2.3 mmHg | Standard Deviation 13.69 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 3 (n=6,8,9,23) | 2.7 mmHg | Standard Deviation 12.33 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 26 | -8.5 mmHg | Standard Deviation 13.46 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 13 | 0.8 mmHg | Standard Deviation 13.28 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 6 (n=7,8,8,23) | 3.4 mmHg | Standard Deviation 18.13 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 4 (n=6,8,8,22) | 4.5 mmHg | Standard Deviation 15.05 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 10 | 2.8 mmHg | Standard Deviation 14.32 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 52 | -1.1 mmHg | Standard Deviation 15.67 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 2 | 2.8 mmHg | Standard Deviation 16.45 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Week 1 | 2.2 mmHg | Standard Deviation 15.18 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg) | Change from baseline at Day 3 | -1.4 mmHg | Standard Deviation 11.62 |
Primary
Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius)
Time frame: Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52
Population: Safety set
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 13 | 0.13 degrees Celsius | Standard Deviation 0.399 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 6 (n=7,8,8,23) | 0.26 degrees Celsius | Standard Deviation 0.458 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 8 (n=7,7,9,23) | 0.13 degrees Celsius | Standard Deviation 0.547 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Day 2 | 0.07 degrees Celsius | Standard Deviation 0.502 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 10 | 0.34 degrees Celsius | Standard Deviation 0.351 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 1 | -0.07 degrees Celsius | Standard Deviation 0.386 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 39 (n=7,7,9,23) | 0.33 degrees Celsius | Standard Deviation 0.35 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 2 | 0.30 degrees Celsius | Standard Deviation 0.592 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 26 | 0.31 degrees Celsius | Standard Deviation 0.453 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 3 (n=6,8,9,23) | 0.32 degrees Celsius | Standard Deviation 0.556 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 52 | 0.63 degrees Celsius | Standard Deviation 0.836 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 4 (n=7,8,8,23) | 0.16 degrees Celsius | Standard Deviation 0.673 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Day 3 | 0.11 degrees Celsius | Standard Deviation 0.261 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 13 | -0.06 degrees Celsius | Standard Deviation 0.701 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 3 (n=6,8,9,23) | -0.24 degrees Celsius | Standard Deviation 0.403 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 6 (n=7,8,8,23) | -0.18 degrees Celsius | Standard Deviation 0.599 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Day 3 | -0.48 degrees Celsius | Standard Deviation 0.719 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 2 | -0.41 degrees Celsius | Standard Deviation 0.649 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 8 (n=7,7,9,23) | -0.21 degrees Celsius | Standard Deviation 0.654 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 52 | -0.53 degrees Celsius | Standard Deviation 0.819 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 10 | -0.45 degrees Celsius | Standard Deviation 0.727 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 26 | -0.15 degrees Celsius | Standard Deviation 0.532 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 39 (n=7,7,9,23) | -0.10 degrees Celsius | Standard Deviation 0.622 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 4 (n=7,8,8,23) | -0.31 degrees Celsius | Standard Deviation 0.775 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 1 | -0.44 degrees Celsius | Standard Deviation 0.563 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Day 2 | -0.11 degrees Celsius | Standard Deviation 0.714 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 8 (n=7,7,9,23) | 0.01 degrees Celsius | Standard Deviation 0.386 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Day 2 | 0.07 degrees Celsius | Standard Deviation 0.918 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Day 3 | 0.12 degrees Celsius | Standard Deviation 0.319 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 1 | 0.00 degrees Celsius | Standard Deviation 0.548 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 2 | -0.09 degrees Celsius | Standard Deviation 0.478 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 3 (n=6,8,9,23) | 0.02 degrees Celsius | Standard Deviation 0.387 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 4 (n=7,8,8,23) | 0.05 degrees Celsius | Standard Deviation 0.431 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 6 (n=7,8,8,23) | -0.09 degrees Celsius | Standard Deviation 0.5 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 52 | -0.11 degrees Celsius | Standard Deviation 0.401 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 10 | 0.01 degrees Celsius | Standard Deviation 0.553 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 13 | 0.09 degrees Celsius | Standard Deviation 0.473 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 26 | 0.03 degrees Celsius | Standard Deviation 0.324 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 39 (n=7,7,9,23) | -0.11 degrees Celsius | Standard Deviation 0.491 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 6 (n=7,8,8,23) | -0.01 degrees Celsius | Standard Deviation 0.535 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 39 (n=7,7,9,23) | 0.03 degrees Celsius | Standard Deviation 0.518 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 13 | 0.05 degrees Celsius | Standard Deviation 0.525 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 3 (n=6,8,9,23) | 0.01 degrees Celsius | Standard Deviation 0.474 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 2 | -0.08 degrees Celsius | Standard Deviation 0.618 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Day 2 | 0.01 degrees Celsius | Standard Deviation 0.722 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 26 | 0.05 degrees Celsius | Standard Deviation 0.46 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 1 | -0.17 degrees Celsius | Standard Deviation 0.528 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Day 3 | -0.08 degrees Celsius | Standard Deviation 0.541 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 8 (n=7,7,9,23) | -0.02 degrees Celsius | Standard Deviation 0.521 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 52 | -0.03 degrees Celsius | Standard Deviation 0.814 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 10 | -0.05 degrees Celsius | Standard Deviation 0.635 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius) | Change from baseline at Week 4 (n=7,8,8,23) | -0.04 degrees Celsius | Standard Deviation 0.645 |
Primary
Number of Participants With Important Identified and Important Potential Risks (Adverse Events of Special Interest (AESI)) by Risk Name and Weight Bracket
Important identified and important potential risks included the following AESIs: Hepatotoxicity, Thrombocytopenia, Cardiac adverse events, Dorsal root ganglia toxicity and Thrombotic microangiopathy. These were assessed by the investigator.
Time frame: Up to Month 12
Population: Safety set
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| OAV101 1.1e14 vg/kg 8.5-13 kg | Number of Participants With Important Identified and Important Potential Risks (Adverse Events of Special Interest (AESI)) by Risk Name and Weight Bracket | Cardiac adverse events | 0 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Number of Participants With Important Identified and Important Potential Risks (Adverse Events of Special Interest (AESI)) by Risk Name and Weight Bracket | Dorsal root ganglia cell inflammation | 0 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Number of Participants With Important Identified and Important Potential Risks (Adverse Events of Special Interest (AESI)) by Risk Name and Weight Bracket | Hepatotoxicity | 6 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Number of Participants With Important Identified and Important Potential Risks (Adverse Events of Special Interest (AESI)) by Risk Name and Weight Bracket | Thrombotic microangiopathy | 0 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Number of Participants With Important Identified and Important Potential Risks (Adverse Events of Special Interest (AESI)) by Risk Name and Weight Bracket | Transient thrombocytopenia | 4 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Number of Participants With Important Identified and Important Potential Risks (Adverse Events of Special Interest (AESI)) by Risk Name and Weight Bracket | Cardiac adverse events | 2 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Number of Participants With Important Identified and Important Potential Risks (Adverse Events of Special Interest (AESI)) by Risk Name and Weight Bracket | Transient thrombocytopenia | 6 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Number of Participants With Important Identified and Important Potential Risks (Adverse Events of Special Interest (AESI)) by Risk Name and Weight Bracket | Dorsal root ganglia cell inflammation | 0 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Number of Participants With Important Identified and Important Potential Risks (Adverse Events of Special Interest (AESI)) by Risk Name and Weight Bracket | Hepatotoxicity | 5 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Number of Participants With Important Identified and Important Potential Risks (Adverse Events of Special Interest (AESI)) by Risk Name and Weight Bracket | Thrombotic microangiopathy | 0 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Number of Participants With Important Identified and Important Potential Risks (Adverse Events of Special Interest (AESI)) by Risk Name and Weight Bracket | Transient thrombocytopenia | 7 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Number of Participants With Important Identified and Important Potential Risks (Adverse Events of Special Interest (AESI)) by Risk Name and Weight Bracket | Dorsal root ganglia cell inflammation | 0 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Number of Participants With Important Identified and Important Potential Risks (Adverse Events of Special Interest (AESI)) by Risk Name and Weight Bracket | Thrombotic microangiopathy | 0 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Number of Participants With Important Identified and Important Potential Risks (Adverse Events of Special Interest (AESI)) by Risk Name and Weight Bracket | Cardiac adverse events | 1 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Number of Participants With Important Identified and Important Potential Risks (Adverse Events of Special Interest (AESI)) by Risk Name and Weight Bracket | Hepatotoxicity | 9 Participants |
| OAV101 1.1e14 vg/kg Overall | Number of Participants With Important Identified and Important Potential Risks (Adverse Events of Special Interest (AESI)) by Risk Name and Weight Bracket | Dorsal root ganglia cell inflammation | 0 Participants |
| OAV101 1.1e14 vg/kg Overall | Number of Participants With Important Identified and Important Potential Risks (Adverse Events of Special Interest (AESI)) by Risk Name and Weight Bracket | Hepatotoxicity | 20 Participants |
| OAV101 1.1e14 vg/kg Overall | Number of Participants With Important Identified and Important Potential Risks (Adverse Events of Special Interest (AESI)) by Risk Name and Weight Bracket | Transient thrombocytopenia | 17 Participants |
| OAV101 1.1e14 vg/kg Overall | Number of Participants With Important Identified and Important Potential Risks (Adverse Events of Special Interest (AESI)) by Risk Name and Weight Bracket | Cardiac adverse events | 3 Participants |
| OAV101 1.1e14 vg/kg Overall | Number of Participants With Important Identified and Important Potential Risks (Adverse Events of Special Interest (AESI)) by Risk Name and Weight Bracket | Thrombotic microangiopathy | 0 Participants |
Primary
Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket
An AE is any untoward medical occurrence (e.g. any unfavorable and unintended sign \[including abnormal laboratory findings\], symptom or disease) in a clinical investigation participant after providing written informed consent for participation in the study.
Time frame: Up to Month 12
Population: Safety set (The Safety Set comprised all participants who were administered investigational drug.)
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| OAV101 1.1e14 vg/kg 8.5-13 kg | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Any treatment-emergent adverse events related to OAV101 | 7 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Any treatment-emergent adverse events | 7 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Any severe treatment-emergent adverse events | 1 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Any serious treatment-emergent adverse events | 3 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Serious treatment-emergent adverse events related to OAV101 | 1 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Treatment-emergent adverse events leading to study discontinuation | 0 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Treatment-emergent adverse events leading to death | 0 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Treatment-emergent adverse events of special interest | 7 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Treatment-emergent adverse events leading to study discontinuation | 0 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Serious treatment-emergent adverse events related to OAV101 | 4 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Any treatment-emergent adverse events | 8 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Treatment-emergent adverse events of special interest | 7 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Treatment-emergent adverse events leading to death | 0 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Any serious treatment-emergent adverse events | 7 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Any severe treatment-emergent adverse events | 4 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Any treatment-emergent adverse events related to OAV101 | 8 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Treatment-emergent adverse events leading to death | 0 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Any severe treatment-emergent adverse events | 3 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Any serious treatment-emergent adverse events | 5 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Serious treatment-emergent adverse events related to OAV101 | 2 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Treatment-emergent adverse events leading to study discontinuation | 0 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Treatment-emergent adverse events of special interest | 9 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Any treatment-emergent adverse events | 9 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Any treatment-emergent adverse events related to OAV101 | 9 Participants |
| OAV101 1.1e14 vg/kg Overall | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Any treatment-emergent adverse events | 24 Participants |
| OAV101 1.1e14 vg/kg Overall | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Any serious treatment-emergent adverse events | 15 Participants |
| OAV101 1.1e14 vg/kg Overall | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Treatment-emergent adverse events leading to death | 0 Participants |
| OAV101 1.1e14 vg/kg Overall | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Any severe treatment-emergent adverse events | 8 Participants |
| OAV101 1.1e14 vg/kg Overall | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Serious treatment-emergent adverse events related to OAV101 | 7 Participants |
| OAV101 1.1e14 vg/kg Overall | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Treatment-emergent adverse events of special interest | 23 Participants |
| OAV101 1.1e14 vg/kg Overall | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Any treatment-emergent adverse events related to OAV101 | 24 Participants |
| OAV101 1.1e14 vg/kg Overall | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket | Treatment-emergent adverse events leading to study discontinuation | 0 Participants |
Primary
Summary of Participants Meeting Criteria for Potentially Clinically Significant Vital Sign Values by Weight Bracket - Systolic and Diastolic Blood Pressure
Change from baseline in vital signs measurements - systolic and diastolic blood pressure (mmHg). Systolic Blood Pressure-Low:\<=5th percentile of the age(Any Age), High:\>=90th percentile of the age, gender, and height group (\<18 yrs). Diastolic Blood Pressure-High:\>=90th percentile of the age, gender, and height group(\<18 yrs).
Time frame: 12 months
Population: Safety set
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| OAV101 1.1e14 vg/kg 8.5-13 kg | Summary of Participants Meeting Criteria for Potentially Clinically Significant Vital Sign Values by Weight Bracket - Systolic and Diastolic Blood Pressure | Systolic Blood Pressure (mmHg) Low | 1 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Summary of Participants Meeting Criteria for Potentially Clinically Significant Vital Sign Values by Weight Bracket - Systolic and Diastolic Blood Pressure | Diastolic Blood Pressure (mmHg) High | 7 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Summary of Participants Meeting Criteria for Potentially Clinically Significant Vital Sign Values by Weight Bracket - Systolic and Diastolic Blood Pressure | Systolic Blood Pressure (mmHg) High | 7 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Summary of Participants Meeting Criteria for Potentially Clinically Significant Vital Sign Values by Weight Bracket - Systolic and Diastolic Blood Pressure | Systolic Blood Pressure (mmHg) Low | 0 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Summary of Participants Meeting Criteria for Potentially Clinically Significant Vital Sign Values by Weight Bracket - Systolic and Diastolic Blood Pressure | Diastolic Blood Pressure (mmHg) High | 7 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Summary of Participants Meeting Criteria for Potentially Clinically Significant Vital Sign Values by Weight Bracket - Systolic and Diastolic Blood Pressure | Systolic Blood Pressure (mmHg) High | 8 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Summary of Participants Meeting Criteria for Potentially Clinically Significant Vital Sign Values by Weight Bracket - Systolic and Diastolic Blood Pressure | Systolic Blood Pressure (mmHg) High | 8 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Summary of Participants Meeting Criteria for Potentially Clinically Significant Vital Sign Values by Weight Bracket - Systolic and Diastolic Blood Pressure | Systolic Blood Pressure (mmHg) Low | 1 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Summary of Participants Meeting Criteria for Potentially Clinically Significant Vital Sign Values by Weight Bracket - Systolic and Diastolic Blood Pressure | Diastolic Blood Pressure (mmHg) High | 8 Participants |
| OAV101 1.1e14 vg/kg Overall | Summary of Participants Meeting Criteria for Potentially Clinically Significant Vital Sign Values by Weight Bracket - Systolic and Diastolic Blood Pressure | Systolic Blood Pressure (mmHg) Low | 2 Participants |
| OAV101 1.1e14 vg/kg Overall | Summary of Participants Meeting Criteria for Potentially Clinically Significant Vital Sign Values by Weight Bracket - Systolic and Diastolic Blood Pressure | Diastolic Blood Pressure (mmHg) High | 22 Participants |
| OAV101 1.1e14 vg/kg Overall | Summary of Participants Meeting Criteria for Potentially Clinically Significant Vital Sign Values by Weight Bracket - Systolic and Diastolic Blood Pressure | Systolic Blood Pressure (mmHg) High | 23 Participants |
Primary
Summary of Participants Meeting Criteria for Potentially Clinically Significant Vital Sign Values by Weight Bracket - Temperature
Change from baseline in vital signs measurements - temperature (degrees Celsius) Temperature-Low:\<=35ºC(Any Age),High:\>=38.4ºC(\<18 yrs).
Time frame: 12 months
Population: Safety set
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| OAV101 1.1e14 vg/kg 8.5-13 kg | Summary of Participants Meeting Criteria for Potentially Clinically Significant Vital Sign Values by Weight Bracket - Temperature | Temperature (ºC) Low | 0 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Summary of Participants Meeting Criteria for Potentially Clinically Significant Vital Sign Values by Weight Bracket - Temperature | Temperature (ºC) High | 1 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Summary of Participants Meeting Criteria for Potentially Clinically Significant Vital Sign Values by Weight Bracket - Temperature | Temperature (ºC) High | 0 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Summary of Participants Meeting Criteria for Potentially Clinically Significant Vital Sign Values by Weight Bracket - Temperature | Temperature (ºC) Low | 0 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Summary of Participants Meeting Criteria for Potentially Clinically Significant Vital Sign Values by Weight Bracket - Temperature | Temperature (ºC) Low | 0 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Summary of Participants Meeting Criteria for Potentially Clinically Significant Vital Sign Values by Weight Bracket - Temperature | Temperature (ºC) High | 1 Participants |
| OAV101 1.1e14 vg/kg Overall | Summary of Participants Meeting Criteria for Potentially Clinically Significant Vital Sign Values by Weight Bracket - Temperature | Temperature (ºC) Low | 0 Participants |
| OAV101 1.1e14 vg/kg Overall | Summary of Participants Meeting Criteria for Potentially Clinically Significant Vital Sign Values by Weight Bracket - Temperature | Temperature (ºC) High | 2 Participants |
Secondary
Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development.
The World Health Organization-Multicentre Growth Reference Study (WHO-MGRS) and Bayley scale of Infant and Toddler Development was modified and combined into a single scale expressly for this study, to measure developmental motor milestones. These were assessed via the milestone checklist, formed of 10 yes/no questions with optional video documentation. The developmental milestones are: head control, sitting with support, sitting without support, sitting without support for 30 seconds, hands-and-knees crawling, pulls to stand, standing with assistance, walking with assistance, standing alone and walking alone. A yes response indicates that the patient reached a particular development milestone.
Time frame: Baseline, Week 26 and Week 52
Population: Full analysis set
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| OAV101 1.1e14 vg/kg 8.5-13 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Standing with assistance (WHO MGRS) | 1 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Walking with assistance (WHO MGRS) | 0 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Standing with assistance (WHO MGRS) | 0 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Standing with assistance (WHO MGRS) | 2 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Head control (Bayley GM #4) | 7 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Sits without support 30 s (Bayley GM #26) | 6 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Sitting without support (WHO MGRS) | 6 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Walking with assistance (WHO MGRS) | 1 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Pulls to stand (Bayley GM #35) | 1 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Sits with support (Bayley GM #19) | 7 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Hands-and-knees crawling (WHO MGRS) | 1 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Standing alone (WHO MGRS) | 0 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Head control (Bayley GM #4) | 7 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Walking alone (WHO MGRS) | 0 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Standing alone (WHO MGRS) | 0 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Pulls to stand (Bayley GM #35) | 2 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Hands-and-knees crawling (WHO MGRS) | 2 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Sitting without support (WHO MGRS) | 7 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Walking with assistance (WHO MGRS) | 1 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Sits without support 30 s (Bayley GM #26) | 7 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Sitting without support (WHO MGRS) | 7 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Sits without support 30 s (Bayley GM #26) | 7 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Walking alone (WHO MGRS) | 0 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Sits with support (Bayley GM #19) | 7 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Head control (Bayley GM #4) | 7 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Hands-and-knees crawling (WHO MGRS) | 2 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Sits with support (Bayley GM #19) | 7 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Walking alone (WHO MGRS) | 0 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Standing alone (WHO MGRS) | 0 Participants |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Pulls to stand (Bayley GM #35) (n=7,8,8,23) | 2 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Pulls to stand (Bayley GM #35) | 3 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Head control (Bayley GM #4) | 8 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Pulls to stand (Bayley GM #35) | 3 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Walking alone (WHO MGRS) | 2 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Walking alone (WHO MGRS) | 2 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Head control (Bayley GM #4) | 8 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Sits with support (Bayley GM #19) | 7 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Sitting without support (WHO MGRS) | 7 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Sits without support 30 s (Bayley GM #26) | 6 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Hands-and-knees crawling (WHO MGRS) | 3 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Pulls to stand (Bayley GM #35) (n=7,8,8,23) | 3 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Standing with assistance (WHO MGRS) | 3 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Walking with assistance (WHO MGRS) | 3 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Standing alone (WHO MGRS) | 3 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Walking alone (WHO MGRS) | 2 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Hands-and-knees crawling (WHO MGRS) | 3 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Sits with support (Bayley GM #19) | 8 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Sitting without support (WHO MGRS) | 7 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Sits without support 30 s (Bayley GM #26) | 7 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Standing with assistance (WHO MGRS) | 2 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Walking with assistance (WHO MGRS) | 2 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Standing alone (WHO MGRS) | 2 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Head control (Bayley GM #4) | 8 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Sits with support (Bayley GM #19) | 8 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Sitting without support (WHO MGRS) | 5 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Sits without support 30 s (Bayley GM #26) | 5 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Hands-and-knees crawling (WHO MGRS) | 3 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Standing with assistance (WHO MGRS) | 3 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Walking with assistance (WHO MGRS) | 3 Participants |
| OAV101 1.1e14 vg/kg >13-17 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Standing alone (WHO MGRS) | 3 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Walking alone (WHO MGRS) | 4 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Sitting without support (WHO MGRS) | 8 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Standing with assistance (WHO MGRS) | 5 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Head control (Bayley GM #4) | 9 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Sits without support 30 s (Bayley GM #26) | 8 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Pulls to stand (Bayley GM #35) | 5 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Standing with assistance (WHO MGRS) | 5 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Standing with assistance (WHO MGRS) | 5 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Pulls to stand (Bayley GM #35) (n=7,8,8,23) | 5 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Walking with assistance (WHO MGRS) | 5 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Standing alone (WHO MGRS) | 4 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Hands-and-knees crawling (WHO MGRS) | 6 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Walking alone (WHO MGRS) | 4 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Head control (Bayley GM #4) | 9 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Sits without support 30 s (Bayley GM #26) | 8 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Walking with assistance (WHO MGRS) | 4 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Sits with support (Bayley GM #19) | 9 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Sitting without support (WHO MGRS) | 9 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Sitting without support (WHO MGRS) | 8 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Standing alone (WHO MGRS) | 4 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Sits without support 30 s (Bayley GM #26) | 8 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Hands-and-knees crawling (WHO MGRS) | 6 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Sits with support (Bayley GM #19) | 9 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Standing alone (WHO MGRS) | 4 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Walking alone (WHO MGRS) | 4 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Head control (Bayley GM #4) | 9 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Pulls to stand (Bayley GM #35) | 5 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Hands-and-knees crawling (WHO MGRS) | 6 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Walking with assistance (WHO MGRS) | 4 Participants |
| OAV101 1.1e14 vg/kg >17-21 kg | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Sits with support (Bayley GM #19) | 9 Participants |
| OAV101 1.1e14 vg/kg Overall | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Sits with support (Bayley GM #19) | 24 Participants |
| OAV101 1.1e14 vg/kg Overall | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Sitting without support (WHO MGRS) | 22 Participants |
| OAV101 1.1e14 vg/kg Overall | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Sitting without support (WHO MGRS) | 21 Participants |
| OAV101 1.1e14 vg/kg Overall | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Walking with assistance (WHO MGRS) | 8 Participants |
| OAV101 1.1e14 vg/kg Overall | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Head control (Bayley GM #4) | 24 Participants |
| OAV101 1.1e14 vg/kg Overall | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Sitting without support (WHO MGRS) | 21 Participants |
| OAV101 1.1e14 vg/kg Overall | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Sits without support 30 s (Bayley GM #26) | 21 Participants |
| OAV101 1.1e14 vg/kg Overall | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Walking alone (WHO MGRS) | 6 Participants |
| OAV101 1.1e14 vg/kg Overall | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Pulls to stand (Bayley GM #35) | 9 Participants |
| OAV101 1.1e14 vg/kg Overall | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Pulls to stand (Bayley GM #35) (n=7,8,8,23) | 10 Participants |
| OAV101 1.1e14 vg/kg Overall | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Sits with support (Bayley GM #19) | 24 Participants |
| OAV101 1.1e14 vg/kg Overall | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Walking with assistance (WHO MGRS) | 8 Participants |
| OAV101 1.1e14 vg/kg Overall | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Standing with assistance (WHO MGRS) | 7 Participants |
| OAV101 1.1e14 vg/kg Overall | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Sits without support 30 s (Bayley GM #26) | 20 Participants |
| OAV101 1.1e14 vg/kg Overall | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Standing with assistance (WHO MGRS) | 9 Participants |
| OAV101 1.1e14 vg/kg Overall | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Sits with support (Bayley GM #19) | 23 Participants |
| OAV101 1.1e14 vg/kg Overall | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Hands-and-knees crawling (WHO MGRS) | 11 Participants |
| OAV101 1.1e14 vg/kg Overall | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Standing alone (WHO MGRS) | 7 Participants |
| OAV101 1.1e14 vg/kg Overall | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Walking with assistance (WHO MGRS) | 7 Participants |
| OAV101 1.1e14 vg/kg Overall | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Standing alone (WHO MGRS) | 6 Participants |
| OAV101 1.1e14 vg/kg Overall | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Pulls to stand (Bayley GM #35) | 10 Participants |
| OAV101 1.1e14 vg/kg Overall | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Hands-and-knees crawling (WHO MGRS) | 11 Participants |
| OAV101 1.1e14 vg/kg Overall | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Walking alone (WHO MGRS) | 6 Participants |
| OAV101 1.1e14 vg/kg Overall | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Sits without support 30 s (Bayley GM #26) | 21 Participants |
| OAV101 1.1e14 vg/kg Overall | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Head control (Bayley GM #4) | 24 Participants |
| OAV101 1.1e14 vg/kg Overall | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Standing with assistance (WHO MGRS) | 10 Participants |
| OAV101 1.1e14 vg/kg Overall | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Head control (Bayley GM #4) | 24 Participants |
| OAV101 1.1e14 vg/kg Overall | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 52 Standing alone (WHO MGRS) | 7 Participants |
| OAV101 1.1e14 vg/kg Overall | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Baseline Hands-and-knees crawling (WHO MGRS) | 10 Participants |
| OAV101 1.1e14 vg/kg Overall | Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development. | Week 26 Walking alone (WHO MGRS) | 6 Participants |
Secondary
Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE), as Appropriate According to Participant Age
The HFMSE was devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE is formed of 33 assessments. Each motor skill item is scored on a 3 point Likert scale from 0 (no response) to 2 (full response), with a total score range of 0 to 66. A higher score indicates a higher level of ability.
Time frame: Baseline, Week 4, Week 13, Week 26, Week 39 and Week 52
Population: Participants in the Full Analysis Set (FAS) who had an available value for the outcome measure at the specified timepoints. The FAS includes all participants who were administered investigational drug.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE), as Appropriate According to Participant Age | Change from baseline at Week 39 (n=5,5,9,19) | 2.6 HFMSE total score on a scale | Standard Deviation 6.11 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE), as Appropriate According to Participant Age | Change from baseline at Week 13 (n=4,6,9,19) | 1.3 HFMSE total score on a scale | Standard Deviation 7.89 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE), as Appropriate According to Participant Age | Change from baseline at Week 52 (n=5,6,7,18) | 3.0 HFMSE total score on a scale | Standard Deviation 5.24 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE), as Appropriate According to Participant Age | Change from baseline at Week 26 (n=5,6,9,20) | 3.4 HFMSE total score on a scale | Standard Deviation 5.18 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE), as Appropriate According to Participant Age | Change from baseline at Week 4 (n=4,6,9,19) | 3.8 HFMSE total score on a scale | Standard Deviation 2.99 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE), as Appropriate According to Participant Age | Change from baseline at Week 26 (n=5,6,9,20) | 4.3 HFMSE total score on a scale | Standard Deviation 5.05 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE), as Appropriate According to Participant Age | Change from baseline at Week 39 (n=5,5,9,19) | -0.6 HFMSE total score on a scale | Standard Deviation 4.72 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE), as Appropriate According to Participant Age | Change from baseline at Week 52 (n=5,6,7,18) | 3.7 HFMSE total score on a scale | Standard Deviation 5.75 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE), as Appropriate According to Participant Age | Change from baseline at Week 13 (n=4,6,9,19) | 4.5 HFMSE total score on a scale | Standard Deviation 3.99 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE), as Appropriate According to Participant Age | Change from baseline at Week 4 (n=4,6,9,19) | 2.3 HFMSE total score on a scale | Standard Deviation 2.66 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE), as Appropriate According to Participant Age | Change from baseline at Week 26 (n=5,6,9,20) | 3.1 HFMSE total score on a scale | Standard Deviation 4.14 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE), as Appropriate According to Participant Age | Change from baseline at Week 4 (n=4,6,9,19) | 3.1 HFMSE total score on a scale | Standard Deviation 3.1 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE), as Appropriate According to Participant Age | Change from baseline at Week 13 (n=4,6,9,19) | 3.9 HFMSE total score on a scale | Standard Deviation 3.98 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE), as Appropriate According to Participant Age | Change from baseline at Week 39 (n=5,5,9,19) | 4.3 HFMSE total score on a scale | Standard Deviation 3.71 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE), as Appropriate According to Participant Age | Change from baseline at Week 52 (n=5,6,7,18) | 4.3 HFMSE total score on a scale | Standard Deviation 4.07 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE), as Appropriate According to Participant Age | Change from baseline at Week 39 (n=5,5,9,19) | 2.6 HFMSE total score on a scale | Standard Deviation 4.87 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE), as Appropriate According to Participant Age | Change from baseline at Week 13 (n=4,6,9,19) | 3.5 HFMSE total score on a scale | Standard Deviation 4.83 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE), as Appropriate According to Participant Age | Change from baseline at Week 4 (n=4,6,9,19) | 3.0 HFMSE total score on a scale | Standard Deviation 2.83 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE), as Appropriate According to Participant Age | Change from baseline at Week 26 (n=5,6,9,20) | 3.6 HFMSE total score on a scale | Standard Deviation 4.45 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE), as Appropriate According to Participant Age | Change from baseline at Week 52 (n=5,6,7,18) | 3.7 HFMSE total score on a scale | Standard Deviation 4.73 |
Secondary
Change From Baseline in Revised Upper Limb Module (RULM), as Appropriate According to Participant Age.
The RULM assesses motor performance in the upper limbs from childhood through adulthood in ambulatory and non-ambulatory individuals with SMA. 'The scale consists of an entry item to establish functional levels and 19 items covering distal to proximal movements. The entry item is a modified version of the Brooke scale, including activities ranging from no functional use of hands (score 0) to full bilateral shoulder abduction (score 6). The entry item does not contribute to the total score but serves as a functional classification of overall upper limb functional ability. Of the remaining 19 items, 18 are scored on a 3 point scoring system and 1 item is scored on a 2 point scoring system. The test is performed unilaterally using the limb preferred by the participant. The total score ranges from 0, if all the items cannot be performed, to 37, if all the activities are achieved fully without any compensation. ' Higher scores indicate higher levels of motor ability.
Time frame: Baseline, Week 4, Week 13, Week 26, Week 39 and Week 52
Population: Participants in the Full Analysis Set (FAS) who had an available value for the outcome measure at the specified timepoints. The FAS includes all participants who were administered investigational drug.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Revised Upper Limb Module (RULM), as Appropriate According to Participant Age. | Change from baseline at Week 52 (n=3,6,8,17) | 6.0 RULM total score on a scale | Standard Deviation 3.46 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Revised Upper Limb Module (RULM), as Appropriate According to Participant Age. | Change from baseline at Week 4 | 2.8 RULM total score on a scale | Standard Deviation 2.36 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Revised Upper Limb Module (RULM), as Appropriate According to Participant Age. | Change from baseline at Week 26 | 5.3 RULM total score on a scale | Standard Deviation 2.5 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Revised Upper Limb Module (RULM), as Appropriate According to Participant Age. | Change from baseline at Week 13 | 4.3 RULM total score on a scale | Standard Deviation 1.5 |
| OAV101 1.1e14 vg/kg 8.5-13 kg | Change From Baseline in Revised Upper Limb Module (RULM), as Appropriate According to Participant Age. | Change from baseline at Week 39 (n=4,5,8,17) | 7.3 RULM total score on a scale | Standard Deviation 2.22 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Revised Upper Limb Module (RULM), as Appropriate According to Participant Age. | Change from baseline at Week 39 (n=4,5,8,17) | -0.6 RULM total score on a scale | Standard Deviation 1.34 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Revised Upper Limb Module (RULM), as Appropriate According to Participant Age. | Change from baseline at Week 4 | 1.2 RULM total score on a scale | Standard Deviation 1.33 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Revised Upper Limb Module (RULM), as Appropriate According to Participant Age. | Change from baseline at Week 13 | 0.8 RULM total score on a scale | Standard Deviation 2.32 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Revised Upper Limb Module (RULM), as Appropriate According to Participant Age. | Change from baseline at Week 26 | 1.3 RULM total score on a scale | Standard Deviation 1.21 |
| OAV101 1.1e14 vg/kg >13-17 kg | Change From Baseline in Revised Upper Limb Module (RULM), as Appropriate According to Participant Age. | Change from baseline at Week 52 (n=3,6,8,17) | 0.3 RULM total score on a scale | Standard Deviation 1.63 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Revised Upper Limb Module (RULM), as Appropriate According to Participant Age. | Change from baseline at Week 39 (n=4,5,8,17) | 2.0 RULM total score on a scale | Standard Deviation 4.34 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Revised Upper Limb Module (RULM), as Appropriate According to Participant Age. | Change from baseline at Week 13 | 2.0 RULM total score on a scale | Standard Deviation 2.56 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Revised Upper Limb Module (RULM), as Appropriate According to Participant Age. | Change from baseline at Week 52 (n=3,6,8,17) | 1.8 RULM total score on a scale | Standard Deviation 4.71 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Revised Upper Limb Module (RULM), as Appropriate According to Participant Age. | Change from baseline at Week 4 | 1.0 RULM total score on a scale | Standard Deviation 1.93 |
| OAV101 1.1e14 vg/kg >17-21 kg | Change From Baseline in Revised Upper Limb Module (RULM), as Appropriate According to Participant Age. | Change from baseline at Week 26 | 1.6 RULM total score on a scale | Standard Deviation 2.88 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Revised Upper Limb Module (RULM), as Appropriate According to Participant Age. | Change from baseline at Week 52 (n=3,6,8,17) | 2.0 RULM total score on a scale | Standard Deviation 4.02 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Revised Upper Limb Module (RULM), as Appropriate According to Participant Age. | Change from baseline at Week 39 (n=4,5,8,17) | 2.5 RULM total score on a scale | Standard Deviation 4.29 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Revised Upper Limb Module (RULM), as Appropriate According to Participant Age. | Change from baseline at Week 13 | 2.1 RULM total score on a scale | Standard Deviation 2.52 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Revised Upper Limb Module (RULM), as Appropriate According to Participant Age. | Change from baseline at Week 4 | 1.4 RULM total score on a scale | Standard Deviation 1.89 |
| OAV101 1.1e14 vg/kg Overall | Change From Baseline in Revised Upper Limb Module (RULM), as Appropriate According to Participant Age. | Change from baseline at Week 26 | 2.3 RULM total score on a scale | Standard Deviation 2.74 |