Clinical Trials List
Protocol NumberCOAV101A12306
NCT Number(ClinicalTrials.gov Identfier)NCT04851873
Completed
2021-09-30 - 2023-08-01
Phase III
Recruiting1
Study ended1
A Phase IIIb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerability and efficacy of gene replacement therapy with intravenous OAV101 (AVXS-101) in pediatric patients with spinal muscular atrophy (SMA)
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Trial Applicant
NOVARTIS (TAIWAN) CO., LTD.
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Sponsor
Novartis Pharmaceuticals
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Trial scale
Multi-Regional Multi-Center
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Update
2026/02/01
Investigators and Locations
Co-Principal Investigator
- 呂立 未分科
- NI-CHUNG LEE 未分科
- WANG-TSO LEE 未分科
- WUH-LIANG HWU 未分科
- 陳俊安 未分科
- 謝正宜 未分科
- WEN-CHIN WENG 未分科
The Actual Total Number of Participants Enrolled
0 Recruiting
Principal Investigator
Yuh-Jyh Jong
Co-Principal Investigator
- 王晨華
- 施相宏 未分科
- 劉怡慶
- Wen-Chen Liang
The Actual Total Number of Participants Enrolled
0 Study ended
Condition/Disease
Pediatric patients with spinal muscular atrophy (SMA)
Objectives
Primary ObjectiveTo evaluate the safety and tolerability over a 12-month period following a single intravenous (IV) infusion of OAV101 in participants with spinal muscular atrophy (SMA) weighing ≥8.5 kg and ≤21 kg.Secondary ObjectivesTo assess the efficacy of OAV101 administered intravenously in participants with SMA weighing ≥8.5 kg and ≤21 kg at 6 months and 12 months post-treatment, based on the following measures of change from baseline:Developmental milestones corresponding to motor development indices, assessed according to the World Health Organization Multicentre Growth Reference Study (WHO-MGRS) and Bayley Scales of Infant and Toddler Development, Third Edition (Bayley-III);Hammersmith Functional Motor Scale – Expanded (HFMSE);Revised Upper Limb Module (RULM), adjusted for participant age.
Test Drug
OAV101
Active Ingredient
onasemnogene abeparvovec
Dosage Form
recombinant adeno-associated virus serotype 9 (AAV9) vector suspension for intravenous infusion
Dosage
mL
Endpoints
Efficacy Assessments:
Developmental motor milestones will be evaluated using the following standardized measures and definitions:
World Health Organization Multicentre Growth Reference Study (WHO-MGRS) and Bayley Scales of Infant and Toddler Development, Third Edition (Bayley-III);
Hammersmith Functional Motor Scale – Expanded (HFMSE);
Revised Upper Limb Module (RULM), adjusted for participant age.
Primary Safety Assessments:
The safety and tolerability of OAV101 treatment will be evaluated through assessment of adverse events (AEs), laboratory test results, vital signs, and cardiac safety monitoring.
Developmental motor milestones will be evaluated using the following standardized measures and definitions:
World Health Organization Multicentre Growth Reference Study (WHO-MGRS) and Bayley Scales of Infant and Toddler Development, Third Edition (Bayley-III);
Hammersmith Functional Motor Scale – Expanded (HFMSE);
Revised Upper Limb Module (RULM), adjusted for participant age.
Primary Safety Assessments:
The safety and tolerability of OAV101 treatment will be evaluated through assessment of adverse events (AEs), laboratory test results, vital signs, and cardiac safety monitoring.
Inclution 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
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 Criteria
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.
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.
The Estimated Number of Participants
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Taiwan
2 participants
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Global
24 participants
