At a glance
ClinicalIndex Comparison RecordPhase 3Completed· 24 enrolled
Drug / intervention
OAV101genetic
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
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)
In Brief
A Phase 3 clinical trial evaluating OAV101 for Spinal Muscular Atrophy. Completed, enrolled 24 participants across 13 sites in 9 countries.
Detailed 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.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsSpinal Muscular Atrophy
CountriesAustralia, Belgium, Canada, France, Italy, Portugal, Taiwan, United Kingdom, United States
Collaborators--
Timeline
Phase 3CompletedFinished
20222023202420252026
First PostedApr 2021
Enrollment StartSep 2021
Primary CompletionJun 2023
TodayJul 2026
First PostedApr 21, 2021
Enrollment StartSep 8, 2021
Primary CompletionJun 13, 2023
TodayJul 2, 2026
Enrollment to primary: 1.8 yearsPosted 5.2 years ago
Interventions
OAV101genetic
Gene Therapy - 1.1e14 vector genome (vg)/kg as a one-time IV infusion was administered over approximately 60 minutes.