At a glance
ClinicalIndex Comparison RecordPhase 4Completed· 16 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 IV Open-label, Single-arm, Single-dose, Multicenter Study to Evaluate the saFEty, toLerability and effIcacy of Gene Replacement Therapy With intravenousOAV101(AVXS101) in Pediatric Patients From Latin America With Spinal Muscular Atrophy (SMA) - OFELIA
In Brief
A Phase 4 clinical trial evaluating OAV101 for Muscular Atrophy, Spinal. Completed, enrolled 16 participants across 5 sites in 2 countries.
Detailed Summary
This was a phase IV Open-label, single-arm, single-dose, multicenter study, to evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with SMA with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene ≤ 24 months and weighing ≤ 17 kg, over a 18-month period post infusion.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsMuscular Atrophy, Spinal
CountriesArgentina, Brazil
Collaborators--
Timeline
Phase 4CompletedFinished
20222023202420252026
First PostedOct 2021
Enrollment StartNov 2021
Primary CompletionAug 2023
TodayJul 2026
First PostedOct 11, 2021
Enrollment StartNov 4, 2021
Primary CompletionAug 8, 2023
TodayJul 2, 2026
Enrollment to primary: 1.8 yearsPosted 4.7 years ago
Interventions
OAV101genetic
A single Gene Therapy IV infusion at 1.1e14 vector genome (vg)/kg over approximately 60 minutes