At a glance
ClinicalIndex Comparison RecordPhase 3Completed· 27 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.
Phase IIIb, Open-label, Single-arm, Multi-center Study to Evaluate the Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally (1.2 x 10^14 Vector Genomes) to Participants 2 to < 18 Years of Age With Spinal Muscular Atrophy (SMA) Who Have Discontinued Treatment With Nusinersen (Spinraza®) or Risdiplam (Evrysdi®)
In Brief
A Phase 3 clinical trial evaluating OAV101 for Spinal Muscular Atrophy. Completed, enrolled 27 participants across 13 sites in 9 countries.
Detailed Summary
This was a Phase IIIb open-label, single arm, multi-center study to evaluate the safety, tolerability and efficacy of OAV101B in participants with SMA aged 2 to \<18 years after the discontinuation of treatment with nusinersen or risdiplam. The study aimed to enroll approximately 28 participants across each of 2 age brackets (2 to \<6 years, and 6 to \<18 years).
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsSpinal Muscular Atrophy
CountriesAustralia, Belgium, Canada, France, Italy, Japan, Netherlands, Spain, United States
Collaborators--
Timeline
Phase 3CompletedFinished
2023202420252026
First PostedMay 2022
Enrollment StartJan 2023
Primary CompletionNov 2024
TodayJul 2026
First PostedMay 23, 2022
Enrollment StartJan 12, 2023
Primary CompletionNov 29, 2024
TodayJul 2, 2026
Enrollment to primary: 1.9 yearsPosted 4.1 years ago
Interventions
OAV101genetic
Intrathecal administration of OAV101 at a dose of 1.2 x 10\^14 vector genomes, one time dose