CI

At a glance

ClinicalIndex Comparison Record
Phase 3Completed· 126 enrolled
Drug / intervention
OAV101 +1 moregenetic
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.

Search/NCT05089656
NCT05089656Phase 3Completed
CompareCT.gov

A Randomized, Sham-controlled, Double-blind Study to Evaluate the Efficacy and Safety of Intrathecal OAV101 in Type 2 Spinal Muscular Atrophy (SMA) Patients Who Are ≥ 2 to < 18 Years of Age, Treatment Naive, Sitting, and Never Ambulatory

Novartis Pharmaceuticals·interventional·Posted Oct 22, 2021·Updated Jan 13, 2026

In Brief

A Phase 3 clinical trial evaluating OAV101 and Sham control for Type 2 Spinal Muscular Atrophy. Completed, enrolled 126 participants across 42 sites in 13 countries.

Detailed Summary

This was a Phase III multi-center, single dose (1.2 x 10\^14 vector genomes), randomized, sham controlled, double-blind study that investigates the efficacy, safety and tolerability of OAV101B in treatment naive, sitting and never ambulatory SMA patients 2 to \<18 years of age.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsType 2 Spinal Muscular Atrophy
CountriesBrazil, China, Denmark, India, Malaysia, Mexico, Saudi Arabia, Singapore, South Africa, Taiwan, Thailand, United States, Vietnam
Collaborators--

Timeline

Phase 3CompletedFinished
20222023202420252026
First PostedOct 22, 2021
Enrollment StartFeb 1, 2022
Primary CompletionNov 12, 2024
Study CompletionApr 29, 2025
TodayJul 2, 2026
Enrollment to primary: 2.8 yearsPosted 4.7 years ago

Interventions

OAV101genetic

Gene therapy

Sham controlprocedure

The sham procedure will consist of a small needle prick on the lower back at the location where the LP injection is normally made. The needle will break the skin, but no needle insertion for lumbar puncture will occur.