MedTrace Logo

Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)

NCT05089656 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 126

Last updated 2026-01-13

Study results available
· View outcomes & findings →

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.

Conditions

  • Type 2 Spinal Muscular Atrophy

Interventions

GENETIC

OAV101

Gene therapy

PROCEDURE

Sham control

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.

Sponsors & Collaborators

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
CROSSOVER

Eligibility

Min Age
2 Years
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-02-01
Primary Completion
2024-11-12
Completion
2025-04-29
FDA Drug
Yes

Countries

  • United States
  • Brazil
  • China
  • Denmark
  • India
  • Malaysia
  • Mexico
  • Saudi Arabia
  • Singapore
  • South Africa
  • Taiwan
  • Thailand
  • Vietnam

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05089656 on ClinicalTrials.gov