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Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials

NCT05335876 · Status: RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 175

Last updated 2026-01-20

No results posted yet for this study

Summary

This is a global, prospective, multi-center study that is designed to assess the long-term safety and efficacy of OAV101 in patients who participated in an OAV101 clinical trial. The assessments of safety and efficacy in Study COAV101A12308 will continue for 5 years after enrollment in this study.

Conditions

  • Spinal Muscular Atrophy (SMA)

Interventions

BIOLOGICAL

onasemnogene abeparvovec

Onasemnogene abeparvovec is a non-replicating recombinant adeno-associated virus serotype 9 containing the human survival motor neuron gene under the control of the ytomegalovirus enhancer/chicken β-actin-hybrid promoter. Onasemnogene abeparvovec is administered as a one-time intravenous (IV) infusion or intrathecal (IT) injection. Dosage determined by participant weight.

Sponsors & Collaborators

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
0 Years
Max Age
100 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-12-19
Primary Completion
2030-09-26
Completion
2031-02-27
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Belgium
  • Brazil
  • Canada
  • China
  • Denmark
  • France
  • Italy
  • Japan
  • Malaysia
  • Netherlands
  • Saudi Arabia
  • Singapore
  • Spain
  • Taiwan
  • Thailand
  • United Kingdom
  • Vietnam

Study Locations

More Related Trials

Entities

Drugs
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 NCT05335876 on ClinicalTrials.gov