Clinical Trial

Long-Term Follow-up Study for Patients From AVXS-101-CL-101

Study acronym: START
Active, Not Recruiting
View on ClinicalTrials.gov →
Summary
This is a long term, safety follow up study of patients in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1 delivering onasemnogene abeparvovec-xioi. Patients will roll over from the parent study into this long-term study for continuous safety monitoring for up to 15 years.
Protocol Amendment History 15 amendments
This ClinicalTrials.gov record has been amended 15 times since 2018-02-02; most recent amendment 2025-04-23.
Status change: Enrolling by Invitation → Active, Not Recruiting 2020-06-10
Trial Details
NCT Number NCT03421977
Lead Sponsor Novartis Gene Therapies
Conditions Spinal Muscular Atrophy 1
Enrollment 13 participants
Start Date 2017-09-21
Primary Completion 2030-12-02 (estimated)
Study Completion 2030-12-02 (estimated)
Updated on ClinicalTrials.gov 2025-04-25