Clinical Trial

Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi

Active, Not Recruiting Phase 3
View on ClinicalTrials.gov →
Summary
This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi. Participants will roll over from their respective previous (parent) study into this long-term study for continuous monitoring of safety as well as monitoring of continued efficacy and durability of response to onasemnogene abeparvovec-xioi treatment.
Protocol Amendment History 29 amendments
This ClinicalTrials.gov record has been amended 29 times since 2019-07-31; most recent amendment 2025-04-23.
Status change: Enrolling by Invitation → Active, Not Recruiting 2022-12-01
Status change: Recruiting → Enrolling by Invitation 2020-04-01
Status change: Not Yet Recruiting → Recruiting 2019-12-03
Trial Details
NCT Number NCT04042025
Lead Sponsor Novartis Gene Therapies
Conditions Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III, SMA
Enrollment 85 participants
Start Date 2020-02-10
Primary Completion 2035-12-31 (estimated)
Study Completion 2035-12-31 (estimated)
Updated on ClinicalTrials.gov 2025-04-25