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.
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III, SMA
Active, not recruiting
Any participant with SMA who received onasemnogene abeparvovec-xioi gene replacement therapy in a Novartis Gene Therapies-sponsored clinical study
Participant/parent/legal guardian willing and able to complete the informed consent process and comply with study procedures and visit schedule
Parent/legal guardian unable or unwilling to participate in the long-term follow-up safety study
July 11, 2023
Primary Contact Information
For more information on this trial, visit clinicaltrials.gov.
For more information and to contact the study team: