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.
Conditions
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III, SMA
Recruitment Status
Active, not recruiting
Eligibility Criteria
Inclusion Criteria:
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
Exclusion Criteria:
Parent/legal guardian unable or unwilling to participate in the long-term follow-up safety study
Intervention
Intervention Type
Intervention Name
Biological
Onasemnogene Abeparvovec-xioi
Phase
Phase 3
Gender
All
Min Age
N/A
Max Age
N/A
Download Date
February 22, 2024
Principal Investigator
N/A
Primary Contact Information
For more information on this trial, visit clinicaltrials.gov.
Contact
For more information and to contact the study team: