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Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
Not Recruiting
Trial ID: NCT04042025
Purpose
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.
Official Title
A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101
Stanford Investigator(s)
John W. Day, MD, PhD
Professor of Neurology (Adult Neurology), of Pediatrics (Genetics) and, by courtesy, of Pathology
Eligibility
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(s):
biological: Onasemnogene Abeparvovec-xioi
Not Recruiting
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
John Day
650-725-1442