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

Not Recruiting

Trial ID: NCT04042025


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
John W. Day, MD, PhD

Professor of Neurology (Adult Neurology), of Pediatrics (Genetics) and, by courtesy, of Pathology


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


biological: Onasemnogene Abeparvovec-xioi

Not Recruiting

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
John Day