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

Trial ID or NCT#



not recruiting iconNOT RECRUITING


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

Eligibility Criteria

Sexes Eligible for Study: All
Accepts Healthy Volunteers: No
Inclusion Criteria:
  1. - 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:
  1. - Parent/legal guardian unable or unwilling to participate in the long-term follow-up safety study


John W. Day, MD, PhD
John W. Day, MD, PhD
Neuromuscular neurologist, Neurophysiologist
Professor of Neurology (Adult Neurology), of Pediatrics (Genetics) and, by courtesy, of Pathology

Contact us to find out if this trial is right for you.


John Day