Trial Search Results

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

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.

Stanford is currently not accepting patients for this trial.

Lead Sponsor:

Novartis Gene Therapies

Stanford Investigator(s):


  • Biological: Onasemnogene Abeparvovec-xioi


Phase 3


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

Ages Eligible for Study

N/A - N/A

Genders Eligible for Study


Not currently accepting new patients for this trial

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
John Day
Not Recruiting