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Long-Term Follow-up Study for Patients From AVXS-101-CL-101
Sponsor: Novartis Gene Therapies
Summary
This is a long term, safety follow up study of patients in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1 delivering onasemnogene abeparvovec-xioi. Patients will roll over from the parent study into this long-term study for continuous safety monitoring for up to 15 years.
Official title: A Long Term Follow up Safety Study of Patients in the AVXS-101-CL-101 Gene Replacement Therapy Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS 101
Key Details
Gender
All
Age Range
Any - Any
Study Type
OBSERVATIONAL
Enrollment
13
Start Date
2017-09-21
Completion Date
2030-12-02
Last Updated
2025-04-25
Healthy Volunteers
No
Conditions
Interventions
Onasemnogene Abeparvovec-xioi
Patients received treatment with onasemnogene abeparvovec-xioi in the parent study, AVXS-101-CL-101
Locations (1)
Nationwide Children's Hospital
Columbus, Ohio, United States