Status:
ACTIVE_NOT_RECRUITING
Long-Term Follow-up Study for Patients From AVXS-101-CL-101
Lead Sponsor:
Novartis Gene Therapies
Conditions:
Spinal Muscular Atrophy 1
Eligibility:
All Genders
Brief 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 ...
Detailed Description
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 ...
Eligibility Criteria
Inclusion
- Patient who received onasemnogene abeparvovec-xioi in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1.
- Parent/legal guardian willing and able to complete the informed consent process, comply with study procedures and visit schedule.
Exclusion
- 1\. Parent/legal guardian unable or unwilling to participate in the long term follow up safety study.
Key Trial Info
Start Date :
September 21 2017
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
December 2 2030
Estimated Enrollment :
13 Patients enrolled
Trial Details
Trial ID
NCT03421977
Start Date
September 21 2017
End Date
December 2 2030
Last Update
April 25 2025
Active Locations (1)
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1
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205