Long-Term Follow-up Study for Patients From AVXS-101-CL-101
About the study
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.
Who can take part
You may be eligible to participate in the study if you meet the following criteria:
INCLUSION CRITERIA
Inclusion Criteria:
- 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 CRITERIA
Exclusion Criteria:
1. Parent/legal guardian unable or unwilling to participate in the long term follow up safety study.
Study Locations
Enter your ZIP code/Postal code/PIN code to locate study sites near you:
How to Apply
Contact the study center to learn if this study is a good match for you.
Study’s details
Contition
Spinal Muscular Atrophy 1
Participants needed
13
Est. Completion Date
Dec 31, 2033
Treatment type
Observational
Sponsor
Novartis
ClinicalTrials.gov identifier
NCT03421977
Study number
AVXS 101-LT-001
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