This is a long-term safety follow-up study for patients who received onasemnogene abeparvovec-xioi (Zolgensma) in the AVXS-101-CL-101 gene replacement therapy trial for SMA Type 1. Patients roll over from the parent study into this long-term study for continuous safety monitoring for up to 15 years. Eligibility requires having received onasemnogene abeparvovec-xioi in AVXS-101-CL-101 and a parent or legal guardian willing and able to complete the informed consent process and comply with study procedures and the visit schedule.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: 1. Patient who received onasemnogene abeparvovec-xioi in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1. 2. Parent/legal guardian willing and able to complete the informed consent process, 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.