Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
Trial ID or NCT#
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.
A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101
- - 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
- - Parent/legal guardian unable or unwilling to participate in the long-term follow-up safety study
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