Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi

Brief summary (registry, in English)

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.

What you commit to

• Study type: Interventional · Phase 3
• Participants enrolled: 85
• From 10 Feb 2020 to the primary completion date, 31 Dec 2035
• 30 sites in 9 countries
• Visits and tests: ask the trial team, the registry does not list them

Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.

Who cannot take part

Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.

Who the study is looking for

Age in the registry: - to 64 years · Sex: All · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (30)

Sites in registry order. Pick your city on the disease page to sort them by distance.