NCT03421977Active, not recruiting
Long-Term Follow-up Study for Patients From AVXS-101-CL-101
A Long Term Follow up Safety Study of Patients in the AVXS-101-CL-101 Gene Replacement Therapy Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS 101
Source: ClinicalTrials.gov · Last updated 22 Sept 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Active, not recruiting
- Study type
- Observational
- Enrollment
- 13 (actual)
- First submitted
- 30 Jan 2018
- Start date
- 21 Sept 2017
- Primary completion date
- 2 Dec 2030
- Lead sponsor
- Novartis Gene Therapies
- Diseases
- Spinal muscular atrophy
- NCT number
- NCT03421977
What you commit to
- Study type: Observational
- Participants enrolled: 13
- From 21 Sept 2017 to the primary completion date, 2 Dec 2030
- 1 site in 1 country
- 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.
Other
- 1\. Parent/legal guardian unable or unwilling to participate in the long term follow up safety study.
Who the study is looking for
- 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.
Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Nationwide Children's Hospital Coordinator of this site? Confirm its status | Columbus | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.