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
Status: Active_not_recruiting
Location: See location...
Intervention Type: Biological
Study Type: Observational
SUMMARY
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.
Eligibility
Participation Requirements
Sex: All
Healthy Volunteers: f
View:
• 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.
Locations
United States
Ohio
Nationwide Children's Hospital
Columbus
Time Frame
Start Date: 2017-09-21
Completion Date: 2030-12-02
Participants
Target number of participants: 13
Related Therapeutic Areas
Sponsors
Leads: Novartis Gene Therapies