Status:
ACTIVE_NOT_RECRUITING
Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
Lead Sponsor:
Novartis Gene Therapies
Conditions:
Spinal Muscular Atrophy Type I
Spinal Muscular Atrophy Type II
Eligibility:
All Genders
Phase:
PHASE3
Brief Summary
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...
Eligibility Criteria
Inclusion
- 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
Exclusion
- Parent/legal guardian unable or unwilling to participate in the long-term follow-up safety study
Key Trial Info
Start Date :
February 10 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 31 2035
Estimated Enrollment :
85 Patients enrolled
Trial Details
Trial ID
NCT04042025
Start Date
February 10 2020
End Date
December 31 2035
Last Update
April 25 2025
Active Locations (31)
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1
Stanford University Medical Center
Palo Alto, California, United States, 94304
2
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
3
Ann Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
4
John Hopkins Hospital - David M. Rubenstein Child Health Building
Baltimore, Maryland, United States, 21287