Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
Running, not enrolling · Phase 3
Conditions studied: Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III, SMA
In brief
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.
Key facts
- Study ID
- NCT04042025
- Run by
- Novartis Gene Therapies
- People needed
- 85
- Starts
- 2020-02-10
- Expected to finish
- 2035-12-31
- Last updated by the study team
- 2025-04-25
Who can join
Age: any. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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
You may not qualify if…
- Parent/legal guardian unable or unwilling to participate in the long-term follow-up safety study
Where it is running
- Stanford University Medical Center — Palo Alto, California, United States
- Children's Hospital Colorado — Aurora, Colorado, United States
- Ann Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
- John Hopkins Hospital - David M. Rubenstein Child Health Building — Baltimore, Maryland, United States
- Massachusetts General Hospital — Boston, Massachusetts, United States
- Boston Children's Hospital — Boston, Massachusetts, United States
- Spectrum Health Hospitals Helen DeVos Children's Hospital — Grand Rapids, Michigan, United States
- Washington Unviersity School of Medicine in Saint Louis — St Louis, Missouri, United States
- Columbia University Medical Center — New York, New York, United States
- Duke University — Durham, North Carolina, United States
- Nationwide Children's Hospital — Columbus, Ohio, United States
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
- Clinic for Special Children — Strasburg, Pennsylvania, United States
- Children's Health Specialty Center Dallas Campus — Dallas, Texas, United States
- University of Utah Health — Salt Lake City, Utah, United States
- Children's Hospital of The King's Daughters — Norfolk, Virginia, United States
- Virginia Commonwealth University — Richmond, Virginia, United States
- University of Wisconsin, Madison — Madison, Wisconsin, United States
- Sydney Children's Hospital — Randwick, New South Wales, Australia
- Universitair Ziekenhuis Gent — Ghent, Belgium
- Centre de Référence des Maladies Neuromusculaires — Liège, Belgium
- Children's Hospital of Eastern Ontario Research Institute — Ottawa, Ontario, Canada
- Hôpital Armand Trousseau — Paris, France
- Instituto Gianninia Gaslini — Genova, Italy
- Universita Degli Studi Di Milano — Milan, Italy
Full record on ClinicalTrials.gov
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