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Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
Sponsor: Novartis Gene Therapies
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 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.
Official title: A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101
Key Details
Gender
All
Age Range
Any - Any
Study Type
INTERVENTIONAL
Enrollment
85
Start Date
2020-02-10
Completion Date
2035-12-31
Last Updated
2025-04-25
Healthy Volunteers
No
Conditions
Interventions
Onasemnogene Abeparvovec-xioi
Onasemnogene abeparvovec-xioi is a non-replicating recombinant adeno-associated virus serotype 9 containing the human survival motor neuron gene under the control of the cytomegalovirus enhancer/chicken β-actin-hybrid promoter. Onasemnogene abeparvovec-xioi administered as a one-time intravenous (IV) infusion or intrathecal (IT) injection. Dosage determined by participant weight.
Locations (31)
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
Istituto Neurologico di Ricerca
Milan, Italy
Fondazione Policlinico Universitario Agostino Gemelli
Roma, Italy
Tokyo Women's Medical University Hospital
Tokyo, Japan
National Taiwan University Hospital
Taipei, Taiwan
Great Ormond Street Hospital for Children NHS Foundation Trust
London, United Kingdom
The Newcastle Upon Tyne Hospitals NHS Foundation Trust
Newcastle upon Tyne, United Kingdom