What this study is
The purpose of this study is to provide a single clinical study with a uniform approach to monitoring long-term safety and efficacy in participants who received delandistrogene moxeparvovec in a previous clinical study. No study drug will be administered as part of this study.
What's being tested
- delandistrogene moxeparvovec
Intervention · GENETIC
No study drug will be administered as part of this study. Eligible participants who received treatment with delandistrogene moxeparvovec during a previous clinical study will be included.
Study details
- Official title
- A Phase 3, Multinational, Long-term Follow-up Study to Evaluate Safety and Efficacy in Subjects Who Have Previously Received SRP-9001 in a Clinical Study
- Sponsor
- Sarepta Therapeutics, Inc.
- Conditions
- Duchenne Muscular Dystrophy
- Enrolling
- 400 people
- Starts
- 2023-09-27
- Est. completion
- 2033-10-31
- Design
- Open-label, single group, Phase 3
- Record
- View on ClinicalTrials.gov ↗
Who can join
Sex MALEHealthy volunteers Not acceptedYou may qualify if
- Received delandistrogene moxeparvovec for Duchenne muscular dystrophy in a previous clinical study.
- Has (a) parent(s) or legal caregiver(s) or is ≥18 years of age and able to understand and comply with the study visit schedule and all other protocol requirements.
Sign in and we'll check these against your records.Full criteria on ClinicalTrials.gov ↗
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Study locations (38)
38 openArkansas Children's Hospital
Little Rock, Arkansas, United States
UCLA Medical Center
Los Angeles, California, United States
Lucile Packard Children's Hospital Stanford (LPCH)
Palo Alto, California, United States
University of California, Davis
Sacramento, California, United States
Rady Children's Hospital
San Diego, California, United States
Children's Hospital of Colorado
Aurora, Colorado, United States
University of Florida Clinical Research Center
Gainesville, Florida, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
University of Iowa ICTS Clinical Research Unit
Iowa City, Iowa, United States
The Johns Hopkins Hospital, Rubenstein Child Health Bldg
Baltimore, Maryland, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Washington University School of Medicine
St Louis, Missouri, United States
Columbia University Pediatric Neuromuscular Center
New York, New York, United States
University of Rochester Medical Center
Rochester, New York, United States
Lenox Baker Children's Hospital (Duke University)
Durham, North Carolina, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Oregon Health and Science University
Portland, Oregon, United States
Chiildren's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Children's Health Specialty Center
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
Children's Hospital Wisconsin
Milwaukee, Wisconsin, United States
16 sites outside the US
Universitair Ziekenhuis Gent, Neuromuscular Reference Centre (NMRC)
Ghent, East Flanders, Belgium
Universitätsklinikum Essen - Klinik für Kinderheilkunde I
Essen, North Rhine-Westphalia, Germany
Hong Kong Children's Hospital
Hong Kong, Hong Kong
UOC Neurologia Pediatrica e Malattie Muscolari, Istituto G. Gaslini, Istituto Pediatrico di Ricovero e Cura a Carattere Scientifico
Genova, Italy
UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, Italy
UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gemelli IRCCS, Universita Cattoclica del Sacro Cuore
Roma, Italy
Kobe University Hospital
Kobe, Hyōgo, Japan
National Center of Neurology and Psychiatry
Kodaira, Tokyo, Japan
Tokyo Women's Medical University Hospital
Tokyo, Japan
Hospital Sant Joan de Deu
Esplugues de Llobregat, Barcelona, Spain
Hospital Universitario y Politecnico La fe. Neurology Department
Valencia, Spain
Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, Taiwan
National Taiwan University Hospital
Taipei, Taiwan
Great Ormond Street Hospital For Children NHS Foundation Trust
London, Greater London, United Kingdom
Oxford University Hospitals NHS Foundation Trust
Oxford, Oxfordshire, United Kingdom
Institute of Translational and Clinical Research
Newcastle upon Tyne, United Kingdom
Study team
Sponsor
Sarepta Therapeutics, Inc.
Study ID SRP-9001-305
Frequently asked questions
The sponsor hasn't published answers to common questions for this trial.