A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study

NCT05967351 · tracked via the Priya Life Science USA tracker

Sponsor

Sarepta Therapeutics, Inc.

Phase

Phase 3

Started

2023-09-27

Last updated

2026-02-06

Condition(s) studied

Duchenne Muscular Dystrophy

Investigational drug(s) / intervention(s)

delandistrogene moxeparvovec

delandistrogene moxeparvovec: 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 summary

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.

Eligibility

Sex

MALE

Min age

—

Max age

—

Healthy volunteers

Inclusion Criteria: * 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. Exclusion Criteria: * Not applicable

Primary outcome measure(s)

Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI) — Up to 10 years

Trial sites (38)

Facility	City	Region
Arkansas Children's Hospital	Little Rock	Arkansas
UCLA Medical Center	Los Angeles	California
Lucile Packard Children's Hospital Stanford (LPCH)	Palo Alto	California
University of California, Davis	Sacramento	California
Rady Children's Hospital	San Diego	California
Children's Hospital of Colorado	Aurora	Colorado
University of Florida Clinical Research Center	Gainesville	Florida
Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois
University of Iowa ICTS Clinical Research Unit	Iowa City	Iowa
The Johns Hopkins Hospital, Rubenstein Child Health Bldg	Baltimore	Maryland
Boston Children's Hospital	Boston	Massachusetts
Washington University School of Medicine	St Louis	Missouri
Columbia University Pediatric Neuromuscular Center	New York	New York
University of Rochester Medical Center	Rochester	New York
Lenox Baker Children's Hospital (Duke University)	Durham	North Carolina
Nationwide Children's Hospital	Columbus	Ohio
Oregon Health and Science University	Portland	Oregon
Chiildren's Hospital of Philadelphia	Philadelphia	Pennsylvania
Children's Health Specialty Center	Dallas	Texas
University of Utah Health	Salt Lake City	Utah
Children's Hospital of The King's Daughters	Norfolk	Virginia
Children's Hospital Wisconsin	Milwaukee	Wisconsin
Universitair Ziekenhuis Gent, Neuromuscular Reference Centre (NMRC)	Ghent	East Flanders
Universitätsklinikum Essen - Klinik für Kinderheilkunde I	Essen	North Rhine-Westphalia
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
National Center of Neurology and Psychiatry	Kodaira	Tokyo
Tokyo Women's Medical University Hospital	Tokyo	Japan
Hospital Sant Joan de Deu	Esplugues de Llobregat	Barcelona
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
Oxford University Hospitals NHS Foundation Trust	Oxford	Oxfordshire
Institute of Translational and Clinical Research	Newcastle upon Tyne	United Kingdom

More Sarepta Therapeutics, Inc. trials in the USA

A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON) Active, not recruiting

A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4 Active, not recruiting

A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency) Active, not recruiting

A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice Enrolling by invitation

A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, and LGMD2A/R1 ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice Active, not recruiting

Other trials for the same condition

A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX) Active, not recruiting

Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53) Recruiting

A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE) Recruiting

Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study Recruiting

Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy Active, not recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05967351 on ClinicalTrials.gov ↗ ← All trials in the USA