A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study
EXPEDITION
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
2 other identifiers
interventional
400
9 countries
38
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_3
Started Sep 2023
Longer than P75 for phase_3
38 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
July 21, 2023
CompletedFirst Posted
Study publicly available on registry
August 1, 2023
CompletedStudy Start
First participant enrolled
September 27, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 31, 2033
ExpectedStudy Completion
Last participant's last visit for all outcomes
October 31, 2033
February 6, 2026
February 1, 2026
10.1 years
July 21, 2023
February 4, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
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
Secondary Outcomes (9)
Change in the North Star Ambulatory Assessment (NSAA) Total Score From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation
Baseline, up to 10 years
Change in Time to Rise From Floor From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation
Baseline, up to 10 years
Change in the Time of 10-meter Walk/Run (10MWR) From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation
Baseline, up to 10 years
Change in Performance of Upper Limb (PUL) (Version 2.0) Total Scores From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation
Baseline, up to 10 years
Change in PUL (Version 2.0) Domain Specific Scores From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation
Baseline, up to 10 years
- +4 more secondary outcomes
Study Arms (1)
Delandistrogene Moxeparvovec
EXPERIMENTALParticipants received delandistrogene moxeparvovec in a previous clinical study.
Interventions
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.
Eligibility Criteria
You 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.
You may not qualify if:
- Not applicable
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Sarepta Therapeutics, Inc.lead
- Hoffmann-La Rochecollaborator
Study Sites (38)
Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
UCLA Medical Center
Los Angeles, California, 90095, United States
Lucile Packard Children's Hospital Stanford (LPCH)
Palo Alto, California, 94304, United States
University of California, Davis
Sacramento, California, 95817, United States
Rady Children's Hospital
San Diego, California, 92123, United States
Children's Hospital of Colorado
Aurora, Colorado, 80045-7106, United States
University of Florida Clinical Research Center
Gainesville, Florida, 32610, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
University of Iowa ICTS Clinical Research Unit
Iowa City, Iowa, 52242, United States
The Johns Hopkins Hospital, Rubenstein Child Health Bldg
Baltimore, Maryland, 21287, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
Washington University School of Medicine
St Louis, Missouri, 63110, United States
Columbia University Pediatric Neuromuscular Center
New York, New York, 10032, United States
University of Rochester Medical Center
Rochester, New York, 14642, United States
Lenox Baker Children's Hospital (Duke University)
Durham, North Carolina, 27705, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Oregon Health and Science University
Portland, Oregon, 97239, United States
Chiildren's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Children's Health Specialty Center
Dallas, Texas, 75207, United States
University of Utah Health
Salt Lake City, Utah, 84132, United States
Children's Hospital of The King's Daughters
Norfolk, Virginia, 23510, United States
Children's Hospital Wisconsin
Milwaukee, Wisconsin, 53226, United States
Universitair Ziekenhuis Gent, Neuromuscular Reference Centre (NMRC)
Ghent, East Flanders, 9000, Belgium
Universitätsklinikum Essen - Klinik für Kinderheilkunde I
Essen, North Rhine-Westphalia, 45147, 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, 16147, Italy
UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, 20122, Italy
UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gemelli IRCCS, Universita Cattoclica del Sacro Cuore
Roma, 00168, Italy
Kobe University Hospital
Kobe, Hyōgo, 650-0017, Japan
National Center of Neurology and Psychiatry
Kodaira, Tokyo, 187-8551, Japan
Tokyo Women's Medical University Hospital
Tokyo, 1628666, Japan
Hospital Sant Joan de Deu
Esplugues de Llobregat, Barcelona, 08950, Spain
Hospital Universitario y Politecnico La fe. Neurology Department
Valencia, 46026, Spain
Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, 807, Taiwan
National Taiwan University Hospital
Taipei, 100225, Taiwan
Great Ormond Street Hospital For Children NHS Foundation Trust
London, Greater London, WC1N 1EJ, United Kingdom
Oxford University Hospitals NHS Foundation Trust
Oxford, Oxfordshire, OX3 9DU, United Kingdom
Institute of Translational and Clinical Research
Newcastle upon Tyne, NE1 3BZ, United Kingdom
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NA
- Masking
- NONE
- Purpose
- OTHER
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 21, 2023
First Posted
August 1, 2023
Study Start
September 27, 2023
Primary Completion (Estimated)
October 31, 2033
Study Completion (Estimated)
October 31, 2033
Last Updated
February 6, 2026
Record last verified: 2026-02