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

February 4, 2026 updated by: Sarepta Therapeutics, Inc.

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

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.

Study Overview

Status

Enrolling by invitation

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

400

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Belgium
    • East Flanders
      • Ghent, East Flanders, Belgium, 9000
        • Universitair Ziekenhuis Gent, Neuromuscular Reference Centre (NMRC)
  • Germany
    • North Rhine-Westphalia
      • Essen, North Rhine-Westphalia, Germany, 45147
        • Universitätsklinikum Essen - Klinik für Kinderheilkunde I
  • Hong Kong
      • Hong Kong, Hong Kong
        • Hong Kong Children's Hospital
  • Italy
      • Genova, Italy, 16147
        • UOC Neurologia Pediatrica e Malattie Muscolari, Istituto G. Gaslini, Istituto Pediatrico di Ricovero e Cura a Carattere Scientifico
      • Milan, Italy, 20122
        • UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
      • Roma, Italy, 00168
        • UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gemelli IRCCS, Universita Cattoclica del Sacro Cuore
  • Japan
      • Tokyo, Japan, 1628666
        • Tokyo Women's Medical University Hospital
    • Hyōgo
      • Kobe, Hyōgo, Japan, 650-0017
        • Kobe University Hospital
    • Tokyo
      • Kodaira, Tokyo, Japan, 187-8551
        • National Center of Neurology and Psychiatry
  • Spain
      • Valencia, Spain, 46026
        • Hospital Universitario y Politecnico La fe. Neurology Department
    • Barcelona
      • Esplugues de Llobregat, Barcelona, Spain, 08950
        • Hospital Sant Joan de Déu
  • Taiwan
      • Kaohsiung City, Taiwan, 807
        • Kaohsiung Medical University Chung-Ho Memorial Hospital
      • Taipei, Taiwan, 100225
        • National Taiwan University Hospital
  • United Kingdom
      • Newcastle upon Tyne, United Kingdom, NE1 3BZ
        • Institute of Translational and Clinical Research
    • Greater London
      • London, Greater London, United Kingdom, WC1N 1EJ
        • Great Ormond Street Hospital for Children NHS Foundation Trust
    • Oxfordshire
      • Oxford, Oxfordshire, United Kingdom, OX3 9DU
        • Oxford University Hospitals NHS Foundation Trust
  • United States
    • Arkansas
      • Little Rock, Arkansas, United States, 72202
        • Arkansas Children's Hospital
    • California
      • Los Angeles, California, United States, 90095
        • UCLA Medical Center
      • Palo Alto, California, United States, 94304
        • Lucile Packard Children's Hospital Stanford (LPCH)
      • Sacramento, California, United States, 95817
        • University of California, Davis
      • San Diego, California, United States, 92123
        • Rady Children's Hospital
    • Colorado
      • Aurora, Colorado, United States, 80045-7106
        • Children's Hospital of Colorado
    • Florida
      • Gainesville, Florida, United States, 32610
        • University of Florida Clinical Research Center
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Ann & Robert H. Lurie Children's Hospital of Chicago
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa ICTS Clinical Research Unit
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • The Johns Hopkins Hospital, Rubenstein Child Health Bldg
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Boston Children's Hospital
    • Missouri
      • St Louis, Missouri, United States, 63110
        • Washington University School of Medicine
    • New York
      • New York, New York, United States, 10032
        • Columbia University Pediatric Neuromuscular Center
      • Rochester, New York, United States, 14642
        • University of Rochester Medical Center
    • North Carolina
      • Durham, North Carolina, United States, 27705
        • Lenox Baker Children's Hospital (Duke University)
    • Ohio
      • Columbus, Ohio, United States, 43205
        • Nationwide Children's Hospital
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health and Science University
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Chiildren's Hospital of Philadelphia
    • Texas
      • Dallas, Texas, United States, 75207
        • Children's Health Specialty Center
    • Utah
      • Salt Lake City, Utah, United States, 84132
        • University of Utah Health
    • Virginia
      • Norfolk, Virginia, United States, 23510
        • Children's Hospital of The King's Daughters
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Children's Hospital Wisconsin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Other
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Delandistrogene Moxeparvovec
Participants received delandistrogene moxeparvovec in a previous clinical study.
Genetic: 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.
Other Names:
  • SRP-9001
  • delandistrogene moxeparvovec-rokl
  • ELEVIDYS

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure
Time Frame
Change in the North Star Ambulatory Assessment (NSAA) Total Score From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation
Time Frame: Baseline, up to 10 years
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
Time Frame: Baseline, up to 10 years
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
Time Frame: Baseline, up to 10 years
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
Time Frame: Baseline, up to 10 years
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
Time Frame: Baseline, up to 10 years
Baseline, up to 10 years
Change in Forced Vital Capacity Percent (FVC%) Predicted From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation
Time Frame: Baseline, up to 10 years
Baseline, up to 10 years
Change in Peak Expiratory Flow Percent (PEF%) Predicted From Pre-infusion Baseline to the End of the Study Participation of Delandistrogene Moxeparvovec
Time Frame: Baseline, up to 10 years
Baseline, up to 10 years
Change in Cardiac Magnetic Resonance Imaging (MRI) Findings From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation
Time Frame: Baseline, up to 10 years
Baseline, up to 10 years
Change in Musculoskeletal MRI Findings From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation
Time Frame: Baseline, up to 10 years
Baseline, up to 10 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sarepta Therapeutics, Inc.

Collaborators

Hoffmann-La Roche

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 27, 2023

Primary Completion (Estimated)

October 31, 2033

Study Completion (Estimated)

October 31, 2033

Study Registration Dates

First Submitted

July 21, 2023

First Submitted That Met QC Criteria

July 21, 2023

First Posted (Actual)

August 1, 2023

Study Record Updates

Last Update Posted (Actual)

February 6, 2026

Last Update Submitted That Met QC Criteria

February 4, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SRP-9001-305
  • 2023-505043-39-00 (Other Identifier: Clinical Trials Information System (CTIS))

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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