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

Status

Indicates the current recruitment status or the expanded access status.
Enrolling by invitation

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
400

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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 Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.
  • Name: Sarepta Therapeutics Inc. For Clinical Trial Information, Select Option 4
  • Phone Number: 1-888-SAREPTA (1-888-727-3782)
  • Email: SareptAlly@sarepta.com

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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Sarepta Therapeutics, Inc.

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
September 27, 2023

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
October 31, 2033

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
October 31, 2033

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
July 21, 2023

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 21, 2023

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
August 1, 2023

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
February 6, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 4, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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