Duchenne Muscular DystrophyPhase 3EXPEDITIONNCT05967351
A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study
A Phase 3 study testing delandistrogene moxeparvovec in people with Duchenne Muscular Dystrophy, run by Sarepta Therapeutics, Inc. at 38 sites in 9 countries.
Trial 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. Read more about this study
- Who is it for?
- Any age, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- delandistrogene moxeparvovec
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 400 participants
- Where
- 38 sitesin 9 countries
- Study dates
- 27 Sep 2023to 31 Oct 2033
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- Not applicable
Trial locations and contacts
38 sites in 9 countries. Contact the study team or a site near you to ask about taking part.
38 sites in 9 countries
United States22 sites
Children's Hospital of Colorado
Aurora, Colorado, 80045-7106
The Johns Hopkins Hospital, Rubenstein Child Health Bldg
Baltimore, Maryland, 21287
Boston Children's Hospital
Boston, Massachusetts, 02115
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Nationwide Children's Hospital
Columbus, Ohio, 43205
Children's Health Specialty Center
Dallas, Texas, 75207
Lenox Baker Children's Hospital (Duke University)
Durham, North Carolina, 27705
University of Florida Clinical Research Center
Gainesville, Florida, 32610
University of Iowa ICTS Clinical Research Unit
Iowa City, Iowa, 52242
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
UCLA Medical Center
Los Angeles, California, 90095
Children's Hospital Wisconsin
Milwaukee, Wisconsin, 53226
Columbia University Pediatric Neuromuscular Center
New York, New York, 10032
Children's Hospital of The King's Daughters
Norfolk, Virginia, 23510
Lucile Packard Children's Hospital Stanford (LPCH)
Palo Alto, California, 94304
Chiildren's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Oregon Health and Science University
Portland, Oregon, 97239
University of Rochester Medical Center
Rochester, New York, 14642
University of California, Davis
Sacramento, California, 95817
University of Utah Health
Salt Lake City, Utah, 84132
Rady Children's Hospital
San Diego, California, 92123
Washington University School of Medicine
St Louis, Missouri, 63110
Italy3 sites
UOC Neurologia Pediatrica e Malattie Muscolari, Istituto G. Gaslini, Istituto Pediatrico di Ricovero e Cura a Carattere Scientifico
Genova, 16147
UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, 20122
UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gemelli IRCCS, Universita Cattoclica del Sacro Cuore
Roma, 00168
Japan3 sites
Kobe University Hospital
Kobe, Hyōgo, 650-0017
National Center of Neurology and Psychiatry
Kodaira, Tokyo, 187-8551
Tokyo Women's Medical University Hospital
Tokyo, 1628666
United Kingdom3 sites
Great Ormond Street Hospital For Children NHS Foundation Trust
London, Greater London, WC1N 1EJ
Institute of Translational and Clinical Research
Newcastle upon Tyne, NE1 3BZ
Oxford University Hospitals NHS Foundation Trust
Oxford, Oxfordshire, OX3 9DU
Spain2 sites
Hospital Sant Joan de Deu
Esplugues de Llobregat, Barcelona, 08950
Hospital Universitario y Politecnico La fe. Neurology Department
Valencia, 46026
Taiwan2 sites
Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, 807
National Taiwan University Hospital
Taipei, 100225
Belgium1 site
Universitair Ziekenhuis Gent, Neuromuscular Reference Centre (NMRC)
Ghent, East Flanders, 9000
Germany1 site
Universitätsklinikum Essen - Klinik für Kinderheilkunde I
Essen, North Rhine-Westphalia, 45147
About this study
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
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.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- delandistrogene moxeparvovec
- Keywords
- Duchenne Muscular Dystrophy, DMD, Pediatric, Long-term, Follow-up, Safety, Efficacy
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
delandistrogene moxeparvovecGenetic
Also called SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS
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 group | Role | What they receive |
|---|---|---|
| Delandistrogene Moxeparvovec | Experimental | Participants received delandistrogene moxeparvovec in a previous clinical study. Receives: Genetic: delandistrogene moxeparvovec |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: Baseline, up to 10 years
Change in Musculoskeletal MRI Findings From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation
Measured: Baseline, up to 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Aug 2023
- Study start27 Sep 2023
- Primary completion31 Oct 2033 (estimated)
- Study completion31 Oct 2033 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by Sarepta Therapeutics, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 400 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Hoffmann-La Roche
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05967351
- Sponsor's protocol ID
- SRP-9001-305
- Other IDs
- 2023-505043-39-00, 2023
- Acronym
- EXPEDITION
- Status last verified
- Feb 2026
- First submitted
- 21 Jul 2023
- Last updated
- 6 Feb 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Mendell JR, Muntoni F, McDonald CM, Mercuri EM, Deconinck N, Ciafaloni E, Komaki H, Leon-Astudillo C, Nascimento A, Proud C, Schara-Schmidt U, Veerapandiyan A, Zaidman CM, Furgerson M, Asher DR, Vivien M, Sun Y, Murphy AP, Pordeli P, Torre CO, Manfrini M, Gerber M, Rodino-Klapac LR; EMBARK Study Group. Three-Year Follow-Up of Delandistrogene Moxeparvovec in Duchenne Muscular Dystrophy: Long-Term Phase 3 EMBARK Trial Results. Neurol Ther. 2026 Oct 27. doi: 10.1007/s40120-026-01059-y. Online ahead of print.
PubMed 42944976
Study at a glance
From the registry record, last updated by the sponsor on 6 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05967351
- Acronym / sponsor ID
- EXPEDITION / SRP-9001-305
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne Muscular Dystrophy, DMD, Pediatric, Long-term, Follow-up, Safety, Efficacy
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Enrolling by invitation
- Enrolment
- 400 participants (estimated)
- Lead sponsor
- Sarepta Therapeutics, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Hoffmann-La Roche
- Study start
- 27 Sep 2023 (actual)
- Primary completion
- 31 Oct 2033 (estimated)
- Study completion
- 31 Oct 2033 (estimated)
- First posted
- 1 Aug 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Delandistrogene Moxeparvovec | Experimental | Genetic: delandistrogene moxeparvovecParticipants received delandistrogene moxeparvovec in a previous clinical study. |
Who may qualify
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- 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.
Key exclusion criteria (1 listed)
- Not applicable
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| delandistrogene moxeparvovecAlso: SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYSNo 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. | Genetic | Delandistrogene Moxeparvovec |
Primary outcome
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
9 secondary outcomes
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
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
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
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
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
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
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
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
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Feb 2026.
- Derived from this study: PubMed 42944976Mendell JR, Muntoni F, McDonald CM, Mercuri EM, Deconinck N, Ciafaloni E, Komaki H, Leon-Astudillo C, Nascimento A, Proud C, Schara-Schmidt U, Veerapandiyan A, Zaidman CM, Furgerson M, Asher DR, Vivien M, Sun Y, Murphy AP, Pordeli P, Torre CO, Manfrini M, Gerber M, Rodino-Klapac LR; EMBARK Study Group. Three-Year Follow-Up of Delandistrogene Moxeparvovec in Duchenne Muscular Dystrophy: Long-Term Phase 3 EMBARK Trial Results. Neurol Ther. 2026 Oct 27. doi: 10.1007/s40120-026-01059-y. Online ahead of print.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
The record lists no contact. Use the official record to reach the sponsor.
38 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing delandistrogene moxeparvovec in people with Duchenne Muscular Dystrophy, run by Sarepta Therapeutics, Inc. at 38 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: enrolling by invitation, as of the sponsor's last update on 6 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 38 sites in United States, Italy, Japan, United Kingdom, Spain and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05967351, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Feb 2026.