Duchenne Muscular DystrophyPhase 1ENDEAVORNCT04626674
A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort
A Phase 1 study testing delandistrogene moxeparvovec in people with Duchenne Muscular Dystrophy, run by Sarepta Therapeutics, Inc. at 12 sites in United States.
Trial summary
Cohort 8 (non-ambulatory participants) is currently enrolling new participants. Enrollment for Cohorts 1 through 7 has been completed. This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with Duchenne Muscular Dystrophy (DMD). Read more about this study
- Who is it for?
- Ages 2 years and older, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- delandistrogene moxeparvovec
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 83 participants
- Where
- 12 sitesUnited States
- Study dates
- 23 Nov 2020to 29 Feb 2028
- Phase
- Phase 1
- 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 2 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- For Cohorts 1-8: Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
- Cohort 8: Non-ambulatory per protocol-specified criteria at the time of Screening, has a performance upper limb (PUL) entry item score ≥2 at the Screening visit and has a total PUL score of ≥12 and ≤40 at the time of Screening.
- Cohorts 1, 2, 3, 5, 7 and 8 only: Stable dose equivalent of oral glucocorticoids for at least 12 weeks before screening and the dose is expected to remain constant (except for modifications to accommodate changes in weight) throughout the first year of the study.
- Cohort 1: Is ambulatory, and ≥4 to <8 years of age at the time of Screening.
- Cohort 2: Is ambulatory, and ≥8 to <18 years of age at the time of Screening.
- Cohort 3: Non-ambulatory per protocol specified criteria at the time of Screening.
Show all 16Show fewer
- Cohort 4: Is ambulatory and ≥3 to <4 years of age at the time of Screening.
- Cohort 5a: Is ambulatory and ≥4 to <9 years of age with time to rise from the floor ≤7 seconds at the screening visit.
- Cohort 5b: Non-ambulatory per protocol specified criteria at the time of Screening.
- Cohort 6: Is ambulatory, and ≥2 to <3 years of age at the time of Screening.
- Cohort 7: Non-ambulatory per protocol-specified criteria at the time of Screening.
- Cohorts 4 and 6: Do not yet require use of chronic steroids for treatment of their DMD, in the opinion of the Investigator, and are not receiving steroids at the time of Screening.
- Genetic mutation inclusion criteria vary by cohort.
- All Cohorts:
- Ability to cooperate with motor assessment testing.
- rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.
Who cannot join
- Cohort 8: Any confounding factors that would prevent the use of oral sirolimus including a known hypersensitivity to sirolimus or any of its excipients.
- Has a concomitant illness, autoimmune disease, chronic drug treatment, and/or cognitive delay/impairment that in the opinion of the Investigator creates unnecessary risks for gene transfer.
- Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol-specified time limits.
- Abnormality in protocol-specified diagnostic evaluations or laboratory tests.
- Note: Other inclusion/exclusion criteria apply.
Trial locations and contacts
12 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4 contact
12 sites in 1 country
United States12 sites, 11 recruiting
- Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
SareptAlly@Sarepta.com
Bridget McGowan, MD
- Active, not recruiting
Nationwide Children's Hospital
Columbus, Ohio, 43205
- Recruiting
Duke University Medical Center
Durham, North Carolina, 27705
SareptAlly@Sarepta.com
Natalie Katz, MD
- Recruiting
Neurology Rare Disease Center
Flower Mound, Texas, 75028
972-999-1011, Research@neuromdcenter.com
Diana Castro, MD
- Recruiting
University of Florida
Gainesville, Florida, 32610
ssalabarria@ufl.edu
Carmen Leon-Astudillo, MD
- Recruiting
University of California, San Diego
La Jolla, California, 92037
Gabriella Penner, gpenner@health.ucsd.edu
Andrew Skalsky, MD
- Recruiting
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
501-364-1868, KurakuVV@archildrens.org
Aravindhan Veerapandiyan, MD
- Recruiting
University of California, Los Angeles
Los Angeles, California, 90095
SareptAlly@Sarepta.com
- Recruiting
Children's Hospital of The King's Daughters
Norfolk, Virginia, 23507
Proud.Research@chkd.org
Crystal Proud, MD
- Recruiting
Stanford University
Palo Alto, California, 94304
neuromuscularresearch@stanford.edu
John Day, MD
- Recruiting
University of California, Davis
Sacramento, California, 95616
Craig McDonald, MD
- Recruiting
Washington University in St. Louis
St Louis, Missouri, 63110
314-362-6981, NeuromusclePediatricResearch@wustl.edu
Craig Zaidman, MD
About this study
Official title: An Open-Label, Systemic Gene Delivery Study Using Commercial Process Material to Evaluate the Safety of and Expression From SRP-9001 in Subjects With Duchenne Muscular Dystrophy (ENDEAVOR)
Cohort 8 (non-ambulatory participants) is currently enrolling new participants. Enrollment for Cohorts 1 through 7 has been completed.
This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with Duchenne Muscular Dystrophy (DMD). The maximum participant duration for this study is 156 weeks.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- delandistrogene moxeparvovec
- Keywords
- Duchenne Muscular Dystrophy, Gene-Delivery, Gene Therapy, DMD, Ambulatory Non-ambulatory, Pediatric, Adult, Dystrophin
- 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
Single IV infusion of delandistrogene moxeparvovec
| Study group | Role | What they receive |
|---|---|---|
| Delandistrogene Moxeparvovec | Experimental | Participants will receive a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1. Receives: Genetic: delandistrogene moxeparvovec |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part 1 (Cohorts 1 to 5): Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12, as Measured by Western Blot
Measured: Baseline, Week 12
Part 1 (Cohorts 6 to 8): Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot
Measured: Week 12
Cohort 8: Number of Participants with Acute Liver Injury (ALI)
Measured: Baseline up to Week 72
Secondary outcomes (11)
Vector Shedding, Measured in Urine, Saliva, and Stool Samples Post-Infusion
Measured: Day 1 up to Week 104
Level of Antibody Titers to Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74)
Measured: Day 2 up to Week 156
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Adverse Events of Special Interest (AESIs)
Measured: Baseline up to Week 156
Cohort 8: Number of Participants With Infections, Edema, Wound-healing Complications, Hyperlipidemia, Angioedema, and Intestinal Lung Disease/ Non-infectious Pneumonitis
Measured: Baseline up to Week 72
Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12, as Measured by Immunofluorescence (IF) Fiber Intensity
Measured: Baseline, Week 12
Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12, as Measured by IF Percent Dystrophin Positive Fibers (PDPF)
Measured: Baseline, Week 12
Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by IF Fiber Intensity
Measured: Week 12
Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by IF PDPF
Measured: Week 12
Cohort 8: Number of Participants With Hepatic AEs, Hepatic Biomarkers, and Laboratory Assessments Indicative of Either Acute Hepatocellular Injury or Acute Liver Dysfunction
Measured: Baseline up to Week 72
Cohort 8: Number of Participants with Severe ALI
Measured: Baseline up to Week 72
Cohort 8: Duration of Steroids Administered
Measured: Baseline up to Week 72
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Nov 2020
- Study start23 Nov 2020
- Primary completion31 Dec 2027 (estimated)
- Study completion29 Feb 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 83 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Hoffmann-La Roche
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Sarepta Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04626674
- Sponsor's protocol ID
- SRP-9001-103
- Acronym
- ENDEAVOR
- Status last verified
- Sep 2026
- First submitted
- 6 Nov 2020
- Last updated
- 11 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Potter RA, Moeller IH, Khan S, Haegel H, Hollenstein A, Steiner G, Wandel C, Murphy AP, Asher DR, Palatinsky E, Griffin DA, Mason S, Iannaccone ST, Zaidman CM, Rodino-Klapac LR. Immunologic investigations into transgene directed immune-mediated myositis following delandistrogene moxeparvovec gene therapy. Sci Rep. 2025 Jan 2;15(1):4. doi: 10.1038/s41598-024-84077-w.
PubMed 39747998Zaidman CM, Proud CM, McDonald CM, Lehman KJ, Goedeker NL, Mason S, Murphy AP, Guridi M, Wang S, Reid C, Darton E, Wandel C, Lewis S, Malhotra J, Griffin DA, Potter RA, Rodino-Klapac LR, Mendell JR. Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged >/=4 to <8 Years) with Duchenne Muscular Dystrophy: 1-Year Interim Results from Study SRP-9001-103 (ENDEAVOR). Ann Neurol. 2023 Nov;94(5):955-968. doi: 10.1002/ana.26755. Epub 2023 Sep 7.
PubMed 37539981
Study at a glance
From the registry record, last updated by the sponsor on 11 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Systemic Gene Delivery Study Using Commercial Process Material to Evaluate the Safety of and Expression From SRP-9001 in Subjects With Duchenne Muscular Dystrophy (ENDEAVOR)
- ClinicalTrials.gov ID
- NCT04626674
- Acronym / sponsor ID
- ENDEAVOR / SRP-9001-103
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne Muscular Dystrophy, Gene-Delivery, Gene Therapy, DMD, Ambulatory Non-ambulatory, Pediatric, Adult, Dystrophin
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 83 participants (estimated)
- Lead sponsor
- Sarepta Therapeutics, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Hoffmann-La Roche
- Study start
- 23 Nov 2020 (actual)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 29 Feb 2028 (estimated)
- First posted
- 12 Nov 2020 (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.
Cohort 8 (non-ambulatory participants) is currently enrolling new participants. Enrollment for Cohorts 1 through 7 has been completed.
This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with Duchenne Muscular Dystrophy (DMD). The maximum participant duration for this study is 156 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Delandistrogene Moxeparvovec | Experimental | Genetic: delandistrogene moxeparvovecParticipants will receive a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1. |
Who may qualify
- Ages
- 2 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (16 listed)
- For Cohorts 1-8: Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
- Cohort 8: Non-ambulatory per protocol-specified criteria at the time of Screening, has a performance upper limb (PUL) entry item score ≥2 at the Screening visit and has a total PUL score of ≥12 and ≤40 at the time of Screening.
- Cohorts 1, 2, 3, 5, 7 and 8 only: Stable dose equivalent of oral glucocorticoids for at least 12 weeks before screening and the dose is expected to remain constant (except for modifications to accommodate changes in weight) throughout the first year of the study.
- Cohort 1: Is ambulatory, and ≥4 to <8 years of age at the time of Screening.
- Cohort 2: Is ambulatory, and ≥8 to <18 years of age at the time of Screening.
Key exclusion criteria (5 listed)
- Cohort 8: Any confounding factors that would prevent the use of oral sirolimus including a known hypersensitivity to sirolimus or any of its excipients.
- Has a concomitant illness, autoimmune disease, chronic drug treatment, and/or cognitive delay/impairment that in the opinion of the Investigator creates unnecessary risks for gene transfer.
- Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol-specified time limits.
- Abnormality in protocol-specified diagnostic evaluations or laboratory tests.
- Note: Other inclusion/exclusion criteria apply.
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, ELEVIDYSSingle IV infusion of delandistrogene moxeparvovec | Genetic | Delandistrogene Moxeparvovec |
Primary outcome
Part 1 (Cohorts 1 to 5): Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12, as Measured by Western Blot
Time frame: Baseline, Week 12
Part 1 (Cohorts 6 to 8): Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot
Time frame: Week 12
Cohort 8: Number of Participants with Acute Liver Injury (ALI)
Time frame: Baseline up to Week 72
11 secondary outcomes
Vector Shedding, Measured in Urine, Saliva, and Stool Samples Post-Infusion
Time frame: Day 1 up to Week 104
Level of Antibody Titers to Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74)
Time frame: Day 2 up to Week 156
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Adverse Events of Special Interest (AESIs)
Time frame: Baseline up to Week 156
Cohort 8: Number of Participants With Infections, Edema, Wound-healing Complications, Hyperlipidemia, Angioedema, and Intestinal Lung Disease/ Non-infectious Pneumonitis
Time frame: Baseline up to Week 72
Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12, as Measured by Immunofluorescence (IF) Fiber Intensity
Time frame: Baseline, Week 12
Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12, as Measured by IF Percent Dystrophin Positive Fibers (PDPF)
Time frame: Baseline, Week 12
Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by IF Fiber Intensity
Time frame: Week 12
Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by IF PDPF
Time frame: Week 12
Cohort 8: Number of Participants With Hepatic AEs, Hepatic Biomarkers, and Laboratory Assessments Indicative of Either Acute Hepatocellular Injury or Acute Liver Dysfunction
Time frame: Baseline up to Week 72
Cohort 8: Number of Participants with Severe ALI
Time frame: Baseline up to Week 72
Cohort 8: Duration of Steroids Administered
Time frame: Baseline up to Week 72
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 11 Sep 2026.
- Derived from this study: PubMed 39747998Potter RA, Moeller IH, Khan S, Haegel H, Hollenstein A, Steiner G, Wandel C, Murphy AP, Asher DR, Palatinsky E, Griffin DA, Mason S, Iannaccone ST, Zaidman CM, Rodino-Klapac LR. Immunologic investigations into transgene directed immune-mediated myositis following delandistrogene moxeparvovec gene therapy. Sci Rep. 2025 Jan 2;15(1):4. doi: 10.1038/s41598-024-84077-w.
- Derived from this study: PubMed 37539981Zaidman CM, Proud CM, McDonald CM, Lehman KJ, Goedeker NL, Mason S, Murphy AP, Guridi M, Wang S, Reid C, Darton E, Wandel C, Lewis S, Malhotra J, Griffin DA, Potter RA, Rodino-Klapac LR, Mendell JR. Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged >/=4 to <8 Years) with Duchenne Muscular Dystrophy: 1-Year Interim Results from Study SRP-9001-103 (ENDEAVOR). Ann Neurol. 2023 Nov;94(5):955-968. doi: 10.1002/ana.26755. Epub 2023 Sep 7.
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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.
Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4 contact
Medical Director study director
Sarepta Therapeutics, Inc.
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing delandistrogene moxeparvovec in people with Duchenne Muscular Dystrophy, run by Sarepta Therapeutics, Inc. at 12 sites in United States.
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 16 inclusion and 5 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: looking for participants now, as of the sponsor's last update on 11 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Contact Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4 (1-888-SAREPTA (1-888-727-3782), SareptAlly@Sarepta.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Part 1 (Cohorts 1 to 5): Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12, as Measured by Western Blot (time frame: Baseline, Week 12). The study lists 3 primary outcomes in all. 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 NCT04626674, which also lists 2 linked publications.
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 11 Sep 2026.