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
Sarepta Therapeutics, Inc. · with Hoffmann-La Roche · NCT04626674
Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.
Key facts
- Compensation
- Not posted. Ask the study team before screening.
- Who can join
- Ages 2+ · Men only
- Healthy volunteers
- Not accepted (a condition is required)
- Study type
- Interventional · Phase 1
- Target enrollment
- 83
- US sites recruiting
- 11
- Study start
- November 23, 2020
- Record last updated
- September 11, 2026
Status re-checked against ClinicalTrials.gov within the last 12 hours.
What the study is about
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.
Being studied: delandistrogene moxeparvovec
Conditions: Duchenne Muscular Dystrophy
Eligibility criteria
Copied from the official record. The study team makes the final decision at screening.
Show full criteria
Inclusion Criteria
- 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.
- 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.
Exclusion Criteria
- 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.
Recruiting US locations
Arkansas Children's Hospital
Little Rock, AR 72202
Site contact: 501-364-1868 · KurakuVV@archildrens.org
University of California, San Diego
La Jolla, CA 92037
Site contact: Gabriella Penner · gpenner@health.ucsd.edu
University of California, Los Angeles
Los Angeles, CA 90095
Site contact: SareptAlly@Sarepta.com
Stanford University
Palo Alto, CA 94304
Site contact: neuromuscularresearch@stanford.edu
University of California, Davis
Sacramento, CA 95616
Site contact: Craig McDonald, MD
University of Florida
Gainesville, FL 32610
Site contact: ssalabarria@ufl.edu
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, IL 60611
Site contact: SareptAlly@Sarepta.com
Washington University in St. Louis
St Louis, MO 63110
Site contact: 314-362-6981 · NeuromusclePediatricResearch@wustl.edu
Duke University Medical Center
Durham, NC 27705
Site contact: SareptAlly@Sarepta.com
Neurology Rare Disease Center
Flower Mound, TX 75028
Site contact: 972-999-1011 · Research@neuromdcenter.com
Children's Hospital of The King's Daughters
Norfolk, VA 23507
Site contact: Proud.Research@chkd.org
Study contacts (from the official record)
- Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4 · 1-888-SAREPTA (1-888-727-3782) · SareptAlly@Sarepta.com
Similar studies in Arkansas
All phase 1 studies in Arkansas →- RecruitingHealthy volunteersPhase 1
Study to Evaluate the Safety, Pharmacokinetic, and Pharmacodynamic Effects of MY006 in Healthy Volunteers and Patients With Peanut Allergy
Mabylon AG
- Location
- Little Rock, AR + 7 other US sites
- Eligibility basics
- Ages 12–55
- RecruitingHealthy volunteersPhase 1
First-in-Human Trial to Assess REGN20423 in Healthy Adult Participants and Adult Participants With Atopic Dermatitis
Regeneron Pharmaceuticals
- Location
- North Little Rock, AR + 5 other US sites
- Eligibility basics
- Ages 18–65
- RecruitingHealthy volunteersPhase 1
A 3-part Study of SYX-5219 in Healthy Volunteers and Participants With Atopic Dermatitis
Sitryx Therapeutics Ltd
- Location
- Arkansas City, AR + 5 other US sites
- Eligibility basics
- Ages 18–65
- RecruitingPhase 1Overnight stay mentioned
A Study of LTX-001 In Adult Participants With Acute Exacerbation of Schizophrenia
Leal Therapeutics, Inc
- Location
- Little Rock, AR + 9 other US sites
- Eligibility basics
- Ages 18–55