Immunologic investigations into transgene directed immune-mediated myositis following delandistrogene moxeparvovec gene therapy.
Rachael A Potter, Ida H Moeller, Sohrab Khan...
https://pubmed.ncbi.nlm.nih.gov/39747998Actively Recruiting
Phase 1
Age: 2Years +
MALE
ID04626674
Gene Transfer Therapy Study to Evaluate Safety and Expression of Delandistrogene Moxeparvovec in Duchenne Muscular Dystrophy Participants
Led by Sarepta Therapeutics, Inc. · Updated on 2026-04-06
83
Participants Needed
7
Research Sites
379 weeks
Total Duration
On this page
Sponsors
S
Sarepta Therapeutics, Inc.
Lead Sponsor
H
Hoffmann-La Roche
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and expression of delandistrogene moxeparvovec, a gene transfer therapy, in males with Duchenne Muscular Dystrophy (DMD). This open-label Phase 1 study includes participants across different cohorts based on ambulatory status and age. Enrollment for Cohorts 1 through 7 is complete, and Cohort 8 is currently enrolling new participants. Participants receive a single intravenous infusion of delandistrogene moxeparvovec. The study involves multiple cohorts differentiated by age and motor function abilities, including ambulatory and non-ambulatory participants. Some cohorts require stable glucocorticoid use, while others do not yet require steroid treatment. Cohort 8 participants must meet specific motor function scores and are monitored for specific safety concerns such as acute liver injury. Throughout the study, participants undergo assessments including measurement of dystrophin expression by Western blot at baseline and week 12. Safety monitoring includes tracking acute liver injury up to week 72 for Cohort 8. The total participation duration can be up to 156 weeks. The study evaluates changes in dystrophin protein levels as the primary outcome, along with safety and tolerability of the gene therapy over time.
CONDITIONS
Official Title
A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
Who Can Participate
Age: 2Years +
MALE
Eligibility Criteria
You may qualify if you...
- Definitive diagnosis of Duchenne Muscular Dystrophy based on clinical findings and prior genetic testing
- For Cohort 1: Ambulatory males aged 4 to less than 8 years at screening
- For Cohort 2: Ambulatory males aged 8 to less than 18 years at screening
- For Cohort 3: Non-ambulatory males per protocol criteria at screening
- For Cohort 4: Ambulatory males aged 3 to less than 4 years at screening
- For Cohort 5a: Ambulatory males aged 4 to less than 9 years with time to rise from floor 7 seconds or less at screening
- For Cohort 5b: Non-ambulatory males per protocol criteria at screening
- For Cohort 6: Ambulatory males aged 2 to less than 3 years at screening
- For Cohort 7: Non-ambulatory males per protocol criteria at screening
- For Cohort 8: Non-ambulatory males per protocol criteria with performance upper limb scores meeting specified values at screening
- Ability to cooperate with motor assessment testing
- Stable oral glucocorticoid dose for at least 12 weeks prior to screening for Cohorts 1, 2, 3, 5, 7, and 8, expected to remain stable for first study year
- No steroid use at screening for Cohorts 4 and 6, who do not yet require chronic steroids
- rAAVrh74 antibody titers not elevated according to protocol
- Genetic mutation inclusion criteria vary by cohort
You will not qualify if you...
- Presence of concomitant illness, autoimmune disease, chronic drug treatment, or cognitive impairment that increases risks for gene transfer
- Previous exposure to gene therapy, investigational medication, or treatments to increase dystrophin expression within protocol-specified time limits
- Abnormalities in protocol-specified diagnostic tests or laboratory evaluations
- For Cohort 8: Known hypersensitivity to sirolimus or excipients preventing oral sirolimus use
- Other inclusion/exclusion criteria as specified in the protocol
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Trial Site Locations
Total: 7 locations
1
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
Actively Recruiting
2
Stanford University
Palo Alto, California, United States, 94304
Actively Recruiting
3
University of California, Davis
Sacramento, California, United States, 95616
Actively Recruiting
4
Washington University in St. Louis
St Louis, Missouri, United States, 63110
Actively Recruiting
5
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Active, Not Recruiting
6
Neurology Rare Disease Center
Flower Mound, Texas, United States, 75028
Actively Recruiting
7
Children's Hospital of The King's Daughters
Norfolk, Virginia, United States, 23507
Actively Recruiting
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Research Team
S
Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Frequently Asked Questions
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Published Research Related To This Trial
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).
Craig M Zaidman, Crystal M Proud, Craig M McDonald...
https://pubmed.ncbi.nlm.nih.gov/37539981