NCT04626674 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
| NCT ID | NCT04626674 |
| Status | Recruiting |
| Phase | Phase 1 |
| Sponsor | Sarepta Therapeutics, Inc. |
| Condition | Duchenne Muscular Dystrophy |
| Study Type | INTERVENTIONAL |
| Enrollment | 83 participants |
| Start Date | 2020-11-23 |
| Primary Completion | 2027-12-31 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 83 participants in total. It began in 2020-11-23 with a primary completion date of 2027-12-31.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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). The maximum participant duration for this study is 156 weeks.
Eligibility 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.
Contact & Investigator
Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4
✉ SareptAlly@Sarepta.com📞 1-888-SAREPTA (1-888-727-3782)
Medical Director
STUDY DIRECTOR
Sarepta Therapeutics, Inc.
Frequently Asked Questions
Who can join the NCT04626674 clinical trial?
This trial is open to male participants only, aged 2 Years or older, studying Duchenne Muscular Dystrophy. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT04626674 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT04626674 currently recruiting?
Yes, NCT04626674 is actively recruiting participants. Contact the research team at SareptAlly@Sarepta.com for enrollment information.
Where is the NCT04626674 trial being conducted?
This trial is being conducted at Little Rock, United States, La Jolla, United States, Los Angeles, United States, Palo Alto, United States and 8 additional locations.
Who is sponsoring the NCT04626674 clinical trial?
NCT04626674 is sponsored by Sarepta Therapeutics, Inc.. The principal investigator is Medical Director at Sarepta Therapeutics, Inc.. The trial plans to enroll 83 participants.