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

Part of paid clinical trials in Little Rock, Arkansas.

Sponsor
Sarepta Therapeutics, Inc.
Study ID
NCT04626674
Phase
PHASE1
Status
Recruiting
Apply to this trial

Conditions

Eligibility Criteria

Sex
MALE
Age
2 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • delandistrogene moxeparvovec — GENETIC
    Single IV infusion of delandistrogene moxeparvovec

Study Details

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.

Key Dates

First listed
Nov 12, 2020
Start date
Nov 23, 2020
Status verified
Jun 2026
Primary completion
Dec 31, 2027
Completion
Feb 29, 2028

Study Design

Enrollment
83 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Delandistrogene Moxeparvovec
    Participants will receive a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1.

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 ]

Central Contacts

  • Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4
    1-888-SAREPTA (1-888-727-3782)
    [email protected]

Locations (7)

FacilityCityStateZIPSite coordinators
Arkansas Children's HospitalLittle RockArkansas72202
[email protected]
501-364-1868
Aravindhan Veerapandiyan, MD (PRINCIPAL_INVESTIGATOR)
Stanford UniversityPalo AltoCalifornia94304
John Day, MD (PRINCIPAL_INVESTIGATOR)
University of California, DavisSacramentoCalifornia95616
Craig McDonald, MD (PRINCIPAL_INVESTIGATOR)
Washington University in St. LouisSt LouisMissouri63110
[email protected]
314-362-6981
Craig Zaidman, MD (PRINCIPAL_INVESTIGATOR)
Nationwide Children's HospitalColumbusOhio43205-
Neurology Rare Disease CenterFlower MoundTexas75028
[email protected]
972-999-1011
Diana Castro, MD (PRINCIPAL_INVESTIGATOR)
Children's Hospital of The King's DaughtersNorfolkVirginia23507
Crystal Proud, MD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Little Rock, AR

By condition
Duchenne muscular dystrophy
By specialty
Pediatrics
By research site
Arkansas Children's Hospital· Little Rock, ARStanford University· Palo Alto, CAUniversity of California, Davis· Sacramento, CAWashington University in St. Louis· St Louis, MONationwide Children's Hospital· Columbus, OHNeurology Rare Disease Center· Flower Mound, TX

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