Phase
Condition
N/ATreatment
delandistrogene moxeparvovec
Clinical Study ID
Ages > 2 Male
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
For Cohorts 1-8: Has a definitive diagnosis of DMD based on documented clinicalfindings and prior genetic testing.
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.
Cohort 8: Non-ambulatory per protocol-specified criteria at the time of Screening,has a performance upper limb (PUL) entry item score ≥3 at the Screening visit andhas a total PUL score of ≥20 and ≤40 at the time of Screening.
Ability to cooperate with motor assessment testing.
Cohorts 1, 2, 3, 5, 7 and 8 only: Stable dose equivalent of oral glucocorticoids forat least 12 weeks before screening and the dose is expected to remain constant (except for modifications to accommodate changes in weight) throughout the firstyear of the study.
Cohorts 4 and 6: Do not yet require use of chronic steroids for treatment of theirDMD, in the opinion of the Investigator, and are not receiving steroids at the timeof Screening.
rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.
Genetic mutation inclusion criteria vary by cohort.
Exclusion
Exclusion Criteria:
Has a concomitant illness, autoimmune disease, chronic drug treatment, and/orcognitive delay/impairment that in the opinion of the Investigator createsunnecessary risks for gene transfer.
Exposure to gene therapy, investigational medication, or any treatment designed toincrease dystrophin expression within protocol-specified time limits.
Abnormality in protocol-specified diagnostic evaluations or laboratory tests.
Cohort 8: Any confounding factors that would prevent the use of oral sirolimusincluding a known hypersensitivity to sirolimus or any of its excipients.
Other inclusion/exclusion criteria apply.
Study Design
Study Description
Connect with a study center
Arkansas Children's Hospital
Little Rock, Arkansas 72202
United StatesActive - Recruiting
Stanford University
Palo Alto, California 94304
United StatesActive - Recruiting
University of California, Davis
Sacramento, California 95616
United StatesActive - Recruiting
Stanford University
Palo Alto 5380748, California 5332921 94304
United StatesSite Not Available
University of California, Davis
Sacramento 5389489, California 5332921 95616
United StatesSite Not Available
Washington University in St. Louis
Saint Louis, Missouri 21205
United StatesSite Not Available
Washington University in St. Louis
St Louis, Missouri 63110
United StatesActive - Recruiting
Washington University in St. Louis
St Louis 4407066, Missouri 4398678 21205
United StatesSite Not Available
Nationwide Children's Hospital
Columbus, Ohio 43205
United StatesSite Not Available
Nationwide Children's Hospital
Columbus 4509177, Ohio 5165418 43205
United StatesSite Not Available
Neurology Rare Disease Center
Flower Mound, Texas 75028
United StatesActive - Recruiting
Children's Hospital of The King's Daughters
Norfolk, Virginia 23507
United StatesActive - Recruiting
Children's Hospital of The King's Daughters
Norfolk 4776222, Virginia 6254928 23507
United StatesSite Not Available

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