Phase
Condition
Muscular Dystrophy
Treatment
Plasmapheresis
delandistrogene moxeparvovec
Clinical Study ID
Ages 4-8 Male
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
Ambulatory per protocol specified criteria.
Has a definitive diagnosis of DMD prior to Screening based on documentation ofclinical findings and prior confirmatory genetic testing.
Ability to cooperate with motor assessment testing.
Has elevated AAVrh74 antibody titers per protocol-specified requirements.
A pathogenic frameshift mutation, nonsense mutation or premature stop codon orpathogenic variant in the DMD gene that is expected to lead to absence of dystrophinprotein with exception of a mutation in exon 8 and/or 9.
Stable daily dose of oral corticosteroids for at least 12 weeks prior to Screening,and the dose is expected to remain constant throughout the study (except formodifications to accommodate changes in weight).
Exclusion
Exclusion Criteria:
Has reduced left ventricular ejection fraction on the screening ECHO or clinicalsigns and/or symptoms of cardiomyopathy.
Presence of any other clinically significant illness, including cardiac, pulmonary,hepatic, renal, hematologic, immunologic, or behavioral disease, or infection ormalignancy or concomitant illness or requirement for chronic drug treatment that inthe opinion of the Investigator creates unnecessary risks for gene transfer or amedical condition or extenuating circumstance that, in the opinion of theInvestigator, might compromise the participant's ability to comply with the protocolrequired testing or procedures or compromise the participant's wellbeing, safety, orclinical interpretability.
Exposure to gene therapy, investigational medication, or other protocol-specifiedtreatment within the protocol specified time limits.
Abnormality in protocol-specified diagnostic evaluations or laboratory tests. .
Note: Other inclusion or exclusion criteria could apply.
Study Design
Connect with a study center
Washington University School of Medicine in St. Louis
Saint Louis, Missouri 63110
United StatesActive - Recruiting
Nationwide Children's Hospital
Columbus, Ohio 43205
United StatesActive - Recruiting
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