A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Sarepta Therapeutics, Inc.
With
Hoffmann-La Roche
Age
4–7
Sex
MALE
Healthy volunteers
Not accepted
You may be eligible if
Is ambulatory and from 4 to under 8 years of age at time of randomization.
Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
Ability to cooperate with motor assessment testing.
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 for modifications to accommodate changes in weight).
rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.
A pathogenic frameshift mutation or premature stop codon contained between exons 18 and 79 (inclusive), with the exception of mutation fully contained within exon 45.
You may not be if
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.
Presence of any other clinically significant illness, medical condition, or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risk for gene transfer.
Clareo Health | A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
