A study to evaluate the safety and tolerability of a single dose of a drug named SRP-5051 in patients with Duchenne muscular dystrophy (DMD)
Goal of this study
To treat Duchenne muscular dystrophy.
Duchenne muscular dystrophy (DMD) is a genetic disease due to the absence of a protein called dystrophin. DMD is characterized by progressive muscle degeneration and weakness, and is due to deterioration of muscle cells which are important for body movement. This study will investigate the efficacy, safety and tolerability of a drug named SRP-5051 in patients with DMD. SRP-5051 will be given to the patient through intravenous infusion on Day 1. Intravenous infusion refers to infusing the drug directly into a vein. Total period of the study will be up to 14 weeks.
Who can participate in this study?
Patients who have been diagnosed with Duchenne muscular dystrophy, genetically diagnosed with an out-of-frame deletion mutation amenable to exon 51 skipping treatment, and who are 12 years and older may be eligible to participate in this study. This study accepts boys and does not accept healthy volunteers.
Important: Additional eligibility criteria apply and the patient’s record will be verified by the study doctor (with the patient’s approval) to confirm eligibility.
ClinicalTrials.gov Identifier: NCT03375255
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