Goal of this study

To treat Duchenne muscular dystrophy.

 

Summary

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 and safety of drugs named SRP-4045 and SRP-4053 in patients with DMD. SRP-4045 and SRP-4053 will be given to the patient through intravenous infusion weekly for a period up to 144 weeks. Intravenous infusion refers to infusing the drug directly into a vein.

Who can participate in this study?

Patients who have been diagnosed with Duchenne muscular dystrophy, genetically diagnosed with out-of-frame deletion mutations amenable to exon 45 or 53 skipping treatment, and who are between 7 and 13 years of age 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: NCT02500381

Contact Information

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