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Clinical Trial NCT04004065

A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

  • ClinicalTrials.gov ID: NCT04004065
  • Not accepting healthy volunteers
  • UTSW Principal Investigator: SUSAN THERESA IANNACCONE

objective

To evaluate the safety and tolerability of multiple ascending doses of SRP-5051 (4 mg/kg, 10 mg/kg, 20 mg/kg, and 30 mg/kg), administered intravenously (IV) every 4 weeks, and determine the maximum tolerated dose (MTD); To evaluate dystrophin protein level in skeletal muscle tissue following SRP-5051 treatment, administered IV every 4 weeks at the doses selected based on data from Part A

If you are interested in this clinical trial, please contact Alexandria Silver on the Children’s Health Research Team.Call 214-456-0876Email