Protocol summaries

Protocol No. PTC124-GD-041-DMD
Principal Investigator Matthew Harmelink, MD
Phase   III
Title A Phase 3, Randomized, Double-Blind, Placebo Controlled Efficacy and Safety Study of Ataluren in Patients with Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension
Associate Disease(s) Duchenne Muscular Dystrophy (DMD)
Description (Summary)
This clinical trial aims to evaluate the long-term efficacy of Ataluren in male patients with nonsense mutation Duchenne muscular dystrophy who are at least 5 years old.
Participating Institutions Children's Wisconsin
ClinicalTrials.gov NCT03179631


Protocol No. MNK14112096 (BRAVE)
Principal Investigator Matthew Harmelink, MD
Phase   II
Title A Multicenter, Randomized, Parallel Group, Double Blind, Multiple Dose, Placebo Controlled Study to Assess the Efficacy and Safety of MNK-1411 in Male Subjects 4 to 8 Years of Age with Duchenne Muscular Dystrophy
Associate Disease(s) Duchenne Muscular Dystrophy (DMD)
Description (Summary)
BRAVE is a clinical trial evaluating the safety and efficacy of MNK-1411 in male subjects 4 to 8 years of age with Duchenne Muscular Dystrophy (DMD).
Participating Institutions Children's Wisconsin
ClinicalTrials.gov NCT03400852


Protocol No.

CAP-1002-DMD-02 (HOPE-2)
Principal Investigator Matthew Harmelink, MD
Phase    II
Title A Phase 2, Randomized, Double-Blind, Placebo-Controlled Trial Evaluating the Safety and Efficacy of Intravenous Delivery of Allogeneic Cardiosphere-Derived Cells in Subjects with Duchenne Muscular Dystrophy
Associate Disease(s) Duchenne Muscular Dystrophy (DMD)
Description (Summary)
HOPE-2 is a clinical trial evaluating the safety and efficacy of a cell therapy called CAP-1002 in study participants with Duchenne muscular dystrophy (DMD) who are at least 10 years old.
Participating Institutions Children's Wisconsin
ClinicalTrials.gov NCT03406780

For the most current information or questions about our clinical trials, please contact our Pediatric Neurology Research Staff at (414) 266-3355 or pedneuromuscular@mcw.edu.

Please contact our Pediatric Neurology Research Staff at

(414) 266-3355

Email: 
pedneuromuscular@mcw.edu

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