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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 Hospital of Wisconsin 
ClinicalTrials.gov  NCT03179631
https://www.clinicaltrials.gov/ct2/show/NCT03179631?term=PTC124-GD-041-DMD&rank=1

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 Hospital of Wisconsin
ClinicalTrials.gov NCT03400852
https://www.clinicaltrials.gov/ct2/show/NCT03400852?term=MNK14112096&rank=1

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 Hospital of Wisconsin
ClinicalTrials.gov NCT03406780
https://www.clinicaltrials.gov/ct2/show/NCT03406780?term=CAP-1002-DMD-02&rank=1

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.

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For more information

Please contact our Pediatric Neurology Research Staff at
  • (414) 266-3355

or pedneuromuscular@mcw.edu.

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