Seeking boys with Duchenne Muscular Dystrophy for a study of an investigational gene therapy drug.
We are comparing the investigational drug to a placebo (which looks like the drug but doesn't contain medicine) to see if the investigational drug is safe and effective in improving muscle function for boys with Duchenne Muscular Dystrophy. All participants will receive the investigational drug for one year and the placebo for one year, but neither they nor their healthcare providers will know which one they receive first.
What we're hoping for
We hope that the investigational drug will help boys with Duchenne Muscular Dystrophy to make dystrophin within their own bodies and that this will improve their muscle function.
ClinicalTrials.gov Identifier: NCT04281485