Seeking boys aged 4 to 16 for a study of an investigational drug to increase dystrophin protein levels in muscles.
Participants in this study will be placed into a group. Each group of participants will be infused with either a dose of the investigational drug or a placebo (looks like the drug but contains no medication). When participants are given the initial dose, they will need to stay at the clinic for about 2 days. After that the drug or placebo will be infused every 4 weeks. After 6 months each participant will receive the study drug.
What we're hoping for
We are evaluating different doses of an investigational drug to assess the safety and effectiveness of increasing dystrophin protein levels in muscle tissue.
ClinicalTrials.gov Identifier: NCT05524883