Conquering Diseases

Study Of An Investigational Drug For Boys With Duchene Muscular Dystrophy


Seeking boys aged 6-14 years old who have previously been treated with adeno-associated virus (AAV) based gene therapy for a study of an investigational drug


Participants in this study will initially be randomly assigned to get either the investigational drug or a placebo (looks like the investigational drug but contains no medicine) for 12 weeks, then all participants will receive the investigational drug for the following 40 weeks. There will be at least 9 study visits and up to 10 phone calls over 14 months.

What we're hoping for

We are studying whether an investigational drug is safe and effective for boys with Duchenne muscular dystrophy (DMD) who were previously treated with gene therapy.

Additional Information Identifier: NCT06100887

 Principal Investigator

Brenda  Wong, MD

UMass Chan Medical School

 Study Contact

Sarah  Figueira



UMass Chan University Campus

55 Lake Ave North 

Worcester,MA 01655