Conquering Diseases

Study Of An Investigational Drug For Boys With Duchenne Muscular Dystrophy (Dmd) Who Can Walk

Description

Seeking boys between the ages of 6 and 12 with Duchenne Muscular Dystrophy (DMD) who can walk for a study of the effectiveness and safety of an investigational drug.

Overview

We are studying whether an investigational drug given in combination with systemic corticosteroids is more effective than a placebo (looks like the drug but contains no medication) given in combination with systemic corticosteroids in improving muscle strength and heart and lung function. Participants will get an infusion of the drug or placebo every 2 weeks for a year.

What we're hoping for

We hope to evaluate whether the investigational drug is more effective than placebo in improving muscle strength and heart and lung function in boys ages 6-12 who have Duchenne Muscular Dystrophy.

Additional Information

ClinicalTrials.gov Identifier: NCT04632940

 Principal Investigator

Brenda  Wong, MD

UMass Memorial Health Care

 Study Contact

Sarah  Figueira

508-856-1604

sarah.figueira@umassmed.edu

 Location

UMMS University Campus

55 Lake Ave North 

Worcester,MA 01655

508-856-8989