Conquering Diseases

Study Of An Investigational Drug For Duchenne Muscular Dystrophy

Description

Seeking boys aged 7 to 21 with Duchenne Muscular Dystrophy with an out-of-frame deletion mutation of the DMD gene amenable to exon 51-skipping treatment for a study of the highest tolerated dose of an investigational drug and if it increases dystrophin levels.

Overview

Participants in this study will be randomly assigned to one of two dose groups of an investigational drug. The drug will be infused once every 4 weeks. There will be about 56 study visits over 2 years.

What we're hoping for

We are studying the highest tolerated dose of an investigational drug and its effect on dystrophin levels in boys with Duchenne Muscular Dystrophy.

Additional Information

ClinicalTrials.gov Identifier: NCT04004065

 Principal Investigator

Brenda  Wong, MD

UMass Memorial Health Care

 Study Contact

Hannah  Alongi

774-455-6611

hannah.alongi@umassmed.edu

 Location

UMMS University Campus

55 Lake Ave North 

Worcester,MA 01655

508-856-8989