Rush Medical Center Home Page Information for healthcare Professionals Rush University
FIND A DOCTOR
PATIENT & VISTOR SERVICES
HEALTH INFORMATION
CLINICAL SERVICES
EVENTS & CLASSES
RUSH NEWS ROOM
CLINICAL TRIALS
RESEARCH AT RUSH
NURSING AT RUSH
WORK AT RUSH
GIVING TO RUSH

Bookmark This Page
Clinical Trails at Rush Nonsense Mutation Dystrophinopathy and Ataluren Treatment Study
Clinical Trial Protocol ID
13011102

Clinical Trial Investigator Name
Peter Heydemann

Clinical Trial Title

Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy



Clinical Trial Protocol Description

Rush University Medical Center is recruiting for the Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in patients with nonsense mutation dystrophinopathy. This is an international study expecting to recruit 225 boys ages 7 to 16 with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation. As of the end of September,  Peter Heydemann, MD, has enrolled two boys with another three ready to start once they soon meet eligibility requirements.

DMD is a disabling and life-threatening disorder caused by defects in the gene for dystrophin, a protein that stabilizes muscle cell membranes. Until now, the only course of treatment involved corticosteroids, which merely slows progression. In previous studies, Ataluren earned a favorable safety profile and an earlier study showed that Ataluren can restore production of the missing dystrophin.

Recruitment remains open until the study is filled. For more information, please contact Susan Rohde at (312) 942-0079.



Clinical Trial Eligibility Criteria

You may be eligible to participate in this study if you:

  • Are a male between the ages of 7 to 16 with a nonsense mutation form of DMD and on a current regiment of corticosteroids

This is a partial list of eligibility requirements. To inquire about your eligibility, please call the contact number provided. If you wish to inquire via email, please include the title of the study in your message.





Contact Name Susan Rohde
Contact Phone (312) 942-0079
Promotional Information

Clinical Trials Home
Pediatric Neurology Trials
Nonsense Mutation Dystrophinopathy and Ataluren Treatment Study

   
Find a Doctor | Patient & Visitor Services | Health Information
Clinical Services | Events & Classes | Rush News Room | Clinical Trials
Research At Rush
Disclaimer | Privacy Statement | Site Map

© Rush University Medical Center, Chicago, Illinois