Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)

PHASE2TerminatedINTERVENTIONAL
Enrollment

6

Participants

Timeline

Start Date

January 13, 2010

Primary Completion Date

March 23, 2010

Study Completion Date

March 23, 2010

Conditions
Duchenne Muscular DystrophyBecker Muscular Dystrophy
Interventions
DRUG

Ataluren

Oral powder

DRUG

Chronic Corticosteroid Therapy

Enrollment was stratified to ensure evaluation of approximately half of the participants were receiving chronic corticosteroid therapy and approximately half of participants were not receiving chronic corticosteroid therapy. Therefore, 3 out of 6 participants were receiving chronic corticosteriod therapy. For the participants receiving chronic corticosteriod therapy, a stable corticosteriod regimen was to be maintained during the study.

Trial Locations (6)

43205

Nationwide Children's Hospital, Columbus

55455

University of Minnesota, Minneapolis

63110

Washington University Medical School, St Louis

95616

University of California-Davis, Davis

02115

Children's Hospital of Boston, Boston

NE1 3BZ

University of Newcastle, Newcastle upon Tyne

Sponsors

Lead Sponsor

All Listed Sponsors
collaborator

Genzyme, a Sanofi Company

INDUSTRY

lead

PTC Therapeutics

INDUSTRY

NCT01009294 - Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD) | Biotech Hunter | Biotech Hunter