Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)

PHASE2CompletedINTERVENTIONAL
Enrollment

174

Participants

Timeline

Start Date

February 29, 2008

Primary Completion Date

December 31, 2009

Study Completion Date

December 31, 2009

Conditions
Duchenne Muscular DystrophyBecker Muscular Dystrophy
Interventions
DRUG

Ataluren

Ataluren will be administered as per the dose and schedule specified in the respective arms.

DRUG

Placebo

Placebo matching to ataluren will be administered as the schedule specified in the respective arm.

Trial Locations (37)

Unknown

University of California-Davis, Sacramento

The Children's Hospital, Aurora

Child Neurology Center of Pensacola, Pensacola

University of Iowa Healthcare, Iowa City

University of Kansas Medical Centre, Kansas City

Children's Hospital of Boston/Harvard Medical School, Boston

University of Minnesota, Minneapolis

Washington University School of Medicine, St Louis

Columbia University Medical School, New York

Duke University Medical Center, Durham

Shriners Hospital for Children, Portland

Children's Hospital of Philadelphia, Philadelphia

Southwestern University, Dallas

University of Utah, Salt Lake City

Royal Children's Hospital, Parkville

Institute For Neuromuscular Research, The Children's Hospital at Westmead, Westmead

University Hospital KU Leuven, Leuven

Alberta Children's Hospital, Calgary

British Columbia Children's Hospital, Vancouver

London Health Sciences Center, London

Service de Neuropediatrie, hôpital La Timone, Marseille

Neuromuscular center of Nantes, Nantes

Groupe Hospitalier La Pitie-Salpetriere, Paris

University Clinic for Children, University of Essen, Essen

University Hospital, Freiburg im Breisgau

Hadassah Medical Center, Hebrew University Hospital, Jerusalem

Ospedale Maggiore Policlinico in Milan, Milan

Ospedale Pediatrico Bambino Gesu, Rome

U.O. Complessa di Neuropsichiatria Infantile, Rome

Hospital Sant Joan de déu, Barcelona

Hospital Universitari La Fe, Valencia

Queen Silvia Children's Hospital, Gothenburg

Astrid Lindgren Pediatric Hospital, Stockholm

Imperial College London, Hammersmith Hospital, London

Univ of Newcastle, Institute of Human Genetics, Newcastle

Robert Jones and Agnes Hunt Orthopaedic NHS Trust, Oswestry

45229-3039

Cincinnati Children's Hospital Medical Center, Cincinnati

Sponsors

Lead Sponsor

All Listed Sponsors
lead

PTC Therapeutics

INDUSTRY