Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)

PHASE3Enrolling by invitationINTERVENTIONAL
Enrollment

270

Participants

Timeline

Start Date

November 30, 2010

Primary Completion Date

April 30, 2025

Study Completion Date

April 30, 2025

Conditions
Duchenne Muscular Dystrophy
Interventions
DRUG

Ataluren

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

Trial Locations (34)

10032

Columbia University Pediatric Neuromuscular Center, New York

16148

University of Pittsburgh Medical Center, Pittsburgh

19104

Children's Hospital of Philadelphia, Philadelphia

23507

Children's Hospital of the King's Daughters, Norfolk

27710

Duke University Medical Center, Durham

30329

Rare Disease Research, LLC, Atlanta

32561

Child Neurology Center of NW Florida NW, Gulf Breeze

43205

Nationwide Children's Hospital, Columbus

45229

Cincinnati Childrens Hospital Medical Center, Cincinnati

48109

University of Michigan, Ann Arbor

52242

University of Iowa Children's Hospital, Iowa City

55455

University of Minnesota, Minneapolis

60612

Rush Univ Medical Center, Chicago

63110

Washington University Medical School, St Louis

66160

University of Kansas Medical Center, Kansas City

75207

Childrens Medical Center Dallas, Dallas

77030

Texas Children's Hospital, Houston

78229

University of Texas Health Science Center at San Antonio, San Antonio

80045

Children's Hospital Colorado - Center for Cancer and Blood Disorders, Aurora

84112

University of Utah, Salt Lake City

85016

Phoenix Childrens Hospital, Phoenix

90027

Children's Hospital of Los Angeles, Los Angeles

90095

UCLA, Los Angeles

94158

University of CA, San Francisco-Benioff Children's Hospital, San Francisco

94304

Stanford University, Palo Alto

95817

University of California-Davis, Sacramento

97239

Shriners Hospital for Children-Portland, Portland

98105

Seattle Children's Hospital, Seattle

02115

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

Unknown

Alberta Children's Hospital, Calgary

British Columbia Children's Hospital, Vancouver

London Health Sciences Centre, London

Children's Hospital of East Ontario, Ottawa

CHU de Québec - Université Laval, Québec

Sponsors

Lead Sponsor

All Listed Sponsors
lead

PTC Therapeutics

INDUSTRY