An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy

PHASE3TerminatedINTERVENTIONAL
Enrollment

219

Participants

Timeline

Start Date

March 20, 2014

Primary Completion Date

June 12, 2018

Study Completion Date

June 12, 2018

Conditions
Muscular Dystrophy, DuchenneMuscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, X-LinkedGenetic Diseases, Inborn
Interventions
DRUG

Ataluren

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

Trial Locations (58)

1011

CHUV Lausanne, Lausanne

1431

Aleksandrovska Hospital, Sofia

2145

The Children's Hospital at Westmead, Westmead

3000

UZ Leuven, Leuven

3052

The Royal Children's Hospital, Parkville

10032

Columbia University College of Physicians & Surgeons, New York

13385

Hospital de la Timone, Marseille

15224

Children's Hospital of Pittsburgh, Pittsburgh

17176

Astrid Lindgren Childrens Hospital, Stockholm

19104

The Children's Hospital of Philadelphia, Philadelphia

20122

Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano, Milan

32561

Child Neurology Center of Northwest Florida, Gulf Breeze

38039

Erciyes University Faculty of Medicine, Talas/Kayseri

43209

Nationwide Children's Hospital, Columbus

44093

CHU de Nantes, Nantes

45122

Universitaetsklinikum Essen, Essen

45229

Children's Hospital Cincinnati, Cincinnati

46026

Hospital Universitari i Politecnic la Fe, Valencia

52242

University of Iowa, Iowa City

55455

University of Minnesota, Minneapolis

60612

Rush University Medical Center, Chicago

63110

Washington University School of Medicine, Division of Endocrinology, Metabolism and Lipid Research, St Louis

66160

University of Kansas Medical Center, Kansas City

75015

Hopital Necker - Enfants Malades, Paris

75207

Childrens Medical Center Dallas, Texas, Dallas

75651

Groupe Hospitalier Pitie-Salpetriere, Paris

77030

Texas Children's Hospital, Houston

79106

University Medical Center Freiburg, Freiburg im Breisgau

80045

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

80337

Universitat Munchen - von Haunersche Kinder Clinic, München

84112

University of Utah, Salt Lake City

90095

University of California, Los Angeles, Los Angeles

91240

Hadassah University Hospital, Jerusalem

94305

Stanford University Medical Center, Stanford

95817

UC Davis Medical Center, Sacramento

97239

Oregon Health & Science University, Portland

98105

Seattle Children's Hospital - Childhood Cancer and Blood Disorders, Seattle

98125

Policlinico Universitario G. Martino, Sicily

8330073

Hospital Clinico Universidad Catolica, Santiago

02115

Children's Hospital Boston, Boston

21.941-912

Universidade Federal do Rio de Janeiro - Instituto de Puericultura e Pediatria Martagao Gesteira, Rio de Janeiro

05403-900

Sao Paulo University -HC/FMUSP, São Paulo

T3B 6A8

Alberta Children's Hospital, Calgary

V6H 3V4

British Columbia Children's Hospital, Vancouver

N6C 2RC

Children's Hospital of Western Ontario, London

Unknown

Hospital Luis Calvo Mackenna, Santiago

635 00

University Hospital Brno, Brno

150 06

Motol University Hospital, Prague

00165

Bambino Gesu Hospital, Rome

00168

U.O. Complessa di Neuropsichiatria Infantile, Rome

502-097

Medical University of Warsaw, Warsaw

110-744

Seoul National University Hospital, Seoul

08950

Hospital Sant Joan de Deu, Barcelona

SE-416 85

Queen Silvia Children's Hospital, Gothenburg

06100

Hacettepe Childrens Hospital, Ankara

WC1N 3JH

University College London Institute of Child Health, London

M13 9WL

Royal Manchester Children's Hospital, Manchester

NE1 3BZ

The Newcastle upon Tyne Hospitals, NHS Foundation Trust, Newcastle upon Tyne

Sponsors

Lead Sponsor

All Listed Sponsors
lead

PTC Therapeutics

INDUSTRY

NCT02090959 - An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy | Biotech Hunter | Biotech Hunter