Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

PHASE3CompletedINTERVENTIONAL
Enrollment

360

Participants

Timeline

Start Date

July 6, 2017

Primary Completion Date

March 5, 2022

Study Completion Date

July 25, 2023

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

Ataluren

10, 20 mg/kg

DRUG

PLACEBO

10, 20 mg/kg

Trial Locations (64)

1793

UMHAT Sofiamed, Sofia

2145

The Childrens Hospital at Westmead, Westmead

3052

The Royal Childrens Hospital, Parkville

3080

Seoul National University Hospital, Seoul

6009

Perth Children's Hospital, Nedlands

10002

National Taiwan University Hospital, Taipei

10032

Columbia University College of Physicians & Surgeons, New York

10700

Siriraj Hospital, Bangkok

14389

Instituto Nacional de Rehabilitacion, Tlalpan

15224

University of Pittsburgh School of Medicine, Pittsburgh

23507

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

31217

Hospital Angeles Chihuahua, Chihuahua City

32561

Northwest Florida Clinical Research Group, LLC, Gulf Breeze

34093

Istanbul University- Instanbul Medical Faculty, Istanbul

45229

Cincinnati Children's Hospital Medical Center, Cincinnati

46202

Indiana University Health - Riley Child Neurology, Indianapolis

48109

University of Michigan - CS Mott Children's Hospital, Ann Arbor

48201

Children's Hospital of Michigan, Detroit

50586

Hospital Tunku Azizah Kuala Lumpur, Kuala Lumpur

50612

Pusan National University Yangsan Hospital, Yangsan

55455

University of Minnesota, Minneapolis

59100

University Malaya Medical Centre (UMMC), Pantai

66160

University of Kansas Medical Center, Kansas City

76104

Cook Childrens Medical Center, Fort Worth

77030

Texas Children's Hospital, Houston

80756

Kaohsiung Medical University Chung-Ho Memorial Hospital, Kaohsiung City

84112

University Of Utah, Salt Lake City

85016

Phoenix Childrens Hospital, Phoenix

90027

Children's Hospital of Los Angeles, Los Angeles

92408

Loma Linda University Children's Hospital, San Bernardino

94143

University of California, San Francisco (UCSF) - Benioff Children's Hospital - Oakland, Oakland

94305

Stanford University Medical Center, Palo Alto

95817

University of California (UC) Davis Medical Center, Sacramento

97239

Shriners Hospital for Children, Portland

98105

Seattle Children's Hospital, Seattle

100039

General Hospital of Chinese Armed Police Forces, Beijing

110029

All India Institute of Medical Sciences, New Delhi

125412

Russian National Research Medical University n.a. N.I.Pirogov, structural branch - Research Clinical Institute of Pediatrics n.a. Academician Yu. E. Veltishchev, Moscow

160012

Postgraduate Institute of Medical Education and Research, Chandigarh

194100

"Saint Petersburg State Paediatric Medical University based at Consultative and Diagnostic Centre", Saint Petersburg

200032

Children's Hospital of Fudan University, Shanghai

350005

The First Affiliated Hospital of Fujian Medical University, Fuzhou

380005

Panchshil Hospital, Ahmedabad

400016

P.D. Hinduja Hospital, Māhīm

410008

Xiangya Hospital Central South University, Hunan

500082

Nizam's Institute of Medical Sciences (NIMS), Hyderabad

518038

Shenzhen Children's Hospital, Shenzhen

560029

National Institute of Mental Health and Neurosciences, Bengaluru

600006

Apollo Children's Hospital Chennai, Chennai

632004

Christian Medical College Hospital Vellore, Vellore

700054

Apollo Gleneagles Hospital, Kolkata

78229-3900

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

Q4101

Queensland Children's Hospital, South Brisbane

30130-100

Hospital de Clínicas da Universidade Federal de Minas Gerais, Belo Horizonte

21.941-912

Universidade Federal do Rio de Janeiro, Rio de Janeiro

05403-000

Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo - FMUSP, São Paulo

N6A5W9

Childrens Hospital London Health Sciences Centre, London

K1H8L1

Childrens Hospital of Eastern Ontario, Ottawa

SAR

Queen Mary Hospital, Hong Kong

Unknown

PTC Clinical Site, Multiple Locations

04530

Instituto Nacional de Pediatría, Mexico City

02-097

Samodzielny Publiczny Centralny Szpital Kliniczny w Warszawie, Warsaw

00936-5067

University of Puerto Rico - School of Medicine, San Juan

06351

Samsung Medical Center, Seoul

Sponsors

Lead Sponsor

All Listed Sponsors
lead

PTC Therapeutics

INDUSTRY

NCT03179631 - Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy | Biotech Hunter | Biotech Hunter