Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)

PHASE3CompletedINTERVENTIONAL
Enrollment

279

Participants

Timeline

Start Date

August 31, 2014

Primary Completion Date

November 30, 2016

Study Completion Date

November 30, 2016

Conditions
Cystic Fibrosis
Interventions
DRUG

Ataluren (PTC124®)

Oral Ataluren TID

DRUG

Placebo

Oral Placebo TID

Trial Locations (88)

1020

Hôpital Universitaire des Enfants Reine Fabiola, Brussels

1090

University Hospital Brussels, Brussels

1900

Hospital de Niños Superiora Sor Maria Ludovica, La Plata

2491

Hagaziekenhuis, The Hague

3000

University Hospital Leuven, Leuven

4032

Prince Charles Hospital, Chermside

5000

Royal Adelaide Hospital, Adelaide

6840

Princess Margaret Hospital, Perth

10003

Beth Israel Medical Center, New York

10016

New York University Langone Medical Center, New York

10032

Columbia University Medical Center, New York

13210

SUNY Upstate Medical University, Syracuse

13353

Charité Universitätsmedizin Berlin, Berlin

17033

Penn State Milton S. Hershey Medical Center, Hershey

19104

Children's Hospital of Philadelphia, Philadelphia

19129

Drexel University College of Medicine, Philadelphia

20122

Lombardia Cystic Fibrosis Center, Milan

29684

Centre de Perharidy, Roscoff

31096

Meyer Children's Hospital, Haifa

32207

Nemours Children's Clinic, Jacksonville

33136

University of Miami, Miami

33155

Miami Children's Hospital, Miami

33701

All Children's Hospital, St. Petersburg

34295

Hôpital Arnaud de Villeneuve, Montpellier

35233

University of Alabama at Birmingham, Birmingham

36608

Pulmonary Associates of Mobile PC, Mobile

37126

University of Verona, Verona

44106

Rainbow Babies & Children's Hospital, Cleveland

44791

St. Josef Hospital GmbH, Bochum

45221

University of Cincinnati, Cincinnati

46202

Indiana University, Indianapolis

50139

Azienda Ospedaliera A Meyer, Florence

50937

Universitätsklinikum Köln, Cologne

53226

Childrens Hospital of Wisconsin, Milwaukee

60123

Ospedali Riuniti di Ancona, Ancona

60590

Christiane Herzog CF-Zentrum, Frankfurt am Main

60611

Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago

63110

Washington University, St Louis

69677

Hôpital Femme-Mère-Enfant, Bron

73112

Santiago Reyes, MD, Oklahoma City

75015

Hôpital Necker-Enfants Malades, Paris

75708

University of Texas Health Science Center, Tyler

77094

Texas Children's Hospital, Houston

80045

Children's Hospital Colorado, Aurora

80336

LMU Klinikum der Universität München, München

80337

Dr. Von Haunersches Kinderspital, München

90027

Children's Hospital Los Angeles, Los Angeles

90806

Miller Children's Hospital Long Beach, Long Beach

91240

Hadassah University Hospital, Jerusalem

94304

Stanford University-Children's Hospital, Palo Alto

94609

Children's Hospital and Research Center at Oakland, Oakland

97448

Centre Hospitalier Regional Sud Reunion, Saint-Pierre

02115

Children's Hospital Boston, Boston

07740

Monmouth Medical Center, Long Branch

07960

Morristown Medical Center, Morristown

B1629ODT

Hospital Universitario Austral, Buenos Aires

C1425EFD

Hospital de Niños Dr. Ricardo Gutiérrez, Buenos Aires

Unknown

Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul, Porto Alegre

University Mulitiprofile Hospital for Active Treatment Sveti Georgi EAD, Plovdiv

University Multiprofile Hospital for Active Treatment Aleksandrovska EAD, Sofia

General Hospital of Thessaloniki Ippokration, Thessaloniki

Ospedale Pediatrico Bambino Gesù IRCCS, Roma

Erasmus MC, Rotterdam

Szpital Dzieciecy Polanki im Macieja Plazynskiego w Gdansku, Gdansk

Hospital Regional Universitario de Malaga, Málaga

Hospital Universitario Virgen del Rocio, Seville

Birmingham Children's Hospital NHS Foundation Trust, Birmingham

Heart of England NHS Foundation Trust, Birmingham

St James's University Hospital, Leeds

Royal Brompton Hospital, London

Southampton University Hospitals NHS Trust, Southampton

05403-000

Instituto da Criança - Hospital das Clínicas, São Paulo

H2W 1R7

Clinical Research Institute of Montreal, Montreal

G1V 4G2

CHU de Quebec - Hopital CHUL, Québec

M5G 1X8

University of Toronto Hospital for Sick Children, Toronto

V6H 3V4

British Columbia Children's Hospital, Vancouver

07745

Universitätsklinikum Jena, Jena

00161

Azienda Policlinico Umberto I, Rome

6525 GA

Radboud University, Nijmegen

34-700

NZOZ Sanatorium Cassia-Villa Medica, Rabka-Zdrój

35-612

NZOZ Podkarpacki Osrodek Pulmonologii i Alergologii, Rzeszów

01-211

Instytut Matki I Dziecka, Warsaw

08035

Hospital Universitario Vall d'Hebron, Barcelona

Hospital University, Barcelona

08950

Hospital Sant Joan de Deu, Esplugues de Llobregat

08208

Hospital de Sabadell, Consorci Sanitari Parc Tauli, Sabadell

G120YN

Southern General Hospital, Glasgow

CF64 2XX

Llandough Hospital, Penarth

Sponsors
All Listed Sponsors
collaborator

Cystic Fibrosis Foundation

OTHER

collaborator

ECFS-Clinical Trial Network (ECFS-CTN)

UNKNOWN

lead

PTC Therapeutics

INDUSTRY

NCT02139306 - Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF) | Biotech Hunter | Biotech Hunter