Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis
NCT ID: NCT02456103
Last Updated: 2020-04-27
Study Results
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View full resultsBasic Information
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TERMINATED
PHASE3
246 participants
INTERVENTIONAL
2015-08-31
2017-06-02
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Ataluren
Participants will be administered ataluren orally at a dose of 10 milligrams/grams (mg/kg) in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening for up to 96 weeks.
Ataluren
Ataluren will be provided as a vanilla-flavored powder to be mixed with water or milk.
Interventions
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Ataluren
Ataluren will be provided as a vanilla-flavored powder to be mixed with water or milk.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Evidence of signed and dated informed consent/assent document(s) indicating that the participant (and/or the participant's parent/legal guardian) has been informed of all pertinent aspects of the trial.
Exclusion Criteria
* Ongoing participation in any other therapeutic clinical trial.
6 Years
ALL
No
Sponsors
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PTC Therapeutics
INDUSTRY
Responsible Party
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Principal Investigators
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Joseph McIntosh, MD
Role: STUDY_DIRECTOR
PTC Therapeutics
Locations
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University of Alabama at Birmingham
Birmingham, Alabama, United States
Miller Children's Hospital Long Beach
Long Beach, California, United States
Children's Hospital Los Angeles
Los Angeles, California, United States
Children's Hospital and Research Center at Oakland
Oakland, California, United States
Stanford University-Children's Hospital
Palo Alto, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Nemours Children's Clinic
Jacksonville, Florida, United States
University of Miami
Miami, Florida, United States
Miami Children's Hospital
Miami, Florida, United States
All Children's Hospital
St. Petersburg, Florida, United States
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Indiana University
Indianapolis, Indiana, United States
Children's Hospital Boston
Boston, Massachusetts, United States
Washington University
St Louis, Missouri, United States
Morristown Medical Center
Morristown, New Jersey, United States
Beth Israel Medical Center
New York, New York, United States
New York University Langone Medical Center
New York, New York, United States
Columbia University Medical Center
New York, New York, United States
University of Cincinnati
Cincinnati, Ohio, United States
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States
Drexel University College of Medicine
Philadelphia, Pennsylvania, United States
Texas Children's Hospital
Houston, Texas, United States
University of Texas Health Science Center
Tyler, Texas, United States
Childrens Hospital of Wisconsin
Milwaukee, Wisconsin, United States
Hospital de Ninos Ricardo Gutierrez
Buenos Aires, , Argentina
Hospital Universitario Austral
Buenos Aires, , Argentina
Royal Adelaide Hospital
Adelaide, , Australia
Prince Charles Hospital
Chermside, , Australia
Princess Margaret Hospital
Perth, , Australia
University Hospital Brussels
Brussels, , Belgium
Hopital Universitaire des Enfants Reine Fabiola
Brussels, , Belgium
University Hospital Leuven
Leuven, , Belgium
Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul
Porto Alegre, , Brazil
University Mulitiprofile Hospital for Active Treatment Sveti Georgi EAD
Plovdiv, , Bulgaria
University Multiprofile Hospital for Active Treatment Aleksandrovska EAD
Sofia, , Bulgaria
Clinical Research Institute of Montreal
Montreal, , Canada
University of Toronto Hospital for Sick Children
Toronto, , Canada
British Columbia Children's Hospital
Vancouver, , Canada
Hoptial Arnaud de Villeneuve
Montpellier, , France
Hopital Necker - Enfants Malades
Paris, , France
Centre de Perharidy
Roscoff, , France
Centre Hospitalier Regional Sud Reunion
Saint-Pierre, , France
Charite-Universitatsmedizin Berlin
Berlin, , Germany
St. Josef Hospital GmbH
Bochum, , Germany
University of Cologne Children's Hospital
Cologne, , Germany
Christiane Herzog CF-Zentrum
Frankfurt am Main, , Germany
Universitatsklinikum Jena
Jena, , Germany
LMU Klinikum der Universitat Muchen
Munich, , Germany
Dr. Von Haunersches Kinderspital
München, , Germany
Ippokratio General Hospital Of Thessaloniki
Thessaloniki, , Greece
Meyer Children's Hospital
Haifa, , Israel
Hadassah University Hospital
Jerusalem, , Israel
Azienda Ospedaliero Universitaria Ospedall Riuniti di Acona-Umberto I G.M. Lancisi G. Salesi
Ancona, , Italy
Azienda Ospedaliera A Meyer
Florence, , Italy
Lombardia Cystic Fibrosis Center
Milan, , Italy
Azienda Policlinico Umberto I
Rome, , Italy
Ospedale Pediatrico Bambino Gesu
Rome, , Italy
Azienda Ospedaliera di Verona
Verona, , Italy
Hagaziekenhuis
s-Gravenweg, South Holland, Netherlands
Radboud University
Nijmegen, , Netherlands
Szpital Dzieciecy Polanki im Macieja Plazynskiego w Gdansku
Gdansk, , Poland
Institute of Mother and Child
Warsaw, , Poland
Hospital Universitario Vall d'Hebron
Barcelona, , Spain
Hospital University
Barcelona, , Spain
Hospital San Juan
Esplugues de Llobregat, , Spain
Hospital Regional Universitario de Malaga
Málaga, , Spain
Hospital de Sabadell, Consorci Sanitari Parc Tauli
Sabadell, , Spain
Hospital Universitario Virgen del Rocio
Seville, , Spain
St James University Hospital
Leeds, , United Kingdom
Royal Brompton Hospital
London, , United Kingdom
Countries
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Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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PTC124-GD-021e-CF
Identifier Type: -
Identifier Source: org_study_id
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