An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
NCT ID: NCT02090959
Last Updated: 2020-08-11
Study Results
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View full resultsBasic Information
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TERMINATED
PHASE3
219 participants
INTERVENTIONAL
2014-03-20
2018-06-12
Brief Summary
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This study may be further extended by amendment until either ataluren becomes commercially available or the clinical development of ataluren in duchenne muscular dystrophy (DMD) is discontinued.
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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 receive ataluren suspension orally 3 times a day (TID), 10 milligrams/kilogram (mg/kg) at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for up to 144 weeks.
Ataluren
Ataluren will be administered per the dose and schedule specified in the arm.
Interventions
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Ataluren
Ataluren will be administered per the dose and schedule specified in the arm.
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 his parent/legal guardian) has been informed of all pertinent aspects of the trial. Note: If the study candidate is considered a child under local regulation, a parent or legal guardian must provide written consent prior to initiation of study screening procedures and the study candidate may be required to provide written assent. The rules of the responsible Institutional Review Board/Independent Ethic Committee (IRB/IEC) regarding whether 1 or both parents must provide consent and the appropriate ages for obtaining consent and assent from the participant should be followed.
* In participants who are sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during the period of study drug administration and 6-week follow-up period.
* Willingness and ability to comply with scheduled visits, ataluren administration plan, study procedures, laboratory tests, and study restrictions.
Exclusion Criteria
* Ongoing participation in any other therapeutic clinical trial.
* Prior or ongoing medical condition (for example, concomitant illness, psychiatric condition, behavioral disorder, alcoholism, drug abuse), medical history, physical findings, electrocardiogram (ECG) findings, or laboratory abnormality that, in the investigator's opinion, could adversely affect the safety of the participant, makes it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results.
7 Years
15 Years
MALE
No
Sponsors
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PTC Therapeutics
INDUSTRY
Responsible Party
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Principal Investigators
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Francesco Bibbiani, M.D.
Role: STUDY_DIRECTOR
PTC Therapeutics
Locations
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University of California, Los Angeles
Los Angeles, California, United States
UC Davis Medical Center
Sacramento, California, United States
Stanford University Medical Center
Stanford, California, United States
Children's Hospital Colorado - Center for Cancer and Blood Disorders
Aurora, Colorado, United States
Child Neurology Center of Northwest Florida
Gulf Breeze, Florida, United States
Rush University Medical Center
Chicago, Illinois, United States
University of Iowa
Iowa City, Iowa, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Children's Hospital Boston
Boston, Massachusetts, United States
University of Minnesota
Minneapolis, Minnesota, United States
Washington University School of Medicine, Division of Endocrinology, Metabolism and Lipid Research
St Louis, Missouri, United States
Columbia University College of Physicians & Surgeons
New York, New York, United States
Children's Hospital Cincinnati
Cincinnati, Ohio, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Oregon Health & Science University
Portland, Oregon, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
Childrens Medical Center Dallas, Texas
Dallas, Texas, United States
Texas Children's Hospital
Houston, Texas, United States
University of Utah
Salt Lake City, Utah, United States
Seattle Children's Hospital - Childhood Cancer and Blood Disorders
Seattle, Washington, United States
The Children's Hospital at Westmead
Westmead, New South Wales, Australia
The Royal Children's Hospital
Parkville, Victoria, Australia
UZ Leuven
Leuven, , Belgium
Universidade Federal do Rio de Janeiro - Instituto de Puericultura e Pediatria Martagao Gesteira
Rio de Janeiro, , Brazil
Sao Paulo University -HC/FMUSP
São Paulo, , Brazil
Aleksandrovska Hospital
Sofia, , Bulgaria
Alberta Children's Hospital
Calgary, Alberta, Canada
British Columbia Children's Hospital
Vancouver, British Columbia, Canada
Children's Hospital of Western Ontario
London, Ontario, Canada
Hospital Luis Calvo Mackenna
Santiago, Santiago Metropolitan, Chile
Hospital Clinico Universidad Catolica
Santiago, , Chile
University Hospital Brno
Brno, , Czechia
Motol University Hospital
Prague, , Czechia
Hospital de la Timone
Marseille, , France
CHU de Nantes
Nantes, , France
Hopital Necker - Enfants Malades
Paris, , France
Groupe Hospitalier Pitie-Salpetriere
Paris, , France
Universitaetsklinikum Essen
Essen, , Germany
University Medical Center Freiburg
Freiburg im Breisgau, , Germany
Universitat Munchen - von Haunersche Kinder Clinic
München, , Germany
Hadassah University Hospital
Jerusalem, , Israel
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano
Milan, , Italy
Bambino Gesu Hospital
Rome, , Italy
U.O. Complessa di Neuropsichiatria Infantile
Rome, , Italy
Policlinico Universitario G. Martino
Sicily, , Italy
Medical University of Warsaw
Warsaw, , Poland
Seoul National University Hospital
Seoul, , South Korea
Hospital Sant Joan de Deu
Barcelona, , Spain
Hospital Universitari i Politecnic la Fe
Valencia, , Spain
Queen Silvia Children's Hospital
Gothenburg, , Sweden
Astrid Lindgren Childrens Hospital
Stockholm, , Sweden
CHUV Lausanne
Lausanne, , Switzerland
Hacettepe Childrens Hospital
Ankara, , Turkey (Türkiye)
Erciyes University Faculty of Medicine
Talas/Kayseri, , Turkey (Türkiye)
University College London Institute of Child Health
London, , United Kingdom
Royal Manchester Children's Hospital
Manchester, , United Kingdom
The Newcastle upon Tyne Hospitals, NHS Foundation Trust
Newcastle upon Tyne, , United Kingdom
Countries
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References
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Thangarajh M, Elfring GL, Trifillis P, McIntosh J, Peltz SW; Ataluren Phase 2b Study Group. The relationship between deficit in digit span and genotype in nonsense mutation Duchenne muscular dystrophy. Neurology. 2018 Sep 25;91(13):e1215-e1219. doi: 10.1212/WNL.0000000000006245. Epub 2018 Aug 22.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Related Links
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Related Info
Other Identifiers
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PTC124-GD-020e-DMD
Identifier Type: -
Identifier Source: org_study_id
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