Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis
Launched by PTC THERAPEUTICS · May 26, 2015
Trial Information
Current as of May 13, 2025
Terminated
Keywords
ClinConnect Summary
The primary objective of this Phase 3 extension study will be to obtain long-term safety data to augment the overall safety database. The secondary objectives will be to augment the efficacy data collected in the double-blind study (PTC124-GD-021-CF; NCT02139306).
Gender
ALL
Eligibility criteria
- Inclusion Criteria:
- • Completion of study treatment (placebo or active) in the previous Phase 3, double-blind study protocol (Protocol PTC124-GD-021-CF)
- • 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:
- • Known hypersensitivity to any of the ingredients or excipients of the study drug.
- • Ongoing participation in any other therapeutic clinical trial.
About Ptc Therapeutics
PTC Therapeutics is a biotechnology company dedicated to the discovery and development of innovative medicines for the treatment of rare diseases and cancer. With a strong focus on advancing its proprietary drug development pipeline, PTC Therapeutics employs cutting-edge science and technology to address unmet medical needs. The company collaborates with a network of academic institutions, patient advocacy groups, and industry partners to drive research initiatives and clinical trials aimed at improving patient outcomes. Committed to patient-centered care, PTC Therapeutics strives to bring transformative therapies to market that enhance the quality of life for individuals affected by challenging health conditions.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
New York, New York, United States
Jacksonville, Florida, United States
Saint Petersburg, Florida, United States
Miami, Florida, United States
Oakland, California, United States
Boston, Massachusetts, United States
Los Angeles, California, United States
Birmingham, Alabama, United States
Miami, Florida, United States
Indianapolis, Indiana, United States
New York, New York, United States
Saint Louis, Missouri, United States
Aurora, Colorado, United States
Morristown, New Jersey, United States
Cincinnati, Ohio, United States
Hershey, Pennsylvania, United States
Philadelphia, Pennsylvania, United States
London, , United Kingdom
Chicago, Illinois, United States
Sevilla, , Spain
Adelaide, , Australia
Leuven, , Belgium
Berlin, , Germany
Barcelona, , Spain
Leeds, , United Kingdom
Buenos Aires, , Argentina
Rome, , Italy
Verona, , Italy
Brussels, , Belgium
Long Beach, California, United States
New York, New York, United States
Jerusalem, , Israel
Roscoff, , France
Milwaukee, Wisconsin, United States
Houston, Texas, United States
Paris, , France
Saint Petersburg, Florida, United States
Palo Alto, California, United States
Tyler, Texas, United States
Chermside, , Australia
Perth, , Australia
Porto Alegre, , Brazil
Plovdiv, , Bulgaria
Sofia, , Bulgaria
Montreal, , Canada
Toronto, , Canada
Vancouver, , Canada
Saint Pierre, , France
Bochum, , Germany
Frankfurt Am Main, , Germany
Haifa, , Israel
Firenze, , Italy
Milan, , Italy
Nijmegen, , Netherlands
Gdansk, , Poland
Barcelona, , Spain
Sabadell, , Spain
New York, New York, United States
Buenos Aires, , Argentina
Brussels, , Belgium
Montpellier, , France
Cologne, , Germany
Jena, , Germany
Munchen, , Germany
Munich, , Germany
Thessaloniki, , Greece
Ancona, , Italy
Rome, , Italy
S Gravenweg, Zuid Holland, Netherlands
Warsaw, , Poland
Esplugues De Llobregat, , Spain
Malaga, , Spain
Sevilla, Andalucía, Spain
Indianapolis, Indiana, United States
Patients applied
Trial Officials
Joseph McIntosh, MD
Study Director
PTC Therapeutics
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported
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