This site became the new on June 19th. Learn more.
Show more Menu IMPORTANT: Listing of a study on this site does not reflect endorsement by the National Institutes of Health. Talk with a trusted healthcare professional before volunteering for a study. Read more... Menu IMPORTANT: Talk with a trusted healthcare professional before volunteering for a study. Read more... Menu
Give us feedback

Study of Ataluren (PTC124) in Cystic Fibrosis

This study has been terminated.
(CF Data from CF study 021 did not meet end points.)
Information provided by (Responsible Party):
PTC Therapeutics Identifier:
First received: April 4, 2014
Last updated: May 30, 2017
Last verified: May 2017
This is a Phase 3, multicenter, open-label, safety and efficacy study of ataluren in patients with nonsense mutation cystic fibrosis who participated in the previous Phase 3 study with ataluren (PTC124-GD-009-CF; Study 009).

Condition Intervention Phase
Cystic Fibrosis Drug: Ataluren (PTC124) Phase 3

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: No masking
Primary Purpose: Treatment
Official Title: An Open-Label Safety and Efficacy Study for Patients With Nonsense Mutation Cystic Fibrosis Previously Treated With Ataluren (PTC124)

Resource links provided by NLM:

Further study details as provided by PTC Therapeutics:

Primary Outcome Measures:
  • Adverse events and laboratory abnormalities [ Time Frame: 192 weeks ]

Secondary Outcome Measures:
  • Forced expiratory volume in 1 second (FEV1) [ Time Frame: 192 weeks ]
  • Pulmonary exacerbation rate [ Time Frame: 192 weeks ]
  • 12 Lead ECG [ Time Frame: 192 Weeks ]
  • Vital Signs [ Time Frame: 192 Weeks ]

Other Outcome Measures:
  • Change from baseline to end of treatment in spirometric performace as measured by FVC and FEF25-75 [ Time Frame: 192 Weeks ]

Enrollment: 61
Study Start Date: May 2014
Estimated Study Completion Date: May 2017
Estimated Primary Completion Date: May 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Ataluren (PTC124)
Ataluren (PTC124)
Drug: Ataluren (PTC124)
Oral powder for suspension taken 3 times per day (10-, 10-, and 20-mg/kg morning, midday and evening, respectively) for 192 weeks

Detailed Description:
This is a Phase 3, multicenter, open-label, safety and efficacy study of ataluren in patients with nonsense mutation cystic fibrosis who participated in the previous Phase 3 study with ataluren (PTC124-GD-009-CF; Study 009). The primary objective of this study is to determine the long-term safety and tolerability of ataluren in patients with nmCF, as assessed by adverse events and laboratory abnormalities. The secondary objective of this study includes the assessment of the efficacy of ataluren, as measured by FEV1 and pulmonary exacerbation rate, and change from baseline in other safety parameters (eg, 12-lead ECG measurements, vital signs). Study subjects will be enrolled at approximately 17 sites in the United States, Europe and Israel that previously participated in Study PTC124-GD-009-CF. All study subjects will receive 10-, 10-, and 20-mg/kg ataluren (PTC124) 3 times per day (morning, midday and evening, respectively) for 192 weeks. Subjects will be evaluated at clinic visits every 16 weeks and supplemented with interim phone calls in between onsite visits. A final 4-week post-treatment follow-up visit will also be conducted.

Ages Eligible for Study:   6 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Key Inclusion Criteria:

  • Ability to provide written informed consent (parental/guardian consent and subject assent if <18 years of age)
  • Evidence of completed participation in the double-blind study, PTC124-GD-009-CF (Study 009)
  • Body weight >16 kg.
  • Performance of a valid, reproducible spirometry test using the study-specific spirometer during the screening period.
  • Confirmed laboratory values within the central laboratory ranges at screening
  • In male and female subjects who are sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during the study drug administration and 60-day follow-up period
  • Willingness and ability to comply with all study procedures and assessments, including scheduled visits, drug administration plan, laboratory tests, and study restrictions

Key Exclusion Criteria:

  • Chronic use of systemic tobramycin within 4 weeks prior to screening
  • Evidence of pulmonary exacerbation or acute upper or lower respiratory tract infection (including viral illnesses) within 3 weeks prior to screening or between screening and enrollment
  • Any change (initiation, change in type of drug, dose modification, schedule modification, interruption, discontinuation, or re-initiation) in a chronic treatment/prophylaxis regimen for CF or for CF-related conditions within 4 weeks prior to screening.
  • Known hypersensitivity to any of the ingredients or excipients of the study drug
  • Exposure to another investigational drug within 4 weeks prior to screening or ongoing participation in any other therapeutic clinical trial
  • Treatment with intravenous antibiotics within 3 weeks prior to screening
  • History of solid organ or hematological transplantation
  • Ongoing immunosuppressive therapy (other than corticosteroids)
  • Positive hepatitis B surface antigen, hepatitis C antibody test or HIV test
  • Known portal hypertension
  • Pregnancy or breast-feeding
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT02107859

United States, Alabama
University of Alabama-Birmingham
Birmingham, Alabama, United States, 35233
United States, California
Miller Children's Hospital Long Beach
Long Beach, California, United States, 90806
United States, Colorado
Denver Children's Hospital
Aurora, Colorado, United States, 80045
United States, Illinois
Children's Hospital Chicago
Chicago, Illinois, United States, 60614
United States, Massachusetts
Children's Hospital Boston
Boston, Massachusetts, United States, 02115
United States, New York
Beth Israel Medical Center
New York, New York, United States, 10003
United States, Ohio
Rainbow Babies & Children's Hospital
Cleveland, Ohio, United States, 44106
Hôpital Universitaire des Enfants Reine Fabiola
Brussels, Belgium
University Hospital Brussels
Brussels, Belgium
University Hospital Leuven
Leuven, Belgium
Hôpital Necker - Enfants Malades
Paris, France
Hôpital des Enfants
Toulouse, France, 31059
Hadassah University Hospital - Mount Scopus
Jerusalem, Israel, 91240
Università La Sapienza
Roma, Italy
Azienda Ospedaliera di Verona
Verona, Italy
Hospital Universitario La Paz
Madrid, Spain
Karolinska University Hospital, Huddinge
Stockholm, Sweden
Sponsors and Collaborators
PTC Therapeutics
Study Director: Joseph McIntosh, MD PTC Therapeutics, Inc.
  More Information

Additional Information:
Responsible Party: PTC Therapeutics Identifier: NCT02107859     History of Changes
Other Study ID Numbers: PTC124-GD-023-CF
2013-005449-35 ( EudraCT Number )
Study First Received: April 4, 2014
Last Updated: May 30, 2017
Individual Participant Data  
Plan to Share IPD: No

Keywords provided by PTC Therapeutics:
Cystic fibrosis
Nonsense mutation
Premature stop codon

Additional relevant MeSH terms:
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases processed this record on June 23, 2017