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Extension Study of Ataluren for Nonsense Mutation Cystic Fibrosis

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ClinicalTrials.gov Identifier: NCT02456103
Recruitment Status : Terminated (CF Data from CF study 021 did not meet end points)
First Posted : May 28, 2015
Last Update Posted : January 28, 2019
Sponsor:
Information provided by (Responsible Party):
PTC Therapeutics

Brief Summary:
This is an open-label extension study for patients who complete a Phase 3, placebo-controlled study of ataluren in patients with nonsense mutation cystic fibrosis (nmCF) not receiving chronic inhaled aminoglycosides.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: Ataluren Phase 3

Detailed Description:
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).

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 246 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Cystic Fibrosis
Actual Study Start Date : July 31, 2015
Actual Primary Completion Date : June 30, 2017
Actual Study Completion Date : June 30, 2017

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arm Intervention/treatment
Experimental: Ataluren
Ataluren will be orally administered at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening for up to 96 weeks.
Drug: Ataluren
Other Names:
  • PTC124
  • Translarna




Primary Outcome Measures :
  1. Safety profile characterized by type, frequency, severity, timing, and relationship to ataluren of any adverse events or laboratory abnormalities [ Time Frame: 96 weeks ]


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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 patient (and/or his 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02456103


  Show 70 Study Locations
Sponsors and Collaborators
PTC Therapeutics
Investigators
Study Director: Joseph McIntosh, MD PTC Therapeutics

Responsible Party: PTC Therapeutics
ClinicalTrials.gov Identifier: NCT02456103     History of Changes
Other Study ID Numbers: PTC124-GD-021e-CF
First Posted: May 28, 2015    Key Record Dates
Last Update Posted: January 28, 2019
Last Verified: January 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Additional relevant MeSH terms:
Fibrosis
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases