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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT02456103 |
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Date of registration:
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26/05/2015 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis
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Scientific title:
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Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Cystic Fibrosis |
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Date of first enrolment:
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August 31, 2015 |
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Target sample size:
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246 |
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Recruitment status: |
Terminated |
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URL:
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https://clinicaltrials.gov/show/NCT02456103 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 3
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Countries of recruitment
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Argentina
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Australia
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Belgium
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Brazil
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Bulgaria
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Canada
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France
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Germany
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Greece
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Israel
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Italy
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Netherlands
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Poland
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Spain
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United Kingdom
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United States
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Contacts
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Name:
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Joseph McIntosh, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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PTC Therapeutics |
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Key inclusion & exclusion criteria
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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.
Age minimum:
6 Years
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Cystic Fibrosis
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Intervention(s)
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Drug: Ataluren
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Primary Outcome(s)
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Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Serum Biochemistry, Hematology, and Urinalysis) Parameter
[Time Frame: Baseline up to Week 100]
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Number of Participants With Treatment Emergent Adverse Events (TEAEs)
[Time Frame: Baseline up to Week 100]
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Secondary Outcome(s)
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Rate of Pulmonary Exacerbations as Defined by Modified Fuch's Criteria Over 48 Weeks
[Time Frame: Baseline up to Week 48]
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Change From Baseline in Percent-Predicted of Forced Vital Capacity (FVC) as Measured by Spirometry at Week 24
[Time Frame: Baseline, Week 24]
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Change From Baseline in Forced Expiratory Flow Between 25% and 75% of Expiration (FEF25-75) as Measured by Spirometry at Week 24
[Time Frame: Baseline, Week 24]
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Change From Baseline in Percent-Predicted Forced Expiratory Volume in 1 Second (FEV1) as Measured by Spirometry at Week 24
[Time Frame: Baseline, Week 24]
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Secondary ID(s)
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PTC124-GD-021e-CF
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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