Overview

This trial is active, not recruiting.

Condition cystic fibrosis
Treatments ataluren (ptc124®), placebo
Phase phase 3
Sponsor PTC Therapeutics
Collaborator Cystic Fibrosis Foundation Therapeutics
Start date June 2014
End date November 2016
Trial size 208 participants
Trial identifier NCT02139306, 2013-004581-34, PTC124-GD-021-CF

Summary

This is a Phase 3, international, multicenter, randomized, double-blind, placebo-controlled, efficacy and safety study of ataluren in patients with nonsense mutation cystic fibrosis (nmCF) not receiving chronic inhaled aminoglycosides.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification efficacy study
Intervention model parallel assignment
Masking double blind (subject, caregiver, investigator, outcomes assessor)
Primary purpose treatment
Arm
(Experimental)
Oral powder for suspension taken 3 times per day (10-, 10-, and 20-mg/kg morning, midday and evening, respectively) for 48 weeks
ataluren (ptc124®)
(Placebo Comparator)
Matching placebo taken 3 times per day for 48 weeks
placebo

Primary Outcomes

Measure
FEV1 by spirometry
time frame: 48 weeks

Secondary Outcomes

Measure
Rate of pulmonary exacerbations (modified Fuchs criteria)
time frame: 48 weeks
Respiratory HRQL as assessed by the CFQ-R respiratory domain
time frame: 48 weeks
Body weight and BMI
time frame: 48 weeks

Eligibility Criteria

Male or female participants at least 6 years old.

Inclusion Criteria: - Evidence of signed and dated informed consent/assent document(s) indicating that the subject (and/or his parent/legal guardian) has been informed of all pertinent aspects of the trial - Age >=6 years. - Body weight >=16 kg. - Sweat chloride >60 mEq/L - Documentation of the presence of a nonsense mutation in at least 1 allele of the CFTR gene, as determined by genotyping performed at a laboratory certified by the College of American Pathologists (CAP), or under the Clinical Laboratory Improvement Act/Amendment (CLIA), or by an equivalent organization - Verification that a blood sample has been drawn for sequencing of the CFTR gene - Ability to perform a valid, reproducible spirometry test using the study-specific spirometer with demonstration of an FEV1 >=40% and <=90% of predicted - Demonstration at Visit 2 of a valid %-predicted FEV1 within 15% of the Screening % predicted FEV1 value - Resting oxygen saturation (as measured by pulse oximetry) >=92% on room air. - Confirmed screening laboratory values within pre-specified ranges - In 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, study procedures, laboratory tests, and study restrictions Exclusion Criteria: - Known hypersensitivity to any of the ingredients or excipients of the study drug - Previous participation in the Phase 3 trial of ataluren (PTC124-GD-009-CF). - 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 - Chronic use of inhaled aminoglycosides (eg, tobramycin) or use of inhaled aminoglycosides within 4 months prior to screening. - Exposure to another investigational drug within 4 weeks prior to screening - Ongoing participation in any other therapeutic clinical trial - Evidence of pulmonary exacerbation or acute upper or lower respiratory tract infection (including viral illnesses) within 3 weeks prior to screening - Treatment with intravenous antibiotics within 3 weeks prior to screening - Ongoing immunosuppressive therapy (other than corticosteroids) - Ongoing warfarin, phenytoin, or tolbutamide therapy - History of solid organ or hematological transplantation - Major complications of lung disease (including massive hemoptysis, pneumothorax, or pleural effusion) within 8 weeks prior to screening - Known portal hypertension - Positive hepatitis B surface antigen, hepatitis C antibody test, or human immunodeficiency virus (HIV) test - Pregnancy or breast-feeding - Current smoker or a smoking history of >=10 pack-years (number of cigarette packs/day x number of years smoked). - Prior or ongoing medical condition (eg, concomitant illness, alcoholism, drug abuse, psychiatric condition), medical history, physical findings, ECG findings, or laboratory abnormality that, in the investigator's opinion, could adversely affect the safety of the subject, makes it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results

Additional Information

Official title A Phase 3 Efficacy and Safety Study of Ataluren (PTC124®) in Patients With Nonsense Mutation Cystic Fibrosis
Trial information was received from ClinicalTrials.gov and was last updated in November 2015.
Information provided to ClinicalTrials.gov by PTC Therapeutics.