Overview

This trial is active, not recruiting.

Condition cystic fibrosis
Treatment ataluren (ptc124)
Phase phase 3
Sponsor PTC Therapeutics
Start date May 2014
End date June 2019
Trial size 70 participants
Trial identifier NCT02107859, 2013-005449-35, PTC124-GD-023-CF

Summary

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).

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
Ataluren (PTC124)
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

Primary Outcomes

Measure
Adverse events and laboratory abnormalities
time frame: 192 weeks

Secondary Outcomes

Measure
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
Physical Examination
time frame: 192 Weeks

Eligibility Criteria

Male or female participants at least 6 years old.

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) - 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

Additional Information

Official title An Open-Label Safety and Efficacy Study for Patients With Nonsense Mutation Cystic Fibrosis Previously Treated With Ataluren (PTC124)
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.
Trial information was received from ClinicalTrials.gov and was last updated in May 2016.
Information provided to ClinicalTrials.gov by PTC Therapeutics.