Overview

Study of Ataluren (PTC124) in Cystic Fibrosis

Status:
Terminated
Trial end date:
2017-06-05
Target enrollment:
0
Participant gender:
All
Summary
The primary objective of this study is to determine the long-term safety and tolerability of ataluren in participants with nonsense mutation cystic fibrosis (nmCF) who completed participation in the double-blind study PTC124-GD-009-CF (NCT00803205), 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 forced expiratory volume in 1 second (FEV1) and pulmonary exacerbation rate, and other safety parameters (for example, 12-lead electrocardiogram [ECG] measurements, vital signs).
Phase:
Phase 3
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
PTC Therapeutics
Criteria
Key Inclusion Criteria:

- Ability to provide written informed consent (parental/guardian consent and participant
assent if less than [<] 18 years of age).

- Evidence of completed participation in the double-blind study, PTC124-GD-009-CF (Study
009).

- Body weight greater than or equal to (≥) 16 kilograms (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 participants 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
randomization.

- 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 and randomization.

- Known hypersensitivity to any of the ingredients or excipients of the study drug.

- Exposure to another investigational drug within 4 weeks prior to screening.

- 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 human
immunodeficiency virus (HIV) test.

- Known portal hypertension.

- Pregnancy or breast-feeding.