Study of Ataluren (PTC124) in Cystic Fibrosis

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02107859
• Date of registration: 04/04/2014
• Prospective Registration: Yes
• Primary sponsor: PTC Therapeutics
• Public title: Study of Ataluren (PTC124) in Cystic Fibrosis
• Scientific title: An Open-Label Safety and Efficacy Study for Patients With Nonsense Mutation Cystic Fibrosis Previously Treated With Ataluren (PTC124)
• Date of first enrolment: May 23, 2014
• Target sample size: 61
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT02107859
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Belgium, France, Germany, Israel, Italy, Spain, Sweden, United States

Contacts

• Name: Joseph McIntosh, MD
• Affiliation: PTC Therapeutics, Inc.

Key inclusion & exclusion 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.



Age minimum: 6 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: Ataluren

Primary Outcome(s)

Number of Participants With Clinically Significant Laboratory Abnormalities [Time Frame: Baseline (Day 1) up to end of study (Week 196)]

Number of Participants With Treatment-Emergent Adverse Events (TEAEs) [Time Frame: Baseline (Day 1) up to end of study (Week 196)]

Secondary Outcome(s)

Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at the End of Treatment (Week 192), as Assessed by Spirometry [Time Frame: Baseline, Week 192]

Change From Baseline in Heart Rate at Final Visit (Week 196), as Assessed by 12-Lead ECG [Time Frame: Baseline, Week 196]

Percentage of Participants With Pulmonary Exacerbation, As Assessed by Expanded Fuchs' Criteria [Time Frame: Baseline up to Week 192]

Percentage of Participants With Pulmonary Exacerbation, As Assessed by Classic Fuchs' Criteria [Time Frame: Baseline up to Week 192]

Change From Baseline in 12-Lead Electrocardiogram (ECG) Parameters at Final Visit (Week 196) [Time Frame: Baseline, Week 196]

Change From Baseline in Vital Signs at Final Visit (Week 196) [Time Frame: Baseline, Week 196]

Percentage of Participants With Pulmonary Exacerbation, As Assessed by Modified Fuchs Criteria [Time Frame: Baseline up to Week 192]

Secondary ID(s)

PTC124-GD-023-CF

2013-005449-35

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 25/03/2020
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02107859