A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have an Ivacaftor-Responsive CFTR Mutation

• Register: ClinicalTrials.gov
• Last refreshed on: 9 January 2023
• Main ID: NCT02725567
• Date of registration: 14/03/2016
• Prospective Registration: No
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have an Ivacaftor-Responsive CFTR Mutation
• Scientific title: A Phase 3, 2 Part, Open-Label Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have an Ivacaftor-Responsive CFTR Mutation
• Date of first enrolment: March 2016
• Target sample size: 56
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02725567
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Australia, Canada, Ireland, United Kingdom, United States

Key inclusion & exclusion criteria

Inclusion Criteria:

- Confirmed diagnosis of CF by sweat chloride value or CF mutation criteria.

- Have 1 of the following 10 CFTR mutations on at least 1 allele: G551D, G178R, S549N,
S549R, G551S, G1244E, S1251N, S1255P, G1349D or R117H (eligible in regions where
ivacaftor is approved for use). Part A/B group may also have other
ivacaftor-responsive mutations.

- Hematology, serum chemistry, and vital signs results at screening with no clinically
significant abnormalities that would interfere with the study assessments, as judged
by the investigator.

Exclusion Criteria:

- History of any illness or condition that, in the opinion of the investigator, might
confound the results of the study or pose an additional risk in administering study
drug to the subject

- Colonization with organisms associated with a more rapid decline in pulmonary status
at screening (Only for Parts A and B)

- History of abnormal liver function or abnormal liver function at screening

- History of solid organ or hematological transplantation

- Use of any moderate or strong inducers or inhibitors of cytochrome P450 (CYP) 3A
within 2 weeks before Day 1

- Participation in a clinical study involving administration of either an
investigational or a marketed drug within 30 days or 5 terminal half-lives before
screening

- Hemoglobin (Hgb) <9.5 g/dL at screening

- Chronic kidney disease of Stage 3 or above

- Presence of a non-congenital or progressive lens opacity or cataract at Screening

Other protocol defined Inclusion/Exclusion Criteria may apply.



Age minimum: 0 Months
Age maximum: 24 Months
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: ivacaftor

Primary Outcome(s)

Part A/B: Trough concentrations (Ctrough) of ivacaftor, M1 ivacaftor, and M6 ivacaftor [Time Frame: after 4 days of ivacaftor treatment]

Part B: Safety, as determined by number of subjects with adverse events (AEs), clinically relevant abnormal laboratory values (serum chemistry and hematology), standard 12 lead electrocardiograms (ECGs), vital signs, and ophthalmologic examinations [Time Frame: Day 1 up to Week 24]

Part A: Safety, as determined by number of subjects with adverse events (AEs), clinically relevant abnormal laboratory values (serum chemistry and hematology), standard 12 lead electrocardiograms (ECGs), vital signs, and ophthalmologic examinations [Time Frame: Day 1 up to Day 70]

Part A: Peak concentrations (C3-6h) of ivacaftor, M1 ivacaftor, and M6 ivacaftor [Time Frame: after 4 days of ivacaftor treatment]

Part A/B: Safety, as determined by number of subjects with adverse events (AEs), clinically relevant abnormal laboratory values (serum chemistry and hematology), standard 12 lead electrocardiograms (ECGs), vital signs, and ophthalmologic examinations [Time Frame: Day 1 up to Week 24]

Part A: Trough concentrations (Ctrough) of ivacaftor, M1 ivacaftor, and M6 ivacaftor [Time Frame: after 4 days of ivacaftor treatment]

Secondary Outcome(s)

Part B: Peak concentrations (C3-6h) of ivacaftor, M1 ivacaftor, and M6 ivacaftor [Time Frame: through Week 24]

Part A/B: Absolute change from baseline in sweat chloride using quantitative pilocarpine iontophoresis [Time Frame: up to Week 24]

Part B: Absolute change from baseline in sweat chloride using quantitative pilocarpine iontophoresis [Time Frame: up to Week 24]

Part B: Trough concentrations (Ctrough) of ivacaftor, M1 ivacaftor, and M6 ivacaftor [Time Frame: through Week 24]

Secondary ID(s)

2015-001997-16

VX15-770-124

Source(s) of Monetary Support

Please refer to primary and secondary sponsors