Study of Ivacaftor in Cystic Fibrosis Subjects 2 Through 5 Years of Age With a CFTR Gating Mutation

Vertex Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Cystic Fibrosis

Treatments: Drug: Ivacaftor

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT01705145

KIWI

VX11-770-108

Details and patient eligibility

About

The purpose of this study is to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD), of ivacaftor in children with cystic fibrosis (CF) who are 2 through 5 years of age and have a CF Transmembrane Conductance Regulator (CFTR) gating mutation in at least 1 allele.

Part A is designed to evaluate the safety and PK of multiple-dose administration of ivacaftor in participants 2 through 5 years of age and to confirm the doses for Part B. Part B is designed to evaluate the safety, PK, PD, and efficacy of ivacaftor in participants 2 through 5 years of age.

Enrollment: 35 patients

Sex: All

Ages: 2 to 5 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Male or female with confirmed diagnosis of CF
• Must have a CFTR gating mutation in at least 1 allele
• Aged 2 through 5 years at screening and Day 1
• Weight >= 8 kg at screening and Day 1
• Hematology, serum chemistry, coagulation, 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 participant
• An acute upper or lower respiratory infection, or pulmonary exacerbation, or changes in therapy for pulmonary disease within 4 weeks before Day 1
• 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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35 participants in 1 patient group

Ivacaftor

Experimental group

Description:

Part A: Ivacaftor 50 milligram (mg) (for participants weighing less than \[\<\] 14 kilograms \[kg\]) or 75 mg (for participants weighing greater than or equal to \[\>=\] 14 kg) every 12 hours (q12h) from Day 1 through Day 3 and 1 morning dose on Day 4 during Part A of the study.. Part B: Ivacaftor 50 mg (for participants weighing \<14 kg) or 75 mg (for participants weighing \>=14 kg) q12h for 24 weeks during Part B of the study.

Treatment:

Drug: Ivacaftor