Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have a Non-G551D CF Transmembrane Conductance Regulator (CFTR) Gating Mutation

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT01614470
• Date of registration: 05/06/2012
• Prospective Registration: Yes
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have a Non-G551D CF Transmembrane Conductance Regulator (CFTR) Gating Mutation KONNECTION
• Scientific title: A Phase 3, Two-Part, Randomized, Double-Blind, Placebo-Controlled, Crossover Study With an Open-Label Period to Evaluate the Efficacy and Safety of Ivacaftor in Subjects With Cystic Fibrosis Who Have a Non-G551D CFTR Gating Mutation
• Date of first enrolment: July 2012
• Target sample size: 39
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01614470
• Study type: Interventional
• Phase: Phase 3

Countries of recruitment

Belgium, France, United States

Contacts

• Name: Christine De Boeck, MD, PhD
• Affiliation: University of Leuven

Key inclusion & exclusion criteria

Inclusion Criteria:

- Male or female with confirmed diagnosis of CF

- At least 1 allele of the following CFTR gating mutations: G178R, S549N, S549R, G551S,
G970R, G1244E, S1251N, S1255P, G1349D

- Percent predicted forced expiratory volume in 1 second (FEV1) greater than or equal to
(>=) 40 percent (%) predicted normal for age, sex, and height

- 6 years of age or older

- Minimum weight of 15 kilogram (kg) at screening

- Females of childbearing potential must not be pregnant

- Willing to comply with contraception requirements

Exclusion Criteria:

- G551D-CFTR mutation on at least 1 allele

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

- An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in
therapy (including antibiotics) for pulmonary disease within 4 weeks before the first
dose of study drug

- History of solid organ or hematological transplantation

- History of alcohol, medication or illicit drug abuse within 1 year before the first
dose of study drug

- Ongoing participation in another therapeutic clinical study or prior participation in
an investigational drug study within 30 days before screening

- Use of inhaled hypertonic saline treatment

- Use of any inhibitors or inducers of cytochrome (CYP) P450 3A

- Evidence of cataract or lens opacity at screening



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

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: Ivacaftor

Drug: Placebo

Primary Outcome(s)

Part 2: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through 24 Weeks of Treatment (Week 36 Visit) [Time Frame: Baseline (pre-dose Week 12), Week 36]

Part 1: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 8 [Time Frame: Part 1: Baseline (pre-dose Day 1), Week 8]

Secondary Outcome(s)

Part 1: Change From Baseline in Sweat Chloride Through Week 8 [Time Frame: Part 1: Baseline (pre-dose Day 1), Week 8]

Part 2: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [Time Frame: Part 2: Week 20 up to Week 40]

Part 1: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [Time Frame: Part 1: From signing of informed consent up to Week 20]

Part 2: Change From Baseline in Body Mass Index (BMI) at 24 Weeks of Treatment (Week 36 Visit) [Time Frame: Baseline (pre-dose Week 12), Week 36]

Part 2: Change From Baseline in Sweat Chloride Through 24 Weeks of Treatment (Week 36 Visit) [Time Frame: Baseline (pre-dose Week 12), Week 36]

Part 1: Change From Baseline in Body Mass Index (BMI) at Week 8 [Time Frame: Part 1: Baseline (pre-dose Day 1), Week 8]

Part 1: Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 8 [Time Frame: Part 1: Baseline (pre-dose Day 1), Week 8]

Part 2: Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through 24 Weeks of Treatment (Week 36 Visit) [Time Frame: Baseline (pre-dose Week 12), Week 36]

Secondary ID(s)

VX12-770-111

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Cystic Fibrosis Foundation Therapeutics

Results

• Results available: Yes
• Date Posted: 29/10/2014
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01614470