Study of Ivacaftor in Subjects With Cystic Fibrosis Who Have a Non-G551D CFTR Gating Mutatio

Phase 1

Active, not recruiting

Conditions: Cystic Fibrosis
MedDRA version: 14.1Level: PTClassification code 10011762Term: Cystic fibrosisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Registration Number: EUCTR2012-000388-26-BE

Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Not Recruiting

Sex: All

Target Recruitment: 40

Inclusion Criteria

- At least 1 allele of the following CFTR gating mutations: G178R, S549N, S549R, G551S, G970R, G1244E, S1251N, S1255P, G1349D
- FEV1 =40% predicted normal for age, sex, and height
- 6 years of age or older
- Minimum weight of 15 kg at screening
- Females of childbearing potential must not be pregnant
- Willing to comply with contraception requirements

Are the trial subjects under 18? yes
Number of subjects for this age range: 7
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 3
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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 P450 (CYP) 3A
- Evidence of cataract or lens opacity at screening.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the efficacy of ivacaftor in subjects with cystic fibrosis (CF) who have a non-G551D CFTR gating mutation;Secondary Objective: - To evaluate the safety of ivacaftor in subjects with CF who have a non-G551D CFTR<br>gating mutation<br>- To evaluate the durability of efficacy of ivacaftor in subjects with CF who have a<br>non-G551D CFTR gating mutation;Primary end point(s): Absolute change from baseline in percent predicted forced expiratory volume in 1 second (FEV1);Timepoint(s) of evaluation of this end point: Through 8 weeks and 24 weeks of treatment

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): - Change from baseline in body mass index (BMI) <br>- Change from baseline in sweat chloride<br>- Change from baseline in the respiratory domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) <br>- Safety, as determined by adverse events, clinical laboratory values (serum chemistry, hematology, and coagulation), ophthalmologic examinations, ECGs, and vital signs;Timepoint(s) of evaluation of this end point: Through 8 weeks and 24 weeks of treatment and at week 36