Rollover Study of Ivacaftor in Subjects With Cystic Fibrosis and a Non-G551D CFTR Mutatio

Phase 1

Conditions: Cystic Fibrosis
MedDRA version: 17.0Level: 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: EUCTR2012-000389-39-BE

Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 120

Inclusion Criteria

- Subjects entering the ivacaftor arm must have completed the assigned study drug treatment duration in the previous study (Study 110 or Study 111), must have completed the previous study but do not wish to enroll in the ivacaftor arm, or must have completed the previous study but do not meet the inclusion criteria of the ivacaftor arm.
- Subjects entering the observational arm must have completed at least
4 weeks of study drug treatment in their previous study (Study 110 or Study 111)
- Females of childbearing potential entering the ivacaftor arm must not be pregnant
- Subjects entering the ivacaftor arm must be willing to comply with contraception requirements

Are the trial subjects under 18? yes
Number of subjects for this age range: 31
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 87
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Ivacaftor Arm Only:

- 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
- Use of any inhibitors or inducers of cytochrome P450 (CYP) 3A
- Evidence of cataract or lens opacity at or before the Day 1 Visit

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 safety of long-term ivacaftor treatment in subjects with<br>cystic fibrosis (CF);Secondary Objective: - To evaluate the efficacy of long-term ivacaftor treatment in subjects<br>with CF<br>- To evaluate the post treatment safety of ivacaftor in subjects with CF who enrolled in the observational arm;Primary end point(s): Safety;Timepoint(s) of evaluation of this end point: Through Week 60

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): - Absolute change from baseline in percent predicted forced expiratory<br>volume in 1 second (FEV1)- 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>- Pulmonary exacerbation;Timepoint(s) of evaluation of this end point: Through Week 60