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

A Phase 3, Two-Arm, Rollover Study to Evaluate the Safety of Long-Term Ivacaftor Treatment in Subjects 6 Years of Age and Older with Cystic Fibrosis and a Non-G551D CFTR Mutation

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2012-000389-39-GB

Enrollment

120

Registered

2012-04-20

Start date

2012-10-25

Completion date

Unknown

Last updated

2019-06-30

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

cystic fibrosis MedDRA version: 18.0 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Trade Name: Kalydeco Product Name: ivacaftor Product Code: VX-770 Pharmaceutical Form: Film-coated tablet INN or Proposed INN: IVACAFTOR

Eligibility

Sex/Gender

All

Inclusion criteria

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

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

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate the safety of long-term ivacaftor treatment in subjects with cystic fibrosis (CF); Secondary Objective: - To evaluate the efficacy of long-term ivacaftor treatment in subjects with CF - 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	—

Measure

Time frame

Main Objective: To evaluate the safety of long-term ivacaftor treatment in subjects with cystic fibrosis (CF); Secondary Objective: - To evaluate the efficacy of long-term ivacaftor treatment in subjects with CF - 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

Measure	Time frame
Timepoint(s) of evaluation of this end point: Through Week 60; Secondary end point(s): - Absolute change from baseline in percent predicted forced expiratory volume in 1 second (FEV1) - Change from baseline in body mass index (BMI) - Change from baseline in sweat chloride - Change from baseline in the respiratory domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) - Pulmonary exacerbation	—

Countries

Belgium, France, United Kingdom, United States

Contacts

Public ContactClinical Trials and Medical Info

Vertex Pharmaceuticals Incorporated

medicalinfo@vrtx.com+1877634 8789

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026