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

• Register: EUCTR
• Last refreshed on: 30 June 2019
• Main ID: EUCTR2012-000389-39-GB
• Date of registration: 20/04/2012
• Prospective Registration: Yes
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: Rollover Study of Ivacaftor in Subjects With Cystic Fibrosis and a Non-G551D CFTR Mutation
• Scientific title: 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
• Date of first enrolment: 25/10/2012
• Target sample size: 120
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2012-000389-39
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: no; Open: yes; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: no; Other: no; Number of treatment arms in the trial: 1
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Belgium, France, United Kingdom, United States

Contacts

• Name: Clinical Trials and Medical Info
• Affiliation: Vertex Pharmaceuticals Incorporated

• Name: Clinical Trials and Medical Info
• Affiliation: Vertex Pharmaceuticals Incorporated

Key inclusion & exclusion 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:
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


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

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

Intervention(s)

Trade Name: Kalydeco
Product Name: ivacaftor
Product Code: VX-770
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: IVACAFTOR
CAS Number: 873054-44-5
Current Sponsor code: VX-770
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 150-

Primary Outcome(s)

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.

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

Primary end point(s): Safety

Timepoint(s) of evaluation of this end point: Through Week 60

Secondary Outcome(s)

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

Secondary ID(s)

VX12-770-112

Source(s) of Monetary Support

Vertex Pharmaceuticals Incorporated

Ethics review

Status: Approved
Approval date:
Contact:

Results

• Results available: Yes
• Date Posted: 02/11/2016
• Date Completed: 18/04/2016
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2012-000389-39/results