Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have the R117H... | Completed
Study of Ivacaftor in Subjects With... | Completed
Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have the R117H-CF Transmembrane Conductance Regulator (CFTR) Mutation (KONDUCT)
KONDUCT
Trial Source

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Medical Conditions
  • Cystic Fibrosis
Unfortunately contact details are not available for this trial.
Primary Contact Details
Completed
Recruitment Status
NCT01614457
Primary Trial ID Number

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Summary
The purpose of this study is to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis (CF) who have the R117H-CFTR mutation.
Research Details
  • Ivacaftor is the first CFTR modulator to show an improvement in CFTR function and clinical benefit in subjects with CF. Results from Phase 3 studies (VX08-770-102 [Study 102] [NCT00909532] and VX08-770-103 [Study 103] [NCT00909727]) showed that ivacaftor is effective in the treatment of subjects with CF who have the G551D-CFTR mutation, as evidenced by sustained improvements in CFTR channel function (measured by reduction in sweat chloride concentration) and corresponding substantial, durable improvements in lung function, pulmonary exacerbations, respiratory symptoms, and weight gain. Ivacaftor was also well tolerated, as evidenced by the rates and reasons for premature discontinuation and results of safety assessments.

    Ivacaftor (Trade Name Kalydeco; 150 mg tablets) was initially approved in the United States for the treatment of CF in subjects 6 years of age and older who have a G551D mutation in the CFTR gene.
Phase
Phase 3
Study Design
Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
Study Type
Interventional
Intervention
Drug : Ivacaftor, Drug : Placebo

Study Arm Groups : Ivacaftor, Placebo

Intervention Type
See Interventions above
Primary Outcome Measures
    Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 24; Baseline, Week 24
Secondary Outcome Measures
    Change From Baseline in Body Mass Index (BMI) at Week 24; Baseline, Week 24; Change From Baseline in Sweat Chloride Through Week 24; Baseline, Week 24; Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 24; Baseline, Week 24; Time to First Pulmonary Exacerbation; Day 0 to 15, Day 16 to 56, Day 57 to 112, Day 113 to 168; Number of Subjects With Adverse Events (AEs) and Serious Adverse Events (SAEs); Baseline up to follow-up (3 to 4 weeks after last dose [last dose = Week 24])
Publication(s)
Sorry, this information is not available
Result Reports
Check availability of results on the Clinicaltrials.gov website
This information is designed to help you decide whether this trial is of interest. In some cases it is provided as a link to more detailed patient information or it may still be awaited from the organisation running the trial. Please look again shortly if the information you need is not here or, if named, contact the researcher named above.
Gender
Both
Age Range
6 Years - N/A
Who Can Participate
Patients
Number of Participants
Sorry, this information is not available
Participant Inclusion Criteria
    Inclusion Criteria:

    - Male or female with confirmed diagnosis of CF

    - Must have at least 1 allele of the R117H CFTR mutation

    - Percent predicted forced expiratory volume in 1 second (FEV1) 40 percent (%) to 90% (for subjects aged 12 years or older) or 40% to 105% (for subjects aged 6 to 11 years) 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:

    - CFTR gene mutation leading to CFTR channel with gating defect (that is, any 1 of the following mutations: G551D, G178R, G551S, S549N, S549R, G970R, G1244E, S1251N, S1255P, or G1349D)

    - 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

    - Abnormal liver function, at screening, defined as greater than or equal to (>=) 3 time upper limit of normal (ULN), of any 3 or more of the following: serum aspartate transaminase (AST), serum alanine transaminase (ALT), gamma-glutamyl transpeptidase (GGT), serum alkaline phosphatase (ALP), total bilirubin

    - Colonization with organisms associated with a more rapid decline in pulmonary status (for example, Burkholderia cenocepacia, Burkholderia dolosa, and Mycobacterium abscessus) at screening

    - 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

    - Any "non-CF-related" illness within 2 weeks before Day 1 (first dose of study drug). "Illness" was defined as an acute (serious or non-serious) condition (for example, gastroenteritis)

    - Use of any inhibitors or inducers of cytochrome (CYP) P450 3A
Participant Exclusion Criteria
This is in the inclusion criteria above
This information is designed to help you decide whether this trial is of interest. In some cases it is provided as a link to more detailed patient information or it may still be awaited from the organisation running the trial. Please look again shortly if the information you need is not here or, if named, contact the researcher named above.
Trial Location(s)
Belfast
Edinburgh
Trial Contact(s)
Primary Trial Contact
Sorry, this information is not available
Other Trial Contacts
Sorry, this information is not available
Countries Recruiting
United Kingdom, United States
This information is designed to help you decide whether this trial is of interest. In some cases it is provided as a link to more detailed patient information or it may still be awaited from the organisation running the trial. Please look again shortly if the information you need is not here or, if named, contact the researcher named above.
Scientific Title
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Ivacaftor in Subjects With Cystic Fibrosis Who Have the R117H-CFTR Mutation
EudraCT Number
Not available for this trial
Funder(s)
  • Cystic Fibrosis Foundation Therapeutics
Other Study ID Numbers
VX11-770-110
Sponsor(s)
Vertex Pharmaceuticals Incorporated
Key Dates

Recruitment Start Date

Jul 2012

Recruitment End Date

Oct 2013

Trial Start Date
Date Not Available
Trial End Date
Date Not Available
Date Assigned

05 Jun 2012

Last Updated

31 Jan 2015