MedDataX Logo

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

NCT ID: NCT01614457

Last Updated: 2015-02-12

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

70 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-07-31

Study Completion Date

2013-10-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have a Non-G551D CF Transmembrane Conductance Regulator (CFTR) Gating Mutation

NCT01614470

Cystic Fibrosis
COMPLETED PHASE3

A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have an Ivacaftor-Responsive CFTR Mutation

NCT02725567

Cystic Fibrosis
COMPLETED PHASE3

Study of Ivacaftor in Cystic Fibrosis Subjects 2 Through 5 Years of Age With a CFTR Gating Mutation

NCT01705145

Cystic Fibrosis
COMPLETED PHASE3

Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Homozygous for the F508del-CFTR Mutation

NCT00953706

Cystic Fibrosis
TERMINATED PHASE2

Study of Ivacaftor in Cystic Fibrosis Subjects Aged 6 to 11 Years With the G551D Mutation

NCT00909727

Cystic Fibrosis
COMPLETED PHASE3

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Cystic Fibrosis

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Ivacaftor

Ivacaftor 150 milligram (mg) tablet orally twice daily for 24 weeks.

Group Type EXPERIMENTAL

Ivacaftor

Intervention Type DRUG

Ivacaftor 150 milligram (mg) tablet orally twice daily for 24 weeks.

Placebo

Placebo matched to Ivacaftor tablet orally twice daily for 24 weeks.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo matched to Ivacaftor tablet orally twice daily for 24 weeks.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Ivacaftor

Ivacaftor 150 milligram (mg) tablet orally twice daily for 24 weeks.

Intervention Type DRUG

Placebo

Placebo matched to Ivacaftor tablet orally twice daily for 24 weeks.

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

Kalydeco VX-770

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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
Minimum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Cystic Fibrosis Foundation

OTHER

Sponsor Role collaborator

Vertex Pharmaceuticals Incorporated

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Richard Moss, MD

Role: PRINCIPAL_INVESTIGATOR

Stanford University

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Birmingham, Alabama, United States

Site Status

Palo Alto, California, United States

Site Status

Hartford, Connecticut, United States

Site Status

Tampa, Florida, United States

Site Status

Chicago, Illinois, United States

Site Status

Iowa City, Iowa, United States

Site Status

Lexington, Kentucky, United States

Site Status

Baltimore, Maryland, United States

Site Status

Boston, Massachusetts, United States

Site Status

Detroit, Michigan, United States

Site Status

Grand Rapids, Michigan, United States

Site Status

St Louis, Missouri, United States

Site Status

Omaha, Nebraska, United States

Site Status

New York, New York, United States

Site Status

Syracuse, New York, United States

Site Status

Valhalla, New York, United States

Site Status

Chapel Hill, North Carolina, United States

Site Status

Philadelphia, Pennsylvania, United States

Site Status

Pittsburgh, Pennsylvania, United States

Site Status

Charleston, South Carolina, United States

Site Status

Nashville, Tennessee, United States

Site Status

Dallas, Texas, United States

Site Status

Fort Worth, Texas, United States

Site Status

Houston, Texas, United States

Site Status

Salt Lake City, Utah, United States

Site Status

Richmond, Virginia, United States

Site Status

Seattle, Washington, United States

Site Status

Morgantown, West Virginia, United States

Site Status

Madison, Wisconsin, United States

Site Status

Belfast, , United Kingdom

Site Status

Edinburgh, , United Kingdom

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States United Kingdom

References

Explore related publications, articles, or registry entries linked to this study.

Moss RB, Flume PA, Elborn JS, Cooke J, Rowe SM, McColley SA, Rubenstein RC, Higgins M; VX11-770-110 (KONDUCT) Study Group. Efficacy and safety of ivacaftor in patients with cystic fibrosis who have an Arg117His-CFTR mutation: a double-blind, randomised controlled trial. Lancet Respir Med. 2015 Jul;3(7):524-33. doi: 10.1016/S2213-2600(15)00201-5. Epub 2015 Jun 9.

Reference Type DERIVED
PMID: 26070913 (View on PubMed)

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

VX11-770-110

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

Roll-Over Study of Ivacaftor in Cystic Fibrosis Pediatric Subjects With a CF Transmembrane Conductance Regulator Gene (CFTR) Gating Mutation
NCT01946412 COMPLETED PHASE3
Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older With the G551D Mutation
NCT00909532 COMPLETED PHASE3
Safety Study of Ivacaftor in Subjects With Cystic Fibrosis
NCT00457821 COMPLETED PHASE2
A Study to Evaluate Efficacy and Safety of Ivacaftor in Subjects With Cystic Fibrosis Aged 3 Through 5 Years Who Have a Specified CFTR Gating Mutation
NCT02742519 TERMINATED PHASE3
A Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects With CF, Homozygous for the F508del-CFTR Mutation
NCT02514473 COMPLETED PHASE3
Pilot Study Testing the Effect of Ivacaftor on Lung Function in Subjects With Cystic Fibrosis and Residual CFTR Function
NCT01685801 COMPLETED PHASE2
A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor
NCT02347657 COMPLETED PHASE3
Study of the Effect of Ivacaftor on Lung Clearance Index in Subjects With Cystic Fibrosis and the G551D Mutation
NCT01262352 COMPLETED PHASE2
Study to Evaluate Safety and Efficacy of VX-661 in Combination With Ivacaftor in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation With an Open-Label Expansion
NCT02070744 COMPLETED PHASE2
Rollover Study of Ivacaftor in Subjects With Cystic Fibrosis and a Non G551D CFTR Mutation
NCT01707290 COMPLETED PHASE3
A Study to Evaluate the Safety of Long-term Ivacaftor Treatment in Participants With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation
NCT03277196 COMPLETED PHASE3
A Study to Evaluate Efficacy of Ivacaftor in Subjects With Cystic Fibrosis Who Have a 3849 + 10KB C→T or D1152H CFTR Mutation
NCT03068312 COMPLETED PHASE3
A Phase 3 Study to Evaluate the Efficacy and Safety of Ivacaftor and VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Mutation
NCT02392234 COMPLETED PHASE3
A Phase 2 Study to Evaluate Effects of VX-661/Ivacaftor on Lung and Extrapulmonary Systems in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
NCT02508207 COMPLETED PHASE2
A Study of the Effects of Lumacaftor/Ivacaftor (LUM/IVA) on Exercise Tolerance in Subjects With Cystic Fibrosis (CF), Homozygous for the F508del-CFTR Mutation
NCT02875366 COMPLETED PHASE4
Study of VX-661 Alone and in Combination With Ivacaftor in Subjects Homozygous or Heterozygous to the F508del-Cystic Fibrosis Transmembrane Conductance Regulator(CFTR) Mutation
NCT01531673 COMPLETED PHASE2
A Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation
NCT02516410 COMPLETED PHASE3
Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Participants 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del
NCT03601637 COMPLETED PHASE3
A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation
NCT01807923 COMPLETED PHASE3
Short Term Effects of Ivacaftor in Non-G551D Cystic Fibrosis Patients
NCT01784419 COMPLETED NA
Study of Lumacaftor in Combination With Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
NCT01897233 COMPLETED PHASE3
A Study to Confirm the Long-term Safety and Effectiveness of Kalydeco in Patients With Cystic Fibrosis Who Have an R117H-CFTR Mutation, Including Pediatric Patients
NCT02722057 COMPLETED
A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation
NCT01807949 COMPLETED PHASE3
An Efficacy and Safety Study of Ivacaftor in Patients With Cystic Fibrosis and Two Splicing Mutations
NCT02934698 COMPLETED PHASE3
Ivacaftor Treatment in 4 Month to 2 Year Old CF Subjects
NCT03783286 COMPLETED