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

This study has been completed.
Sponsor:
Collaborator:
Cystic Fibrosis Foundation
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:
NCT01614457
First received: June 5, 2012
Last updated: January 7, 2014
Last verified: January 2014
  Purpose

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.


Condition Intervention Phase
Cystic Fibrosis
Drug: Ivacaftor
Drug: Placebo
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official 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

Resource links provided by NLM:


Further study details as provided by Vertex Pharmaceuticals Incorporated:

Primary Outcome Measures:
  • Absolute change from baseline in percent predicted forced expiratory volume in 1 second (FEV1) [ Time Frame: Through Week 24 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Change from baseline in body mass index (BMI) [ Time Frame: At Week 24 ] [ Designated as safety issue: No ]
  • Change from baseline in sweat chloride [ Time Frame: Through Week 24 ] [ Designated as safety issue: No ]
  • Change from baseline in the respiratory domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) [ Time Frame: Through Week 24 ] [ Designated as safety issue: No ]
  • Time to first pulmonary exacerbation [ Time Frame: Through Week 24 ] [ Designated as safety issue: No ]

Enrollment: 70
Study Start Date: June 2012
Study Completion Date: October 2013
Primary Completion Date: October 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: Ivacaftor
Ivacaftor treatment for 24 weeks
Drug: Ivacaftor
150 mg, oral use, administered twice a day (q12h)
Other Names:
  • Kalydeco
  • VX-770
Placebo Comparator: Placebo
Placebo for 24 weeks
Drug: Placebo
oral use, administered twice a day (q12h)

Detailed Description:

Ivacaftor is the first cystic fibrosis transmembrane regulator (CFTR) modulator to show an improvement in CFTR function and clinical benefit in patients with CF. Results from Phase 3 studies (VX08-770-102 [Study 102] and VX08-770-103 [Study 103]) showed that ivacaftor is effective in the treatment of patients 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 patients 6 years of age and older who have a G551D mutation in the CFTR gene.

  Eligibility

Ages Eligible for Study:   6 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male or female with confirmed diagnosis of CF
  • Must have at least 1 allele of the R117H CFTR mutation
  • FEV1 40% 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 kg at screening
  • Females of childbearing potential must not be pregnant
  • Willing to comply with contraception requirements

Exclusion Criteria:

  • Any 1 of the following CFTR gating mutations: G551D, G178R, S549N, S549R, G551S, G970R, G1244E, S1251N, S1255P, 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
  • 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
  • Use of any inhibitors or inducers of cytochrome P450 (CYP) 3A
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01614457

  Show 35 Study Locations
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
Cystic Fibrosis Foundation
Investigators
Principal Investigator: Richard Moss, MD Stanford University
  More Information

No publications provided

Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT01614457     History of Changes
Other Study ID Numbers: VX11-770-110
Study First Received: June 5, 2012
Last Updated: January 7, 2014
Health Authority: United States: Food and Drug Administration
United Kingdom: Medicines and Healthcare Products Regulatory Agency

Additional relevant MeSH terms:
Cystic Fibrosis
Fibrosis
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Pathologic Processes

ClinicalTrials.gov processed this record on April 16, 2014