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Rollover Study of Ivacaftor in Subjects With Cystic Fibrosis and a Non G551D CFTR Mutation (KONTINUE)

This study is currently recruiting participants.
Verified April 2013 by Vertex Pharmaceuticals Incorporated
Sponsor:
Collaborator:
Cystic Fibrosis Foundation
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:
NCT01707290
First received: October 9, 2012
Last updated: April 30, 2013
Last verified: April 2013
History of Changes
  Purpose

The purpose of this study is to evaluate the safety of long-term ivacaftor treatment in subjects with cystic fibrosis (CF) from Studies 110 (NCT01614457) or 111 (NCT01614470).


Condition Intervention Phase
Cystic Fibrosis
 Drug: ivacaftor
 Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official 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

Resource links provided by NLM:

MedlinePlus related topics: Cystic Fibrosis
Drug Information available for: Ivacaftor
U.S. FDA Resources

Further study details as provided by Vertex Pharmaceuticals Incorporated:

Primary Outcome Measures:
  • Safety [ Time Frame: Up to 104 weeks ] [ Designated as safety issue: Yes ]
    Subjective reporting of adverse events, clinical laboratory values (serum chemistry and hematology), electrocardiograms (ECGs), ophthalmologic examinations, and vital signs.


Secondary Outcome Measures:
  • Absolute change from baseline in percent predicted forced expiratory volume in 1 second (FEV1) [ Time Frame: Through Week 104 ] [ Designated as safety issue: No ]
  • Change from baseline in body mass index (BMI) [ Time Frame: At Week 104 ] [ Designated as safety issue: No ]
  • Change from baseline in sweat chloride [ Time Frame: Through Week 104 ] [ Designated as safety issue: No ]
  • Change from baseline in the respiratory domain of the Cystic Fibrosis Questionnaire Revised (CFQ R) [ Time Frame: Through Week 104 ] [ Designated as safety issue: No ]
  • Pulmonary exacerbation [ Time Frame: Up to 104 weeks ] [ Designated as safety issue: No ]

Estimated Enrollment: 60
Study Start Date: February 2013
Estimated Study Completion Date: July 2016
Estimated Primary Completion Date: July 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Ivacaftor
150 mg, oral use, every 12 hours (q12h)
 Drug: ivacaftor
150 mg tablet, oral use, every 12 hours (q12h)
Other Names:
  • Kalydeco
  • VX-770
No Intervention: Observational
Day 1 assessments, Long-term Follow-up

Detailed Description:

Ivacaftor is the first 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:

  • Subjects entering the ivacaftor arm must have completed the assigned study drug treatment duration in the previous study (Study 110 or Study 111)
  • 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), 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.
  • 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

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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01707290

Contacts
Contact: Medical Monitor 617.341.6777 medicalinfo@vrtx.com

  Show 45 Study Locations
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
Cystic Fibrosis Foundation
Investigators
Principal Investigator: Joseph Pilewski, MD Children's Hospital of Pittsburgh
  More Information

No publications provided

Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT01707290     History of Changes
Other Study ID Numbers: VX12-770-112
Study First Received: October 9, 2012
Last Updated: April 30, 2013
Health Authority: United States: Food and Drug Administration
France: Agence Nationale de Sécurité du Médicament et des produits de santé
Belgium: Federal Agency for Medicinal Products and Health Products
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 June 18, 2013