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

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborator:
Cystic Fibrosis Foundation
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:
NCT01707290
First received: October 9, 2012
Last updated: October 23, 2014
Last verified: October 2014

October 9, 2012
October 23, 2014
February 2013
June 2016   (final data collection date for primary outcome measure)
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.
Same as current
Complete list of historical versions of study NCT01707290 on ClinicalTrials.gov Archive Site
  • 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 ]
Same as current
Not Provided
Not Provided
 
Rollover Study of Ivacaftor in Subjects With Cystic Fibrosis and a Non G551D CFTR Mutation
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

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), 111 (NCT01614470), and 113 (NCT01685801).

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.

Interventional
Phase 3
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Cystic Fibrosis
Drug: ivacaftor
150 mg tablet, oral use, every 12 hours (q12h)
Other Names:
  • Kalydeco
  • VX-770
  • Experimental: Ivacaftor

    150 mg, oral use, every 12 hours (q12h)

    Subjects in the ivacaftor arm, who have not taken inhaled hypertonic Saline (HS) for at least 4 weeks, are clinically stable and are exacerbation free will have the option to participate in the optional inhaled HS substudy

    Intervention: Drug: ivacaftor
  • No Intervention: Observational
    Day 1 assessments, Long-term Follow-up
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
125
July 2016
June 2016   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • Subjects from Study 110 or Study 111 entering the ivacaftor arm must have completed the assigned study drug treatment duration in the previous study
  • Subjects from Study 113 entering the ivacaftor arm must have completed all study related treatments through the Follow-up Visit and met the Study 113 responder criteria during the previous study.
  • 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 moderate or strong inhibitors or inducers of cytochrome P450 (CYP) 3A
  • Evidence of cataract or lens opacity at or before the Day 1 Visit
Both
6 Years and older
No
Contact information is only displayed when the study is recruiting subjects
United States,   United Kingdom,   Belgium,   France
 
NCT01707290
VX12-770-112
No
Vertex Pharmaceuticals Incorporated
Vertex Pharmaceuticals Incorporated
Cystic Fibrosis Foundation
Principal Investigator: Joseph Pilewski, MD Children's Hospital of Pittsburgh
Vertex Pharmaceuticals Incorporated
October 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP