Rollover Study of Ivacaftor in Subjects With Cystic Fibrosis and a Non G551D CFTR Mutation (KONTINUE)
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)or 113 (NCT01685801).
|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|
- 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.
- 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 ]
|Study Start Date:||February 2013|
|Estimated Study Completion Date:||July 2016|
|Estimated Primary Completion Date:||June 2016 (Final data collection date for primary outcome measure)|
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
150 mg tablet, oral use, every 12 hours (q12h)
No Intervention: Observational
Day 1 assessments, Long-term Follow-up
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.
|Contact: Medical Monitorfirstname.lastname@example.org|
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|Principal Investigator:||Joseph Pilewski, MD||Children's Hospital of Pittsburgh|