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

Cystic Fibrosis Clinical Trial

— KONTINUE  

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT01707290
• Other study ID #: VX12-770-112
• Status: Active, not recruiting
• Phase: Phase 3
• First received: October 9, 2012
• Last updated: December 1, 2015
• Start date: February 2013
• Est. completion date: July 2016

Study information

• Verified date: December 2015
• Source: Vertex Pharmaceuticals Incorporated
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationFrance: Agence Nationale de Sécurité du Médicament et des produits de santéBelgium: Federal Agency for Medicinal Products and Health ProductsUnited Kingdom: Medicines and Healthcare Products Regulatory Agency
• Study type: Interventional

Clinical Trial Summary

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).

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 125
• Est. completion date: July 2016
• Est. primary completion date: June 2016
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 6 Years and older

Eligibility

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

Study Design

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

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• ivacaftor
150 mg tablet, oral use, every 12 hours (q12h)

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated	Cystic Fibrosis Foundation Therapeutics

Countries where clinical trial is conducted

United States,  Belgium,  France,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety	Subjective reporting of adverse events, clinical laboratory values (serum chemistry and hematology), electrocardiograms (ECGs), ophthalmologic examinations, and vital signs.	Up to 104 weeks	Yes
Secondary	Absolute change from baseline in percent predicted forced expiratory volume in 1 second (FEV1)		Through Week 104	No
Secondary	Change from baseline in body mass index (BMI)		At Week 104	No
Secondary	Change from baseline in sweat chloride		Through Week 104	No
Secondary	Change from baseline in the respiratory domain of the Cystic Fibrosis Questionnaire Revised (CFQ R)		Through Week 104	No
Secondary	Pulmonary exacerbation		Up to 104 weeks	No