A Study to Confirm the Long-term Safety and Effectiveness of Kalydeco in Patients With Cystic Fibrosis Who Have an R117H-CFTR Mutation, Including Pediatric Patients

• Status: Active, not recruiting
• Trial end date: 0000-00-00
• Target enrollment: 150
• Participant gender: Both

Summary

The purpose of this study is to confirm the long-term safety and effectiveness of Kalydeco® (ivacaftor) in US CF patients with the R117H-CFTR mutation <18 years of age and to describe the long-term safety and effectiveness of Kalydeco in CF patients with the R117H-CFTR mutation overall and in patients ≥18 years. The long-term safety and effectiveness of Kalydeco will be examined in totality through the evaluation of the primary outcome measures.

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Vertex Pharmaceuticals Incorporated
• Last Updated: 2016-03-22

Criteria

Inclusion Criteria:

Interventional Cohort

• Male or female with confirmed diagnosis of CF

• Must have at least 1 allele of the R117H-CFTR mutation

• Enrolled in the US CFF Patient Registry at the time of entry into the study

• Patients will be 6 to <18 years of age on the date of signed ICF, and where appropriate,date of assent

• If sexually active, male patients who can father a child and female patients of childbearing potential must agree to meet the contraception requirements

Non Interventional Cohort

• Male or female with confirmed diagnosis of CF

• Must have at least 1 allele of the R117H-CFTR mutation

• Enrolled in the US CFF Patient Registry

• With a record of Kalydeco treatment initiation from 01 January 2015 through 31 December 2016

Historical Cohort

• Patients with CF in the CFF Patient Registry as of 01 January 2009

• Must have at least 1 allele of the R117H-CFTR mutation

• Patients with no evidence of any prior Kalydeco exposure

Exclusion Criteria:

Interventional Cohort

• History of any illness or condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject

• Pregnant, planning a pregnancy, breastfeeding, or not willing to follow contraception requirements

• Abnormal liver function at enrollment

• Abnormal renal function at enrollment

• History of solid organ or hematological transplantation

• Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within 30 days before Enrollment.