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

Completed

• Conditions: Cystic Fibrosis

• Registration Number: NCT02722057
• Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief 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.

Detailed Description

Patient follow-up (i.e., collection of outcomes data after treatment initiation) in the Non-Interventional Cohort will be at least 36 months. The study also includes retrieval of retrospective data entered into the registry for 36 months before the initiation of Kalydeco treatment, from patients matched for Non-Interventional Cohorts. This will permit a within-group comparison of outcomes before and after Kalydeco treatment for effectiveness and safety. The interventional cohort will not be utilized.

Study Timeline

• Study Start: 2015-12
• Study First Submitted: 2016-03-22
• Study First Submitted QC: 2016-03-22
• Study First Posted: 2016-03-29
• Primary Completion: 2019-12
• Study Completion: 2019-12
• Status Verified: 2020-02
• Last Update Submitted: 2020-02-04
• Last Update Posted: 2020-02-05

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 368

Inclusion Criteria

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

Not provided

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Lung function measurements (percent predicted forced expiratory volume in 1 second [FEV1] and forced vital capacity [FVC])	36 Months	Spirometry will be performed according to the standard procedure at each site, and FEV1 values as recorded in the registry will be evaluated. All descriptive and summary data collected for FEV1 will be repeated for FVC
Pulmonary exacerbations, use of IV antibiotics	36 Months	Pulmonary exacerbation data will be collected as recorded in the registry.
Nutritional parameters (body mass index [BMI], BMI-for-age z-score, weight, and weight-for-age z-score)	36 Months	Height and weight measurements as recorded in the registry will be evaluated. BMI, BMI-for-age z-score, and weight-for-age z-score will be derived
Death or transplantation	36 months	Death will be collected from the registry database.
Hospitalizations	36 Months	Hospitalizations will be collected from the registry database.
Selected Complications (Symptomatic sinus disease, Pulmonary complications, CF-related diabetes (CFRD) and distal intestinal obstruction syndrome (DIOS), Hepatobiliary complications, Pancreatitis)	36 Months	Information for the above shown CF-related complications as recorded in the registry will be evaluated
Select pulmonary microorganisms (e.g., P. aeruginosa, S. aureus)	36 Months	Data on microorganisms as recorded in the registry will be evaluated