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

N/ACompletedNCT02722057

Vertex Pharmaceuticals Incorporated368 enrolled

Overview

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.

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 Type

OBSERVATIONAL

Enrollment

368

Conditions

Cystic Fibrosis

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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

Outcomes

Lung function measurements (percent predicted forced expiratory volume in 1 second [FEV1] and forced vital capacity [FVC])

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

Time frame: 36 Months

Pulmonary exacerbations, use of IV antibiotics

Pulmonary exacerbation data will be collected as recorded in the registry.

Time frame: 36 Months

Nutritional parameters (body mass index [BMI], BMI-for-age z-score, weight, and weight-for-age z-score)

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

Time frame: 36 Months

Death or transplantation

Death will be collected from the registry database.

Time frame: 36 months

Hospitalizations

Hospitalizations will be collected from the registry database.

Time frame: 36 Months

Selected Complications (Symptomatic sinus disease, Pulmonary complications, CF-related diabetes (CFRD) and distal intestinal obstruction syndrome (DIOS), Hepatobiliary complications, Pancreatitis)

Information for the above shown CF-related complications as recorded in the registry will be evaluated

Time frame: 36 Months

Select pulmonary microorganisms (e.g., P. aeruginosa, S. aureus)

Data on microorganisms as recorded in the registry will be evaluated

Time frame: 36 Months

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

Overview

Conditions

Eligibility

Outcomes

Primary Outcomes