Overview

This trial is active, not recruiting.

Condition cystic fibrosis
Treatment kalydeco
Phase phase 4
Sponsor Vertex Pharmaceuticals Incorporated
Start date December 2015
End date December 2019
Trial size 150 participants
Trial identifier NCT02722057, VX15-770-122

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Observational model cohort
Arm
An Interventional Cohort comprising pediatric R117H-CFTR subjects 6 to <18 years of age receiving Kalydeco as part of the open-label, single-arm component of this study
kalydeco
Kalydeco 150-mg tablet will be administered orally every 12 hours (q12h);
A Non-Interventional Cohort comprising pediatric (<18 years of age) and adult R117H-CFTR patients treated with commercially-available Kalydeco.
A Historical Cohort comprising data from an earlier time period for pediatric (<18 years of age) and adult patients with the R117H-CFTR mutation who have never been exposed to Kalydeco and matched on age, gender, and lung function to patients in the Interventional and Non-Interventional Cohorts.

Primary Outcomes

Measure
Lung function measurements (percent predicted forced expiratory volume in 1 second [FEV1] and forced vital capacity [FVC])
time frame: 36 Months
Pulmonary exacerbations, use of IV antibiotics
time frame: 36 Months
Nutritional parameters (body mass index [BMI], BMI-for-age z-score, weight, and weight-for-age z-score)
time frame: 36 Months
Death or transplantation
time frame: 36 months
Hospitalizations
time frame: 36 Months
Selected Complications (Symptomatic sinus disease, Pulmonary complications, CF-related diabetes (CFRD) and distal intestinal obstruction syndrome (DIOS), Hepatobiliary complications, Pancreatitis)
time frame: 36 Months
Select pulmonary microorganisms (e.g., P. aeruginosa, S. aureus)
time frame: 36 Months

Eligibility Criteria

Male or female participants at least 6 years old.

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.

Additional Information

Official title A Study in US Cystic Fibrosis Patients With the R117H-CFTR Mutation to Confirm the Long-term Safety and Effectiveness of Kalydeco, Including Patients <18 Years of Age, Combining Data Captured in the Cystic Fibrosis Foundation Registry From an Interventional Cohort and a Non-Interventional Cohort
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 both the Interventional and Non-Interventional Cohorts. This will permit a within-group comparison of outcomes before and after Kalydeco treatment for effectiveness and safety.
Trial information was received from ClinicalTrials.gov and was last updated in March 2016.
Information provided to ClinicalTrials.gov by Vertex Pharmaceuticals Incorporated.