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

Cystic Fibrosis Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02722057
• Other study ID #: VX15-770-122
• Status: Completed
• Start date: December 2015
• Est. completion date: December 2019

Study information

• Verified date: February 2020
• Source: Vertex Pharmaceuticals Incorporated
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 368
• Est. completion date: December 2019
• Est. primary completion date: December 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	36 Months
Primary	Pulmonary exacerbations, use of IV antibiotics	Pulmonary exacerbation data will be collected as recorded in the registry.	36 Months
Primary	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	36 Months
Primary	Death or transplantation	Death will be collected from the registry database.	36 months
Primary	Hospitalizations	Hospitalizations will be collected from the registry database.	36 Months
Primary	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	36 Months
Primary	Select pulmonary microorganisms (e.g., P. aeruginosa, S. aureus)	Data on microorganisms as recorded in the registry will be evaluated	36 Months