Clinical trial NCT02722057: 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
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
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Cystic Fibrosis
Lead sponsor: Vertex Pharmaceuticals Incorporated
Sponsor class: INDUSTRY
First posted: 2016-03-29
Enrollment: 368
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Non Interventional Cohort - Patients Enrolled in the US CFF Patient Registry Historical Cohort - Patients with CF in the CFF Patient Registry as of 01 January 2009

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.

Eligibility criteria: 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

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