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
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02722057|
Recruitment Status : Active, not recruiting
First Posted : March 29, 2016
Last Update Posted : August 5, 2019
Vertex Pharmaceuticals Incorporated
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated
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.
|Condition or disease|
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|
|Estimated Enrollment :||150 participants|
|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 Start Date :||December 2015|
|Estimated Primary Completion Date :||December 2019|
|Estimated Study Completion Date :||December 2019|
Cohort 1 - Interventional
The Interventional cohort will not be utilized.
Cohort 2 - Non Interventional
A Non-Interventional Cohort comprising pediatric (<18 years of age) and adult R117H-CFTR patients treated with commercially-available Kalydeco.
Cohort 3 - Historical
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 Non-Interventional Cohort.
Primary Outcome Measures :
- Lung function measurements (percent predicted forced expiratory volume in 1 second [FEV1] and forced vital capacity [FVC]) [ Time Frame: 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 [ Time Frame: 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) [ Time Frame: 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 [ Time Frame: 36 months ]Death will be collected from the registry database.
- Hospitalizations [ Time Frame: 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) [ Time Frame: 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) [ Time Frame: 36 Months ]Data on microorganisms as recorded in the registry will be evaluated
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