- ICH GCP
- US Clinical Trials Registry
- 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
February 4, 2020 updated by: Vertex Pharmaceuticals Incorporated
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
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.
Study Overview
Status
Completed
Conditions
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.
Study Type
Observational
Enrollment (Actual)
368
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
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
Description
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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
Cohorts and Interventions
Group / Cohort |
---|
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.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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
|
36 Months
|
Pulmonary exacerbations, use of IV antibiotics
Time Frame: 36 Months
|
Pulmonary exacerbation data will be collected as recorded in the registry.
|
36 Months
|
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
|
36 Months
|
Death or transplantation
Time Frame: 36 months
|
Death will be collected from the registry database.
|
36 months
|
Hospitalizations
Time Frame: 36 Months
|
Hospitalizations will be collected from the registry database.
|
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
|
Information for the above shown CF-related complications as recorded in the registry will be evaluated
|
36 Months
|
Select pulmonary microorganisms (e.g., P. aeruginosa, S. aureus)
Time Frame: 36 Months
|
Data on microorganisms as recorded in the registry will be evaluated
|
36 Months
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
December 1, 2015
Primary Completion (Actual)
December 1, 2019
Study Completion (Actual)
December 1, 2019
Study Registration Dates
First Submitted
March 22, 2016
First Submitted That Met QC Criteria
March 22, 2016
First Posted (Estimate)
March 29, 2016
Study Record Updates
Last Update Posted (Actual)
February 5, 2020
Last Update Submitted That Met QC Criteria
February 4, 2020
Last Verified
February 1, 2020
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- VX15-770-122
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Cystic Fibrosis
-
Hospital de Clinicas de Porto AlegreUnknownCystic Fibrosis | Cystic Fibrosis Pulmonary Exacerbation | Cystic Fibrosis in Children | Cystic Fibrosis With ExacerbationBrazil
-
University of Colorado, DenverCystic Fibrosis FoundationTerminatedCystic Fibrosis-related Diabetes | Cystic Fibrosis Pulmonary Exacerbation | Cystic Fibrosis in ChildrenUnited States
-
Royal College of Surgeons, IrelandThe Hospital for Sick Children; Imperial College London; Erasmus Medical Center; University College Dublin and other collaboratorsActive, not recruitingCystic Fibrosis | Adherence, Medication | Cystic Fibrosis Gastrointestinal Disease | Cystic Fibrosis in Children | Cystic Fibrosis Liver DiseaseUnited Kingdom, Ireland
-
Herlev and Gentofte HospitalCopenhagen University Hospital, DenmarkActive, not recruitingMyocardial Infarction | Heart Diseases | Heart Failure | Stroke | Cystic Fibrosis | Heart Failure, Diastolic | Heart Failure, Systolic | Left Ventricular Dysfunction | Cystic Fibrosis-related Diabetes | Cystic Fibrosis Gastrointestinal Disease | Cystic Fibrosis of Pancreas | Cystic Fibrosis, Pulmonary | Cystic...Denmark
-
The Hospital for Sick ChildrenCanadian Cystic Fibrosis FoundationActive, not recruitingCystic Fibrosis | Cystic Fibrosis Gastrointestinal Disease | Cystic Fibrosis in ChildrenCanada
-
AzurRx SASCompletedCystic Fibrosis | Cystic Fibrosis Gastrointestinal Disease | Cystic Fibrosis of PancreasTurkey, Hungary
-
Dartmouth-Hitchcock Medical CenterTrustees of Dartmouth CollegeWithdrawnCystic Fibrosis-related Diabetes | Cystic Fibrosis Liver Disease | CF - Cystic FibrosisUnited States
-
Arrowhead PharmaceuticalsTerminatedCystic Fibrosis, PulmonaryAustralia, New Zealand
-
University of PortsmouthUniversity Hospital Southampton NHS Foundation Trust; Loughborough University; Queen Alexandra HospitalTerminated
-
Mack Biotech, Corp.Completed