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
NCT ID: NCT02722057
Last Updated: 2020-02-05
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
368 participants
OBSERVATIONAL
2015-12-31
2019-12-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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COHORT
OTHER
Study Groups
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Cohort 1 - Interventional
The Interventional cohort will not be utilized.
No interventions assigned to this group
Cohort 2 - Non Interventional
A Non-Interventional Cohort comprising pediatric (\<18 years of age) and adult R117H-CFTR patients treated with commercially-available Kalydeco.
No interventions assigned to this group
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.
No interventions assigned to this group
Eligibility Criteria
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Inclusion Criteria
* 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
ALL
No
Sponsors
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Vertex Pharmaceuticals Incorporated
INDUSTRY
Responsible Party
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Other Identifiers
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VX15-770-122
Identifier Type: -
Identifier Source: org_study_id
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