Real-life Follow-up of Cystic Fibrosis Patients Treated With Ivacaftor+Lumacaftor (Orkambi*)

Study Results

Results pending

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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Recruitment Status

COMPLETED

Total Enrollment

852 participants

Study Classification

OBSERVATIONAL

Study Start Date

2016-01-22

Study Completion Date

2018-12-31

Brief Summary

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The purpose of the study is to examine the real-life safety and effectiveness of the novel combination ivacaftor+lumacaftor in eligible patients with cystic fibrosis (CF). All patients with CF were eligible if they were 12 years and older, started ivacaftor+lumacaftor outside of a clinical trial between December 15th 2017 and December 15th 2018 in an accredited CF center in France. Patient followed-up is based on standardized recommendation of the French Cystic Fibrosis Society. Each patient is followed 1 year.

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Detailed Description

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Each patient is followed one year with visits at months 1, 3, 6 and 12.

At each visit, the following data are recorded:

* Treatment discontinuation or not. If the treatment was discontinued, reasons for discontinuation
* Adverse effects
* Lung function (spirometry)
* Body mass index
* Pulmonary exacerbations (intravenous antibiotics)
* Sputum microbiology
* Liver enzymes are measured at each visit

At the initial and 12 visits, a yearly CF examination is proposed to the patients:

* Blood tests
* Chest CT scans
* Body plethysmography

Conditions

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Cystic Fibrosis

Study Design

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Observational Model Type

COHORT

Study Time Perspective

RETROSPECTIVE

Study Groups

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Orkambi treated patients

All patients with CF who started ivacaftor+lumacaftor outside of a clinical trial between January 22nd 2016 and January 22nd 2017.

Ivacaftor+lumacaftor

Intervention Type DRUG

1 year follow-up after initiation of ivacaftor+lumacaftor

Interventions

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Ivacaftor+lumacaftor

1 year follow-up after initiation of ivacaftor+lumacaftor

Intervention Type DRUG

Other Intervention Names

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Orkambi

Eligibility Criteria

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Inclusion Criteria

* Patient aged 12 years or older.
* Patient with Cystic Fibrosis with presence of two mutations DF508 in the CFTR gene
* Patient treated with ivacaftor+lumacaftor (Orkambi)