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

Study Overview
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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.

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

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

Conditions
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Cystic Fibrosis

Intervention / Treatment
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* Drug: Ivacaftor+lumacaftor


Participation Criteria
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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) Exclusion Criteria: Refusal to participate in the study Start of Orkambi as part of a clinical trial

Ages Eligible for Study
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Minimum Age: 12 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Orkambi treated patients<br>All patients with CF who started ivacaftor+lumacaftor outside of a clinical trial between January 22nd 2016 and January 22nd 2017. | Drug: Ivacaftor+lumacaftor<br>* 1 year follow-up after initiation of ivacaftor+lumacaftor<br>* Other names: Orkambi;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Rates of treatment discontinuation |  | 1 year | 
| Timing of treatment discontinuation |  | 1 year | 
| Causes of treatment discontinuation |  | 1 year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Forced expiratory volume in 1 sec (FEV1) | to evaluate lung function | 1 year | 
| Forced vital capacity (FVC) | to evaluate lung function | 1 year | 
| Body mass index | Nutritional status | 1 year | 
| Pulmonary exacerbations | Intravenous antibiotic courses | 1 year | 
| Chloride concentration | Sweat test before and during treatment | 1 year | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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ivacaftor, Cystic Fibrosis, lumacaftor