Ivacaftor in French Patients With Cystic Fibrosis and a G551D Mutation

Study Overview
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Brief Summary
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The purpose of this study is to determine whether the treatment with Ivacaftor remains effective and safe in the patients with cystic fibrosis (and at least one G551D CFTR mutation) in the real life setting, after the drug has been approved by the Health authorities.

Detailed Description
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The aims of our study are: to describe the treated population at initiation of treatment, to evaluate clinical parameters during the year before Ivacaftor was started, at initiation of treatment and during at least one year of treatment, until June 2014. to evaluate the tolerance and safety of this treatment.

Official Title
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Ivacaftor in French Patients With Cystic Fibrosis and a G551D Mutation - Efficacy and Safety Results After the First Year of Treatment in the Real Life Setting.

Conditions
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Cystic Fibrosis

Intervention / Treatment
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* Drug: CF patients with a G551D mutation and treated with Ivacaftor


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: French patients with CF aged 6 or older who are homozygous or heterozygous for the G551D mutation Treated with Ivacaftor First prescription of Ivacaftor before June 1st 2013 (including patients randomized in the VX770 clinical trials) Exclusion Criteria: CF patients younger than 6 years old CF patients who have received lung transplantation CF patients without a G551D mutation.

Ages Eligible for Study
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Minimum Age: 6 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 |
| --- | --- |
| Ivacaftor 1<br>patients with CF who are homozygous or heterozygous for the G551D mutation and treated with Ivacaftor | Drug: CF patients with a G551D mutation and treated with Ivacaftor<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| FEV1 (in liters and in % predicted) | pulmonary function | until one years after initiation of treatment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| pulmonary exacerbations |  | until one years after initiation of treatment | 
| number of hospitalizations and number of days of hospitalization per year |  | until one year after initiation of treatment | 
| number of antibiotic treatments and number of days of antibiotic treatments | number of oral antibiotic treatments and number of days of oral antibiotic treatments, number of IV courses and days of IV antibiotics per year | until one year after initiation of treatment | 
| respiratory colonization | Evolution of bacteria and fungi in sputum | until least one year after initiation of treatment | 
| nutritional status | Weight (and BMI-Zscore for children) | until one year after initiation of treatment | 
| Adverse events | Dates and reasons for interruption and discontinuation of treatment with Ivacaftor Adverse events, indicating what in the physician's opinion might be due to Ivacaftor | until one year after initiation of treatment | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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CFTR, Cystic fibrosis, G551D mutation, Novel treatments, Potentiator, Ivacaftor