Ivacaftor in French Patients With Cystic Fibrosis and a G551D Mutation

Completed

• Conditions: Cystic Fibrosis
• Interventions: Drug: CF patients with a G551D mutation and treated with Ivacaftor

• Registration Number: NCT02194881
• Lead Sponsor: Assistance Publique - Hôpitaux de Paris

Brief Summary

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

The aims of our study are:

1. to describe the treated population at initiation of treatment,

2. 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.

3. to evaluate the tolerance and safety of this treatment.

Study Timeline

• Study First Submitted: 2014-07-17
• Study First Submitted QC: 2014-07-17
• Study First Posted: 2014-07-18
• Study Start: 2014-10
• Primary Completion: 2016-02
• Study Completion: 2016-05
• Status Verified: 2016-07
• Last Update Submitted: 2017-07-31
• Last Update Posted: 2017-08-01

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 57

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Ivacaftor 1	CF patients with a G551D mutation and treated with Ivacaftor	patients with CF who are homozygous or heterozygous for the G551D mutation and treated with Ivacaftor

Primary Outcome Measures

Name	Time	Method
FEV1 (in liters and in % predicted)	until one years after initiation of treatment	pulmonary function

Secondary Outcome Measures

Name	Time	Method
number of antibiotic treatments and number of days of antibiotic treatments	until one year after initiation of treatment	number of oral antibiotic treatments and number of days of oral antibiotic treatments, number of IV courses and days of IV antibiotics per year
number of hospitalizations and number of days of hospitalization per year	until one year after initiation of treatment
pulmonary exacerbations	until one years after initiation of treatment
Adverse events	until one year after initiation of treatment	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
respiratory colonization	until least one year after initiation of treatment	Evolution of bacteria and fungi in sputum
nutritional status	until one year after initiation of treatment	Weight (and BMI-Zscore for children)

Trial Locations

Locations (1)

Hôpital Cochin; 🇫🇷 Paris, France