Cystic Fibrosis
Conditions
Keywords
Cystic fibrosis, CFTR, G551D mutation, Novel treatments, Potentiator, Ivacaftor
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.
Interventions
DRUGCF patients with a G551D mutation and treated with Ivacaftor
Sponsors
Assistance Publique - Hôpitaux de Paris
Vaincre la Mucoviscidose
URC-CIC Paris Descartes Necker Cochin
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
6 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| FEV1 (in liters and in % predicted) | until one years after initiation of treatment | pulmonary function |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 | 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 |
| 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) |
| 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 |
Countries
France
Contacts
STUDY_CHAIRDominique Hubert, MD
Assistance Publique - Hôpitaux de Paris
Outcome results
None listed