Ivacaftor in French Patients With Cystic Fibrosis and a G551D Mutation (IVACAFTOR1)

Assistance Publique - Hôpitaux de Paris

Status: Completed

Conditions: Cystic Fibrosis

Treatments: Drug: CF patients with a G551D mutation and treated with Ivacaftor

Study type: Observational

Funder types: Other

Identifiers

NCT02194881

CCTIRS 13.652

Details and patient eligibility

About

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.

Full 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.

Enrollment: 57 patients

Sex: All

Ages: 6+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

57 participants in 1 patient group

Ivacaftor 1

Description:

patients with CF who are homozygous or heterozygous for the G551D mutation and treated with Ivacaftor

Treatment:

Drug: CF patients with a G551D mutation and treated with Ivacaftor