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

March 23, 2026 updated by: Assistance Publique - Hôpitaux de Paris

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.

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Observational

Enrollment (Actual)

57

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Paris, France, 75014
        • Hôpital Cochin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Patients with CF aged 6 or older and cared in French CF centres

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Retrospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Ivacaftor 1
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

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
pulmonary exacerbations
Time Frame: until one years after initiation of treatment
until one years after initiation of treatment
number of hospitalizations and number of days of hospitalization per year
Time Frame: until one year after initiation of treatment
until one year after initiation of treatment
number of antibiotic treatments and number of days of antibiotic treatments
Time Frame: 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
until one year after initiation of treatment
respiratory colonization
Time Frame: until least one year after initiation of treatment
Evolution of bacteria and fungi in sputum
until least one year after initiation of treatment
nutritional status
Time Frame: until one year after initiation of treatment
Weight (and BMI-Zscore for children)
until one year after initiation of treatment
Adverse events
Time Frame: 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
until one year after initiation of treatment

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Assistance Publique - Hôpitaux de Paris

Collaborators

URC-CIC Paris Descartes Necker Cochin
Vaincre la Mucoviscidose

Investigators

  • Study Chair: Dominique Hubert, MD, Assistance Publique - Hôpitaux de Paris

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 1, 2014

Primary Completion (Actual)

February 1, 2016

Study Completion (Actual)

May 1, 2016

Study Registration Dates

First Submitted

July 17, 2014

First Submitted That Met QC Criteria

July 17, 2014

First Posted (Estimated)

July 18, 2014

Study Record Updates

Last Update Posted (Actual)

March 27, 2026

Last Update Submitted That Met QC Criteria

March 23, 2026

Last Verified

March 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CCTIRS 13.652 (Other Identifier: CCTIRS)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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