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Completed

NCT Number: NCT02194881

Ivacaftor in French Patients With Cystic Fibrosis and a G551D Mutation

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.

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Key information

Conditions

Cystic Fibrosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Lung Diseases Pancreatic Diseases Respiratory Tract Diseases

Age range

6 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Hôpital Cochin

Paris, 75014, France

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

CF patients with a G551D mutation and treated with Ivacaftor

Drug

Primary outcomes

  1. FEV1 (in liters and in % predicted)

    Time frame: until one years after initiation of treatment

    pulmonary function

Secondary outcomes

  1. pulmonary exacerbations

    Time frame: until one years after initiation of treatment

  2. number of hospitalizations and number of days of hospitalization per year

    Time frame: until one year after initiation of treatment

  3. 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

  4. respiratory colonization

    Time frame: until least one year after initiation of treatment

    Evolution of bacteria and fungi in sputum

  5. nutritional status

    Time frame: until one year after initiation of treatment

    Weight (and BMI-Zscore for children)

  6. 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

Sponsors and collaborators

Lead sponsor

Assistance Publique - Hôpitaux de Paris

Other

Collaborators

  • URC-CIC Paris Descartes Necker Cochin
  • Vaincre la Mucoviscidose

Registry information

Official study title

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.

Acronym: IVACAFTOR1

Important dates

Study start
2014
Primary completion
2016
Study completion
2016
First posted
Jul 18, 2014
Registry last updated
Mar 27, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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