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

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: Cystic Fibrosis
• Intervention / Treatment: Drug: CF patients with a G551D mutation and treated with Ivacaftor

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

Study Locations

• France
  • Paris, France, 75014
    • Hôpital COCHIN

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 4 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• 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

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Retrospective

Number of groups / cohorts

1

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)	pulmonary function	until one years after initiation of treatment

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	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	Evolution of bacteria and fungi in sputum	until least one year after initiation of treatment
nutritional status	Weight (and BMI-Zscore for children)	until one year after initiation of treatment
Adverse events	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

• Sponsor: Assistance Publique - Hôpitaux de Paris
• Collaborators: Vaincre la Mucoviscidose
• Investigators: Study Chair: Dominique Hubert, MD, Assistance Publique - Hôpitaux de Paris

Study record dates

Study Major Dates

• Study Start: 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 (Estimate): July 18, 2014

Study Record Updates

• Last Update Posted (Actual): August 1, 2017
• Last Update Submitted That Met QC Criteria: July 31, 2017
• Last Verified: July 1, 2016

More Information

Terms related to this study

• Keywords: Cystic fibrosis; CFTR; G551D mutation; Novel treatments; Potentiator; Ivacaftor
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Pancreatic Diseases; Fibrosis; Cystic Fibrosis; Molecular Mechanisms of Pharmacological Action; Membrane Transport Modulators; Chloride Channel Agonists; Ivacaftor
• Other Study ID Numbers: CCTIRS 13.652