Ivacaftor in French Patients With Cystic Fibrosis and a G551D Mutation

Cystic Fibrosis Clinical Trial

— IVACAFTOR1  

Official 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.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02194881
• Other study ID #: CCTIRS 13.652
• Status: Completed
• Phase: N/A
• First received: July 17, 2014
• Last updated: July 31, 2017
• Start date: October 2014
• Est. completion date: May 2016

Study information

• Verified date: July 2016
• Source: Assistance Publique - Hôpitaux de Paris
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 57
• Est. completion date: May 2016
• Est. primary completion date: February 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

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

Locations

Country	Name	City	State
France	Hôpital Cochin	Paris

Sponsors (2)

Lead Sponsor	Collaborator
Assistance Publique - Hôpitaux de Paris	Vaincre la Mucoviscidose

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	FEV1 (in liters and in % predicted)	pulmonary function	until one years after initiation of treatment
Secondary	pulmonary exacerbations		until one years after initiation of treatment
Secondary	number of hospitalizations and number of days of hospitalization per year		until one year after initiation of treatment
Secondary	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
Secondary	respiratory colonization	Evolution of bacteria and fungi in sputum	until least one year after initiation of treatment
Secondary	nutritional status	Weight (and BMI-Zscore for children)	until one year after initiation of treatment
Secondary	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