Real-life Follow-up of Cystic Fibrosis Patients Treated With Ivacaftor+Lumacaftor (Orkambi*)

Completed

• Conditions: Cystic Fibrosis
• Interventions: Drug: Ivacaftor+lumacaftor

• Registration Number: NCT03475381
• Lead Sponsor: Assistance Publique - Hôpitaux de Paris

Brief Summary

The purpose of the study is to examine the real-life safety and effectiveness of the novel combination ivacaftor+lumacaftor in eligible patients with cystic fibrosis (CF). All patients with CF were eligible if they were 12 years and older, started ivacaftor+lumacaftor outside of a clinical trial between December 15th 2017 and December 15th 2018 in an accredited CF center in France. Patient followed-up is based on standardized recommendation of the French Cystic Fibrosis Society. Each patient is followed 1 year.

Detailed Description

Each patient is followed one year with visits at months 1, 3, 6 and 12.

At each visit, the following data are recorded:

* Treatment discontinuation or not. If the treatment was discontinued, reasons for discontinuation

* Adverse effects

* Lung function (spirometry)

* Body mass index

* Pulmonary exacerbations (intravenous antibiotics)

* Sputum microbiology

* Liver enzymes are measured at each visit

At the initial and 12 visits, a yearly CF examination is proposed to the patients:

* Blood tests

* Chest CT scans

* Body plethysmography

Study Timeline

• Study Start: 2016-01-22
• Study First Submitted: 2018-01-04
• Study First Submitted QC: 2018-03-16
• Study First Posted: 2018-03-23
• Primary Completion: 2018-12-31
• Study Completion: 2018-12-31
• Status Verified: 2021-12
• Last Update Submitted: 2021-12-20
• Last Update Posted: 2021-12-21

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 852

Inclusion Criteria

• Patient aged 12 years or older.
• Patient with Cystic Fibrosis with presence of two mutations DF508 in the CFTR gene
• Patient treated with ivacaftor+lumacaftor (Orkambi)

Exclusion Criteria

• Refusal to participate in the study
• Start of Orkambi as part of a clinical trial

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Orkambi treated patients	Ivacaftor+lumacaftor	All patients with CF who started ivacaftor+lumacaftor outside of a clinical trial between January 22nd 2016 and January 22nd 2017.

Primary Outcome Measures

Name	Time	Method
Causes of treatment discontinuation	1 year
Rates of treatment discontinuation	1 year
Timing of treatment discontinuation	1 year

Secondary Outcome Measures

Name	Time	Method
Chloride concentration	1 year	Sweat test before and during treatment
Forced vital capacity (FVC)	1 year	to evaluate lung function
Forced expiratory volume in 1 sec (FEV1)	1 year	to evaluate lung function
Pulmonary exacerbations	1 year	Intravenous antibiotic courses
Body mass index	1 year	Nutritional status

Trial Locations

Locations (1)

Adult CF center, Service de Pneumologie, Cochin Hospital; 🇫🇷 Paris, France