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

Assistance Publique - Hôpitaux de Paris

Status

Completed

Conditions

Cystic Fibrosis

Treatments

Drug: Ivacaftor+lumacaftor

Study type

Observational

Funder types

Other

Identifiers

NCT03475381

NI17043HLJ

Details and patient eligibility

About

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.

Full 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

Enrollment

852 patients

Sex

All

Ages

12+ years old

Volunteers

No Healthy Volunteers

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