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

Cystic Fibrosis Clinical Trial

— ORKAMBI  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03475381
• Other study ID #: NI17043HLJ
• Status: Completed
• Start date: January 22, 2016
• Est. completion date: December 31, 2018

Study information

• Verified date: December 2021
• Source: Assistance Publique - Hôpitaux de Paris
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 852
• Est. completion date: December 31, 2018
• Est. primary completion date: December 31, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• Ivacaftor+lumacaftor
1 year follow-up after initiation of ivacaftor+lumacaftor

Locations

Country	Name	City	State
France	Adult CF center, Service de Pneumologie, Cochin Hospital	Paris

Sponsors (3)

Lead Sponsor	Collaborator
Assistance Publique - Hôpitaux de Paris	Effi-Stat, Societe Francaise de la Mucoviscidose

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Rates of treatment discontinuation		1 year
Primary	Timing of treatment discontinuation		1 year
Primary	Causes of treatment discontinuation		1 year
Secondary	Forced expiratory volume in 1 sec (FEV1)	to evaluate lung function	1 year
Secondary	Forced vital capacity (FVC)	to evaluate lung function	1 year
Secondary	Body mass index	Nutritional status	1 year
Secondary	Pulmonary exacerbations	Intravenous antibiotic courses	1 year
Secondary	Chloride concentration	Sweat test before and during treatment	1 year