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

NCT ID: NCT03475381

Last Updated: 2021-12-21

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

852 participants

Study Classification

OBSERVATIONAL

Study Start Date

2016-01-22

Study Completion Date

2018-12-31

Brief Summary

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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

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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

Conditions

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Cystic Fibrosis

Keywords

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Cystic Fibrosis ivacaftor lumacaftor

Study Design

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Observational Model Type

COHORT

Study Time Perspective

RETROSPECTIVE

Study Groups

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Orkambi treated patients

All patients with CF who started ivacaftor+lumacaftor outside of a clinical trial between January 22nd 2016 and January 22nd 2017.

Ivacaftor+lumacaftor

Intervention Type DRUG

1 year follow-up after initiation of ivacaftor+lumacaftor

Interventions

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Ivacaftor+lumacaftor

1 year follow-up after initiation of ivacaftor+lumacaftor

Intervention Type DRUG

Other Intervention Names

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Orkambi

Eligibility Criteria

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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
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Effi-Stat

OTHER

Sponsor Role collaborator

Societe Francaise de la Mucoviscidose

OTHER

Sponsor Role collaborator

Assistance Publique - Hôpitaux de Paris

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Pierre-Regis BURGEL, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Hôpitaux Universitaire Paris Centre, AP-HP

Locations

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Adult CF center, Service de Pneumologie, Cochin Hospital

Paris, , France

Site Status

Countries

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France

Other Identifiers

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NI17043HLJ

Identifier Type: -

Identifier Source: org_study_id