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

December 20, 2021 updated by: Assistance Publique - Hôpitaux de Paris
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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Observational

Enrollment (Actual)

852

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Paris, France, 75006
        • Adult CF center, Service de Pneumologie, Cochin Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

12 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Patient with Cystic Fibrosis with presence of two mutations DF508 in the CFTR gene and treated by Orkambi in usual care

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Retrospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Orkambi treated patients
All patients with CF who started ivacaftor+lumacaftor outside of a clinical trial between January 22nd 2016 and January 22nd 2017.
Drug: Ivacaftor+lumacaftor
1 year follow-up after initiation of ivacaftor+lumacaftor
Other Names:
  • Orkambi

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Rates of treatment discontinuation
Time Frame: 1 year
1 year
Timing of treatment discontinuation
Time Frame: 1 year
1 year
Causes of treatment discontinuation
Time Frame: 1 year
1 year

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Assistance Publique - Hôpitaux de Paris

Collaborators

Societe Francaise de la Mucoviscidose
Effi-Stat

Investigators

  • Principal Investigator: Pierre-Regis BURGEL, MD, PhD, Hôpitaux Universitaire Paris Centre, AP-HP

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 22, 2016

Primary Completion (Actual)

December 31, 2018

Study Completion (Actual)

December 31, 2018

Study Registration Dates

First Submitted

January 4, 2018

First Submitted That Met QC Criteria

March 16, 2018

First Posted (Actual)

March 23, 2018

Study Record Updates

Last Update Posted (Actual)

December 21, 2021

Last Update Submitted That Met QC Criteria

December 20, 2021

Last Verified

December 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NI17043HLJ

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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