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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03475381
Recruitment Status : Unknown
Verified May 2018 by Assistance Publique - Hôpitaux de Paris.
Recruitment status was:  Active, not recruiting
First Posted : March 23, 2018
Last Update Posted : May 22, 2018
Sponsor:
Collaborators:
Effi-Stat
Societe Francaise de la Mucoviscidose
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment
Cystic Fibrosis Drug: Ivacaftor+lumacaftor

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

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Study Type : Observational
Actual Enrollment : 685 participants
Observational Model: Cohort
Time Perspective: Retrospective
Official Title: Real-life Follow-up of Cystic Fibrosis Patients Treated With Ivacaftor+Lumacaftor (Orkambi*)
Actual Study Start Date : January 22, 2016
Estimated Primary Completion Date : June 30, 2018
Estimated Study Completion Date : June 30, 2018

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis
Drug Information available for: Ivacaftor

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 Name: Orkambi




Primary Outcome Measures :
  1. Rates of treatment discontinuation [ Time Frame: 1 year ]
  2. Timing of treatment discontinuation [ Time Frame: 1 year ]
  3. Causes of treatment discontinuation [ Time Frame: 1 year ]

Secondary Outcome Measures :
  1. Forced expiratory volume in 1 sec (FEV1) [ Time Frame: 1 year ]
    to evaluate lung function

  2. Forced vital capacity (FVC) [ Time Frame: 1 year ]
    to evaluate lung function

  3. Body mass index [ Time Frame: 1 year ]
    Nutritional status

  4. Pulmonary exacerbations [ Time Frame: 1 year ]
    Intravenous antibiotic courses

  5. Chloride concentration [ Time Frame: 1 year ]
    Sweat test before and during treatment



Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03475381


Locations
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France
Adult CF center, Service de Pneumologie, Cochin Hospital
Paris, France, 75006
Sponsors and Collaborators
Assistance Publique - Hôpitaux de Paris
Effi-Stat
Societe Francaise de la Mucoviscidose
Investigators
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Principal Investigator: Pierre-Regis BURGEL, MD, PhD Hôpitaux Universitaire Paris Centre, AP-HP
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Responsible Party: Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier: NCT03475381    
Other Study ID Numbers: NI17043HLJ
First Posted: March 23, 2018    Key Record Dates
Last Update Posted: May 22, 2018
Last Verified: May 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Assistance Publique - Hôpitaux de Paris:
Cystic Fibrosis
ivacaftor
lumacaftor
Additional relevant MeSH terms:
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Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Ivacaftor
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action