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

Sponsor
Assistance Publique - Hôpitaux de Paris (Other)
Overall Status
Completed
CT.gov ID
NCT03475381
Collaborator
Effi-Stat (Other), Societe Francaise de la Mucoviscidose (Other)
852
Enrollment
1
Location
35.3
Actual Duration (Months)
24.1
Patients Per Site Per Month

Study Details

Study Description

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 Phase

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 Design

Study Type:
Observational
Actual Enrollment :
852 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 :
Jan 22, 2016
Actual Primary Completion Date :
Dec 31, 2018
Actual Study Completion Date :
Dec 31, 2018

Arms and Interventions

Arm 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

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Rates of treatment discontinuation [1 year]

    2. Timing of treatment discontinuation [1 year]

    3. Causes of treatment discontinuation [1 year]

    Secondary Outcome Measures

    1. Forced expiratory volume in 1 sec (FEV1) [1 year]

      to evaluate lung function

    2. Forced vital capacity (FVC) [1 year]

      to evaluate lung function

    3. Body mass index [1 year]

      Nutritional status

    4. Pulmonary exacerbations [1 year]

      Intravenous antibiotic courses

    5. Chloride concentration [1 year]

      Sweat test before and during treatment

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    12 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 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

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

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Assistance Publique - Hôpitaux de Paris
    ClinicalTrials.gov Identifier:
    NCT03475381
    Other Study ID Numbers:
    • NI17043HLJ
    First Posted:
    Mar 23, 2018
    Last Update Posted:
    Dec 21, 2021
    Last Verified:
    Dec 1, 2021
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    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:
    Cystic Fibrosis
    Fibrosis
    Ivacaftor

    Study Results

    No Results Posted as of Dec 21, 2021