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

Overview

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 Type

  • Study Type: Observational
  • Study Design
    • Time Perspective: Retrospective
  • Study Primary Completion Date: December 31, 2018

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

Interventions

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

Arms, Groups and Cohorts

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

Clinical Trial Outcome Measures

Primary Measures

  • Rates of treatment discontinuation
    • Time Frame: 1 year
  • Timing of treatment discontinuation
    • Time Frame: 1 year
  • Causes of treatment discontinuation
    • Time Frame: 1 year

Secondary Measures

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

Participating in This Clinical Trial

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

Gender Eligibility: All

Minimum Age: 12 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Assistance Publique – Hôpitaux de Paris
  • Collaborator
    • Effi-Stat
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Pierre-Regis BURGEL, MD, PhD, Principal Investigator, Hôpitaux Universitaire Paris Centre, AP-HP

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