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Completed

NCT Number: NCT03475381

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

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.

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Key information

Conditions

Cystic Fibrosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Lung Diseases Pancreatic Diseases Respiratory Tract Diseases

Age range

12 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Adult CF center, Service de Pneumologie, Cochin Hospital

Paris, 75006, France

About this study

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

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Ivacaftor+lumacaftor

Drug

1 year follow-up after initiation of ivacaftor+lumacaftor

Other names: Orkambi

Primary outcomes

  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 outcomes

  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

Sponsors and collaborators

Lead sponsor

Assistance Publique - Hôpitaux de Paris

Other

Collaborators

  • Effi-Stat
  • Societe Francaise de la Mucoviscidose
  • URC-CIC Paris Descartes Necker Cochin

Registry information

Acronym: ORKAMBI

Important dates

Study start
2016
Primary completion
2018
Study completion
2018
First posted
Mar 23, 2018
Registry last updated
Dec 1, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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