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

NCT03475381 | Completed

• 1 other identifier: NI17043HLJ
• Study Type: observational
• Target: 852
• Locations: 1 country
• Sites: 1

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.

Conditions

Cystic Fibrosis

Keywords

Cystic Fibrosis; ivacaftor; lumacaftor

Outcome Measures

Primary Outcomes (3)

• Rates of treatment discontinuation

  1 year

• Timing of treatment discontinuation

  1 year

• Causes of treatment discontinuation

  1 year

Secondary Outcomes (5)

• Forced expiratory volume in 1 sec (FEV1)

  1 year

• Forced vital capacity (FVC)

  1 year

• Body mass index

  1 year

• Pulmonary exacerbations

  1 year

• Chloride concentration

  1 year

Study Arms (1)

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

Interventions

Ivacaftor+lumacaftor DRUG

1 year follow-up after initiation of ivacaftor+lumacaftor

Also known as: Orkambi

Orkambi treated patients

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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

You may qualify if:

• 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)

You may not qualify if:

• Refusal to participate in the study
• Start of Orkambi as part of a clinical trial

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead
• Effi-Stat: collaborator
• Societe Francaise de la Mucoviscidose: collaborator
• URC-CIC Paris Descartes Necker Cochin: collaborator

Study Sites (1)

Adult CF center, Service de Pneumologie, Cochin Hospital

Paris, Paris, 75006, France

Location

Related Publications (1)

• Burgel PR, Munck A, Durieu I, Chiron R, Mely L, Prevotat A, Murris-Espin M, Porzio M, Abely M, Reix P, Marguet C, Macey J, Sermet-Gaudelus I, Corvol H, Bui S, Lemonnier L, Dehillotte C, Da Silva J, Paillasseur JL, Hubert D; French Cystic Fibrosis Reference Network Study Group. Real-Life Safety and Effectiveness of Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis. Am J Respir Crit Care Med. 2020 Jan 15;201(2):188-197. doi: 10.1164/rccm.201906-1227OC.

  PMID: 31601120 RESULT

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

lumacaftor, ivacaftor drug combination

Condition Hierarchy (Ancestors)

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

Study Officials

• Pierre-Regis BURGEL, MD, PhD

  Hôpitaux Universitaire Paris Centre, AP-HP

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 4, 2018
• First Posted: March 23, 2018
• Study Start: January 22, 2016
• Primary Completion: December 31, 2018
• Study Completion: December 31, 2018
• Last Updated: December 1, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1)