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

• ClinicalTrials.gov Identifier: NCT03475381
• Recruitment Status: Unknown
• First Posted: March 23, 2018
• Last Update Posted: May 22, 2018
• Sponsor: Assistance Publique - Hôpitaux de Paris
• Collaborators: Effi-Stat; Societe Francaise de la Mucoviscidose
• Information provided by (Responsible Party): Assistance Publique - Hôpitaux de Paris

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
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

Study Design

• 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

Groups and Cohorts

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

Outcome Measures

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

Eligibility Criteria

• 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

Contacts and Locations

Locations

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

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

More Information

• Responsible Party: Assistance Publique - Hôpitaux de Paris
• ClinicalTrials.gov Identifier: NCT03475381
• Other Study ID Numbers: NI17043HLJ
• First Posted: March 23, 2018
• Last Update Posted: May 22, 2018
• Last Verified: May 2018

Individual Participant Data (IPD) Sharing Statement:

• 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; 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