Ivacaftor in French Patients With Cystic Fibrosis and a G551D Mutation

NCT02194881 | Completed

• 1 other identifier: CCTIRS 13.652
• Study Type: observational
• Target: 57
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to determine whether the treatment with Ivacaftor remains effective and safe in the patients with cystic fibrosis (and at least one G551D CFTR mutation) in the real life setting, after the drug has been approved by the Health authorities.

Conditions

Cystic Fibrosis

Keywords

Cystic fibrosis; CFTR; G551D mutation; Novel treatments; Potentiator; Ivacaftor

Outcome Measures

Primary Outcomes (1)

• FEV1 (in liters and in % predicted)

  pulmonary function

  until one years after initiation of treatment

Secondary Outcomes (6)

• pulmonary exacerbations

  until one years after initiation of treatment

• number of hospitalizations and number of days of hospitalization per year

  until one year after initiation of treatment

• number of antibiotic treatments and number of days of antibiotic treatments

  until one year after initiation of treatment

• respiratory colonization

  until least one year after initiation of treatment

• nutritional status

  until one year after initiation of treatment

Study Arms (1)

Ivacaftor 1

patients with CF who are homozygous or heterozygous for the G551D mutation and treated with Ivacaftor

Drug: CF patients with a G551D mutation and treated with Ivacaftor

Interventions

CF patients with a G551D mutation and treated with Ivacaftor DRUG

Ivacaftor 1

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with CF aged 6 or older and cared in French CF centres

You may qualify if:

• French patients with CF aged 6 or older who are homozygous or heterozygous for the G551D mutation
• Treated with Ivacaftor
• First prescription of Ivacaftor before June 1st 2013 (including patients randomized in the VX770 clinical trials)

You may not qualify if:

• CF patients younger than 6 years old
• CF patients who have received lung transplantation
• CF patients without a G551D mutation.

Sponsors & Collaborators

• Vaincre la Mucoviscidose: collaborator
• URC-CIC Paris Descartes Necker Cochin: collaborator
• Assistance Publique - Hôpitaux de Paris: lead

Study Sites (1)

Hôpital Cochin

Paris, 75014, France

Location

Related Publications (1)

• Hubert D, Dehillotte C, Munck A, David V, Baek J, Mely L, Dominique S, Ramel S, Danner Boucher I, Lefeuvre S, Reynaud Q, Colomb-Jung V, Bakouboula P, Lemonnier L. Retrospective observational study of French patients with cystic fibrosis and a Gly551Asp-CFTR mutation after 1 and 2years of treatment with ivacaftor in a real-world setting. J Cyst Fibros. 2018 Jan;17(1):89-95. doi: 10.1016/j.jcf.2017.07.001. Epub 2017 Jul 12.

  PMID: 28711222 RESULT

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

Therapeutics; ivacaftor

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

• Dominique Hubert, MD

  Assistance Publique - Hôpitaux de Paris

  STUDY CHAIR

Study Design

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

Study Record Dates

• First Submitted: July 17, 2014
• First Posted: July 18, 2014
• Study Start: October 1, 2014
• Primary Completion: February 1, 2016
• Study Completion: May 1, 2016
• Last Updated: March 27, 2026

Record last verified: 2026-03

Locations

FR (1)