An Observational Study to Evaluate the Long-term Safety of Ivacaftor in Patients With Cystic Fibrosis

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/35683
• EU PAS number:
  EUPAS4270
• Study ID:
  35683
• Official title and acronym:
  An Observational Study to Evaluate the Long-term Safety of Ivacaftor in Patients With Cystic Fibrosis
• DARWIN EU® study:
  No
• Study countries:
  France

  Ireland

  United Kingdom

  United States
• Study description:
  Cystic fibrosis (CF) is an autosomal recessive disease with serious, chronically debilitating morbidities and high premature mortality, and at present, there is no cure. Cystic fibrosis affects approximately 70,000 individuals worldwide and is caused by mutations in the CF transmembrane conductance regulator gene (CFTR), which result in absent or deficient function of the CF transmembrane conductance regulator (CFTR) protein at the cell surface.Ivacaftor (Kalydeco™, 150-mg tablets) has been approved in the United States, the European Union (EU), Canada and Australia to date, for the treatment of CF in patients 6 years of age and older who have a G551D mutation in the CFTR gene. Ivacaftor is an orally bioavailable small molecule CFTR potentiator that targets the underlying defect in CF. As such, ivacaftor is a member of a new class of drugs - CFTR modulators - that provide a new therapeutic approach to the treatment of CF. Ivacaftor is the first CFTR modulator to show an improvement in CFTR function and clinical benefit in patients with CF.Ivacaftor is intended for chronic, potentially lifelong use. Understanding of the long term effects will be informative to patients and their parents, prescribers, and payers. Existing CF registries provide an established source to obtain long term safety in a real life use for analysis. The patient registries of the CF Trust in the United Kingdom (UK) and the CF Foundation in the United States (US) provide the ideal source to obtain long term safety information because the data collected are extensive and consistent with one another. In addition, the patients with CF from the CF Trust and the CF Foundation patient registries encompass a majority of the patients in the indicated population.Cystic fibrosis patient registries from France and Ireland were considered for this study and will be used for drug utilization analysis.
• Study status:
  Finalised

Research institutions and networks

Institutions

Vertex Pharmaceuticals

Study timelines

Date when funding contract was signed

Planned: 31/07/2013

Actual: 17/07/2013

Study start date

Planned: 31/07/2013

Actual: 31/07/2013

Data analysis start date

Planned: 31/07/2013

Actual: 31/07/2013

Date of interim report, if expected

Planned: 31/12/2013

Actual: 10/12/2013

Date of final study report

Planned: 31/12/2017

Actual: 12/12/2017

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Vertex Pharmaceuticals Incorporated

Study protocol

Initial protocol

Long-term Safety Study Protocol-v2.2-07may2013

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  EU RMP category 1 (imposed as condition of marketing authorisation)