An Observational Study to Evaluate the Utilisation Patterns and Long-term Effects of Lumacaftor and Ivacaftor Combination Therapy in Patients With Cystic Fibrosis

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/39240
• EU PAS number:
  EUPAS15943
• Study ID:
  39240
• Official title and acronym:
  An Observational Study to Evaluate the Utilisation Patterns and Long-term Effects of Lumacaftor and Ivacaftor Combination Therapy 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. The lumacaftor/ivacaftor combination therapy (Orkambi) is indicated for treatment of CF in patients 12 years and older who are homozygous for F508del mutation in the CFTR gene. Understanding long term effects in the overall population of patients receiving treatment and in the specified sub-populations will be informative to patients and their parents, prescribers, and payers. Existing CF registries provide an established source to obtain data on long term effects in a real world use for analysis. The primary objectives of this five-year observational cohort study are to evaluate: 1. Safety outcomes in CF patients who are ≥12 years, homozygous for F508del-CFTR mutation, and treated with Orkambi 2. Frequency and outcome of pregnancies in female patients who are ≥14 years, homozygous for F508del-CFTR mutation, and treated with Orkambi 3. CF disease progression in CF patients who are ≥12 years, homozygous for F508del-CFTR mutation, and treated with Orkambi 4. Drug utilisation / potential off-label use of Orkambi The study will use data collected by existing national CF registries in UK and US (all study objectives), as well as Ireland and France (drug utilization objective only). Data will be analysed separately for each registry for 5 years. Annual analyses results will be combined in a single study report for each year. Each annual report will include data collected during the previous calendar year. For the safety analyses, the Orkambi Safety Cohort will include all patients aged ≥12 years who are homozygous for the F508del-CFTR mutation and have received treatment with Orkambi during the analysis year. The Comparator Safety Cohort will include all patients aged ≥12 years who are heterozygous for the F508del-CFTR mutation with a Class I/II mutation on the second allele and who have never received Orkambi or Kalydeco™.
• Study status:
  Finalised

Research institutions and networks

Institutions

Vertex Pharmaceuticals

UK CF Registry, US CFF Patient Registry, CF Registry of Ireland, CF Registry of France

Study timelines

Date when funding contract was signed

Planned: 31/01/2017

Actual: 31/01/2017

Study start date

Planned: 31/07/2017

Actual: 31/07/2017

Data analysis start date

Planned: 31/07/2017

Actual: 31/07/2017

Date of interim report, if expected

Planned: 31/12/2017

Actual: 28/11/2017

Date of final study report

Planned: 31/12/2021

Actual: 16/11/2021

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Vertex Pharmaceuticals Incorporated

Study protocol

Initial protocol

vx14-809-108_prtl-PASS_v1.2_29Apr2016_Final

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)