Real-World Effects and Utilisation Patterns of Elexacaftor, Tezacaftor, and Ivacaftor Combination Therapy (ELX/TEZ/IVA) in Patients with Cystic Fibrosis (CF)

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/45404
• EU PAS number:
  EUPAS43022
• Study ID:
  45404
• Official title and acronym:
  Real-World Effects and Utilisation Patterns of Elexacaftor, Tezacaftor, and Ivacaftor Combination Therapy (ELX/TEZ/IVA) in Patients with Cystic Fibrosis (CF)
• DARWIN EU® study:
  No
• Study countries:
  Germany

  United States
• Study description:
  Cystic fibrosis (CF) is an autosomal recessive disease with serious, chronically debilitating morbidities, and high premature mortality. ELX/TEZ/IVA is currently indicated for treatment of CF in patients 12 years and older in the EU who have specified CFTR mutations. This 5-year observational post-authorisation safety study (PASS) will evaluate safety, effectiveness / CF disease progression, and pregnancy outcomes in patients with CF who are treated with ELX/TEZ/IVA, as well as its drug utilisation patterns using observational cohorts of patients receiving therapy in a real-world setting. Existing CF registries provide an established source to obtain data on long term effects in real world use for analysis. In the US Cystic Fibrosis Foundation Patient Registry (CFFPR) and German CF Registry, within-cohort evaluation of outcomes in the 5-year periods before and after treatment initiation will be performed. Evaluation of the outcome patterns and trends in the 5-year pre-treatment period will place into context the outcome patterns and trends observed in the post-treatment period. In addition, the European Cystic Fibrosis Society Patient Registry (ECFSPR) will be used to provide additional information for the evaluation of drug utilisation patterns in the European region. Information regarding the safety profile of the therapy under the real-world conditions of use will be informative to patients, caregivers, and prescribers. Existing CF registries provide an established source from which to obtain these data.
• Study status:
  Ongoing

Research institutions and networks

Institutions

Vertex Pharmaceuticals

Mukoviszidose Institut

Germany

European Cystic Fibrosis Society (ECFS)

German CF Register Germany, ECFSPR European region (multiple countries), US CFF Patient Registry United States

Study timelines

Date when funding contract was signed

Planned: 31/08/2021

Actual: 31/08/2021

Study start date

Planned: 31/08/2021

Actual: 31/08/2021

Date of interim report, if expected

Planned: 31/12/2021

Date of final study report

Planned: 31/12/2025

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Vertex Pharmaceuticals Incorporated

Study protocol

Initial protocol

ELX-TEZ-IVA PASS Protocol_Version 2.0_redacted

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  EU RMP category 3 (required)