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

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/39005
• EU PAS number:
  EUPAS30550
• Study ID:
  39005
• Official title and acronym:
  Utilisation Patterns and Real-World Effects of Tezacaftor and Ivacaftor Combination Therapy (TEZ/IVA) in Patients With Cystic Fibrosis (CF)
• DARWIN EU® study:
  No
• Study countries:
  France

  Germany

  Ireland

  United Kingdom

  United States
• Study description:
  Cystic fibrosis (CF) is an autosomal recessive disease with serious, chronically debilitating morbidities, and high premature mortality. Tezacaftor (TEZ) and ivacaftor (IVA) combination therapy is currently indicated for treatment of CF in patients 12 and older in the EU and 6 years and older in the US who have specified CFTR mutations and is intended for chronic and potentially lifelong use. In both the EU and the US, the indicated population includes patients who have two copies of the F508del mutation. Additionally in the European Union (EU), TEZ/IVA is indicated for patients heterozygous for the F508del mutation and one of 14 mutations in which the CFTR protein shows residual activity (referred hereafter as residual function RF mutations), in the United Stated (US) TEZ/IVA is also indicated for patients with at least one copy of 26 RF mutations. Information regarding the safety profile of the therapy under the real-world conditions of use will be informative to patients, caregivers, prescribers, and payers. Existing CF registries provide an established source to obtain these data. The primary objectives of this five-year observational cohort study are to evaluate: 1) safety outcomes in patients with CF who have mutations that are indicated for TEZ/IVA, and are treated with TEZ/IVA, 2) CF disease progression in patients who have mutations that are indicated for TEZ/IVA, and are treated with TEZ/IVA, 3) the frequency and outcome of pregnancies in female patients ≥ 14 years, have mutations that are indicated for TEZ/IVA, and are treated with TEZ/IVA, and 4) drug utilisation / potential off-label use of TEZ/IVA. This study will use data collected by CF patient registries in the US, Germany, and UK (all study objectives), as well as Ireland and France (drug utilisation objective only). Within-cohort evaluation of outcomes in the pre- and post-treatment periods will be performed (US, Germany, UK).
• Study status:
  Finalised

Research institutions and networks

Institutions

Vertex Pharmaceuticals

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

Study timelines

Date when funding contract was signed

Planned: 30/06/2019

Actual: 30/06/2019

Study start date

Planned: 30/06/2019

Actual: 30/06/2019

Date of interim report, if expected

Planned: 31/12/2019

Date of final study report

Planned: 31/12/2023

Actual: 02/12/2022

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Vertex Pharmaceuticals Incorporated

Study protocol

Initial protocol

TEZ IVA PASS Protocol

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)