* Evaluation of ELX/TEZ/IVA in Cystic Fibrosis (CF) Subjects 2 Through 5
Research type
Research Study
Full title
A Phase 3 Study Evaluating the Safety, Tolerability, and Pharmacokinetics of Elexacaftor/Tezacaftor/Ivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 2 Through 5 Years of Age
IRAS ID
292548
Contact name
Jane Davies
Contact email
Sponsor organisation
Vertex Pharmaceuticals (Europe) Ltd
Eudract number
2020-002251-38
Clinicaltrials.gov Identifier
Duration of Study in the UK
0 years, 11 months, 30 days
Research summary
Cystic fibrosis (CF) is a lifelong disease resulting from changes (mutations) in the code for one gene called the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Changes in this gene can affect the amount of the protein made from this gene or how well the protein works. When there is not enough protein or it does not work properly, some fluids in the body become sticky and causes problems in the lungs and other organs. This study is being done to learn more about the safety and tolerability of Elexacaftor/Tezacaftor/Ivacaftor Triple Combination Therapy in patients with Cystic Fibrosis who are 2 to 5 years of age, and who have certain mutations in their CF genes.
This is a Phase 3, 2-part (Parts A and B)study where Part A primary objective will evaluate the PK of elexacaftor (ELX), tezacaftor (TEZ), and ivacaftor (IVA) when dosed in triple combination (TC).Part A study will be conducted in United States only.
This Part B study will evaluate the safety and tolerability of ELX/TEZ/IVA in male and female CF subjects 2 through 5 years of age with certain gene mutations.
Participation in Part B could last for approximately 32 weeks with a screening period that will last up to 4 weeks,a study treatment period that will last up to 24 weeks and a follow-up period that will last up to 4 weeks. Subjects who are eligible will be offered the opportunity to enroll in an open-label extension (OLE) safety study.REC name
North West - Greater Manchester South Research Ethics Committee
REC reference
22/NW/0259
Date of REC Opinion
17 Mar 2021
REC opinion
Further Information Favourable Opinion