Phase 3 Study of VX-445/TEZ/IVA Triple Combination Therapy in CF
Research type
Research Study
Full title
A Phase 3 Study Evaluating the Pharmacokinetics, Safety, and Tolerability of VX-445/TEZ/IVA Triple Combination Therapy in Cystic Fibrosis Subjects 6 Through 11 Years of Age
IRAS ID
269510
Contact name
James Witty
Contact email
Sponsor organisation
Vertex Pharmaceuticals Incorporated
Eudract number
2018-001695-38
Duration of Study in the UK
1 years, 2 months, 1 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 will help researchers learn if the study drugs VX-445/TEZ/IVA is safe and effective in the tested doses. VX- 445, tezacaftor and ivacaftor are a triple combination of therapies targeted at improving the function of this protein.
There is a strong rationale for preventing the progress of the disease by treating patients earlier in life. Currently Vertex VX-445/TEZ/IVA therapy in Phase 3 studies in adult and young CF subjects with 1 or 2 copies of the F508del mutation, namely those with F/MF and F/F genotypes. Given the clinical benefit seen in previous studies of adults with CF, this study is designed to obtain pharmacokinetic (PK),
safety and tolerability information to test the study drug in the pediatric population 6-11 years of age.
Approximately 12 subjects are planned for enrollment for Part A. A review of safety, acceptability, and available Pharmacokinetic (PK) data will be completed after Part A to confirm or adjust the dose chosen for Part B.
Approximately 25 participants with F/MF genotypes and approximately 20 subjects with F/F genotypes are targeted for enrollment in Part B. 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.REC name
North West - Greater Manchester South Research Ethics Committee
REC reference
19/NW/0506
Date of REC Opinion
9 Oct 2019
REC opinion
Further Information Favourable Opinion