Effectiveness and Safety of VX-561 in Subjects Aged 18 Years and Older
Research type
Research Study
Full title
A Phase 2, Randomized, Double-blind Study to Evaluate the Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis
IRAS ID
264919
Contact name
Gordon MacGregor
Contact email
Sponsor organisation
Vertex Pharmaceuticals Incorporated
Eudract number
2018-003970-28
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 2 months, 7 days
Research summary
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 VX-561 is safe and effective in the tested doses. VX-561 is a form of ivacaftor (Kalydeco®) that has undergone a process where some of the molecules are deuterated, which means that it stays in your body longer and may be taken less frequently than ivacaftor. The purpose of this study is to identify a once-daily dose of VX-561 so that future patients may only have to take CFTR modulators once a day. This may be more conve¬nient for CF patients.
The study drug VX-561 lab data indicates a similar potency of VX-561 in human bronchial epithelial (HBE) cells relative to ivacaftor. Safety pharmacology and nonclinical toxicology studies of VX-561 demonstrate a similar safety profile relative to ivacaftor. An early study in healthy participants have shown that VX-561 had a reduced rate of clearance, increased exposure, greater plasma levels at 24 hours, and a longer half-life compared to ivacaftor, thereby supporting a once daily dosing. In healthy subject studies to date, the safety profile of VX-561 is considered similar to ivacaftor. This Study will enrol Male and female participants with CF who have a gating mutation and were previously taking a stable dose of ivacaftor, ages 18 and older.
Including the Screening Period, each participant will participate in the study for approximately 20 weeks. There will be approximately 88 people participating in this study.Summary of Results
"VX18-561-101 was a Phase 2, randomized, doubleâ€blind study, designed to evaluate the efficacy and safety of VXâ€561 in people aged 18 Years and older with cystic fibrosis. A total of 77 patients with a cystic fibrosis transmembrane conductance regulator (CFTR) gating mutation, who had previously been stable on ivacaftor monotherapy, took part in the study.Results for efficacy: Treatment with VX-561 led to improvements in respiratory symptoms, CFTR function and lung function, compared to baseline.
Results for safety: Treatment with VX-561 was generally safe and well tolerated. Adverse events were mild to moderate in severity. The safety profile of VX-561 was consistent with the safety profile of ivacaftor."
REC name
West of Scotland REC 1
REC reference
19/WS/0069
Date of REC Opinion
18 Jun 2019
REC opinion
Further Information Favourable Opinion