A Study of VX-814
Research type
Research Study
Full title
A Phase 1 Study of VX-814
IRAS ID
250949
Contact name
James Witty
Contact email
Sponsor organisation
Vertex Pharmaceuticals Incorporated
Eudract number
2018-002164-19
Duration of Study in the UK
0 years, 7 months, 12 days
Research summary
Research Summary
Alpha-1 Antitrypsin (AAT) is a protein that is mostly made in the liver. The protein is released from the liver, circulates in the blood and travels to the lungs where it plays an important part in maintaining healthy lung function. In some individuals, the AAT protein is trapped in the liver and cannot reach the lungs, leading to AAT Deficiency (AATD). AATD is a genetic disorder. Over time, low levels of AAT protein in the blood can result in lung damage and the build-up in the liver may also damage the liver.\n\nThis study is being done to learn more about the safety and tolerability of VX-814 in healthy participants and participants who are healthy but carry a genetic mutation (called the Z mutation; PiZZ) that puts them at increased risk for developing AATD related disease (lung or liver disease). A genetic mutation is a change in DNA. VX-814 is an investigational medication; “investigational” means the medicine is not approved by Health Authorities like the European Medicines Agency (EMA) in the European Union (EU) and is still being tested.\n\nThe study is split into three parts:\n-Part A will look at single doses of VX-814 in healthy participants. They will stay at the unit for 5 nights in each of three treatment periods (TP) and will receive one dose of study medication during each TP.\n- Part B will look at multiple doses of VX-814 in healthy participants. They will stay at the unit for 14 nights and will receive study medication either once, twice or three times daily for 10 days.\n- Part C will look at multiple doses of VX-814 in people with the PiZZ genotype. They will receive study medication once, twice or three times daily for approximately 27 days and be required to take this at home between their visits.Summary of Results
Not available.REC name
North East - York Research Ethics Committee
REC reference
18/NE/0238
Date of REC Opinion
15 Nov 2018
REC opinion
Further Information Favourable Opinion