VX19-864-101 Efficacy and Safety of VX-864
Research type
Research Study
Full title
A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-864 in PiZZ Subjects
IRAS ID
1003395
Contact name
Alice Turner
Contact email
Eudract number
2019-004881-16
Clinicaltrials.gov Identifier
Research summary
Alpha-1 Antitrypsin Deficiency (AATD) is an inherited condition involving a protein\ncalled Alpha-1 Antitrypsin (AAT). AAT protein is made primarily in the liver. AAT is\nreleased from the liver and circulates in the blood and it protects organs from damage\nduring episodes of inflammation due to infections and other stresses. The main function\nof AAT is to protect the structural proteins in the lungs from damage and helps maintain\nhealthy lung function. In individuals who carry a change in the DNA called the Z\nmutation (also known as PiZZ), the Z protein produced is abnormal and gets trapped in\nthe liver and cannot reach the circulation. This leads to AAT deficiency in the whole body\nincluding the lungs. Over time, the low levels of AAT protein can result in lung damage,\nand the build-up of trapped AAT in the liver may cause liver damage in some individuals\nwith AATD.\nThis study is being conducted to investigate the effect of VX-864 (an investigational\nmedicine) on the levels of AAT proteins in the blood, and to learn more about the safety\nand tolerability of VX-864 in PiZZ individuals who carry the Z AAT mutation. About 40\nparticipants who meet the requirements of the study will be assigned to 1 of 3 VX-814\ngroups or placebo. Study drug dosing is twice daily for approximately 28 days. There are\nabout 10 study visits; some of these visits may be conducted at the participant’s home by\na home health nurse.
REC name
Yorkshire & The Humber - Leeds East Research Ethics Committee
REC reference
20/YH/0233
Date of REC Opinion
14 Sep 2020
REC opinion
Further Information Favourable Opinion