VX19-814-101 Efficacy and Safety of VX-814
Research type
Research Study
Full title
A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-814 in PiZZ Subjects
IRAS ID
273797
Contact name
Clinical Trials and Medical Information
Contact email
Sponsor organisation
Vertex Pharmaceuticals Incorporated
Eudract number
2019-003650-92
Clinicaltrials.gov Identifier
Duration of Study in the UK
0 years, 9 months, 25 days
Research summary
Alpha-1 Antitrypsin Deficiency (AATD) is an inherited condition involving a protein called Alpha-1 Antitrypsin (AAT). AAT protein is made primarily in the liver. AAT is released from the liver and circulates in the blood and it protects organs from damage during episodes of inflammation due to infections and other stresses. The main function of AAT is to protect the structural proteins in the lungs from damage and helps maintain healthy lung function. In individuals who carry a change in the DNA called the Z mutation (also known as PiZZ), the Z protein produced is abnormal and gets trapped in the liver and cannot reach the circulation. This leads to AAT deficiency in the whole body including the lungs. Over time, the low levels of AAT protein can result in lung damage, and the build-up of trapped AAT in the liver may cause liver damage in some individuals with AATD.
This study is being conducted to investigate the effect of VX-814 (an investigational medicine) on the levels of AAT proteins in the blood, and to learn more about the safety and tolerability of VX-814 in PiZZ individuals who carry the Z AAT mutation. About 50 participants who meet the requirements of the study will be assigned to 1 of 4 treatment groups: 3 VX-814 groups and 1 placebo group. Study drug dosing is twice daily for approximately 28 days. There are about 8 study visits; some of these visits may be conducted at the participant’s home by a home health nurse.
REC name
East Midlands - Leicester South Research Ethics Committee
REC reference
20/EM/0021
Date of REC Opinion
28 Feb 2020
REC opinion
Further Information Favourable Opinion