ZF874 - first doses in humans
Research type
Research Study
Full title
A double-blind, randomised, placebo-controlled study to evaluate the safety, tolerability and pharmacokinetics of single ascending and repeated doses of orally administered ZF874 in healthy volunteers and PiMZ subjects
IRAS ID
284787
Contact name
Malcolm Boyce
Contact email
Sponsor organisation
Z Factor Limited
Eudract number
2020-002198-84
Duration of Study in the UK
1 years, 0 months, 0 days
Research summary
ZF874 (the study medicine) is an experimental new medicine for treating alpha-1-antitrypsin deficiency (AATD), an inherited condition that causes lung disease and may also cause liver problems.
AATD is most often caused by a gene (DNA) mutation called the Z mutation – that mutation affects how the body makes a protein called alpha-1-antitrypsin (A1AT). Everyone has two genes for A1AT. About 1 in 25 people has one gene with the Z mutation and sometimes people have both genes with the Z mutation. People with 2 copies of the Z mutation (PiZZ) do not make enough A1AT. There’s currently no effective treatment for AATD. We hope that ZF874 will repair how A1AT is made, thereby increasing the level of A1AT in the blood.
We’re doing this study to find out the side effects and blood levels of ZF874, and the effect of ZF874 on blood levels of A1AT (Part B only). ZF874 has never been given to humans before, so we’ll start with a small dose and gradually increase the dose as the study progresses.
The study has 2 parts (Parts A and B).
In Part A, 4 groups of 8 healthy volunteers, aged 18–65, will receive single doses of ZF874, with an inpatient stay for 4 days and a final follow up visit 6–8 days later. In Part B, 8 subjects, aged 18–65, with 1 copy of the Z mutation (PiMZ), will receive repeat doses of ZF874 for 28 days over 2 inpatient stays with dosing at home and in the clinic. A final follow up visit will occur approximately 56 days from the first dose.A pharmaceutical company (Z Factor) is funding the study.
The study will take place at 1 centre in London.
REC name
North East - York Research Ethics Committee
REC reference
20/NE/0154
Date of REC Opinion
7 Jul 2020
REC opinion
Favourable Opinion