A group of researchers from HMR is looking for people to take part in clinical trials for an experimental new medicine to treat an inherited condition that causes lung disease and may also cause liver problems.
What is the study about?
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. Researchers hope that ZF874 will repair how A1AT is made, thereby increasing the level of A1AT in the blood.
Researchers are 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 they’ll start with a small dose and gradually increase the dose as the study progresses.
How the study will be conducted
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 a centre in London.
If you'd like to find out more
For more information or to take part, please contact email@example.com and quote 20B005. The research is not being conducted by the British Liver Trust.