Safety, tolerability, PK and PD of SLN124
Research type
Research Study
Full title
A randomised, double-blind, placebo-controlled phase 1 single ascending dose study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of subcutaneously administered SLN124 in healthy volunteers
IRAS ID
1003069
Contact name
Denisa Wilkes
Contact email
Eudract number
2020-002544-23
Research summary
The study medicine (SLN124) is an experimental new medicine designed to treat iron
overload disorders (too much iron in the body) such as β-thalassaemia and
myelodysplastic syndrome (MDS) variants. Iron plays an essential role in the body,
including in the production of red blood cells (RBC) that carry oxygen from the lungs to
the rest of the body. Low levels of RBC (anaemia) may cause symptoms such as excessive
tiredness and weakness while too much iron can lead to serious health problems such as
swelling and scarring of the liver (cirrhosis), heart failure and diabetes.
The study medicine is a new type of medicine known as an siRNA; a gene ‘silencing’
therapy – one that is designed to temporarily ‘switch off’ the production of a particular
protein (matriptase-2), which will then increase levels of hepcidin – a hormone that helps
regulate iron levels in the body. This is the first time SLN124 will be given to humans.
We’ll give up to 24 healthy men and women (in up to 3 groups of 8) a single dose of the
study medicine, given by injection(s) under the skin. The first group of participants will
receive a small dose, then we plan to increase the dose in subsequent group(s). We aim to
find out the side effects, blood levels of the study medicine and whether it has any effect
on blood levels of substances linked to iron metabolism and RBC production, to further
assess its potential in iron overload disorders.
Participants will take up to 9 weeks to finish the study. They’ll make up to 4 outpatient
visits and stay on the ward for 4 days and 3 nights in a row.
A pharmaceutical company Silence Therapeutics is funding the study.
The study will take place at 1 centre in London.REC name
London - Surrey Borders Research Ethics Committee
REC reference
20/LO/0820
Date of REC Opinion
19 Aug 2020
REC opinion
Further Information Favourable Opinion