A phase 1 study of SLN-124 in patients with thalassaemia and myelodysplastic syndrome
Research type
Research Study
Full title
A randomised, single-blind, placebo-controlled, phase 1, single-ascending and multiple-dose study in adult subjects with alpha/beta-thalassaemia and very low- and low-risk myelodysplastic syndrome to investigate the safety, tolerability, pharmacokinetic, and pharmacodynamic response of SLN124
IRAS ID
1003403
Contact name
Kristin Greenough
Contact email
Sponsor organisation
Silence Therapeutics plc
Eudract number
2019-004639-22
Research summary
SLN is a novel treatment mechanism that adjusts the body’s regulation of iron levels and so can reduce levels. The aim of the study is to learn about the effects of an experimental medicine called SLN124, in people with thalassaemia or MDS, whose bodies produce fewer healthy red blood cells than normal and who can store too much iron in their bodies. Specifically, the research team want to find the most effective and safe dose of SLN124, which can then be used in larger research studies in the future to understand how safe the medicine is and how well it works. The study will compare the effect of SLN124 with a placebo (a substance that is known to have no effect on the body).
The study consists of two parts. In both parts, those taking part will either receive SLN124 or placebo (also known as a ‘dummy’ therapy, since placebo has no physical effect on the body). A computer decides at random who receives SLN124 or placebo. There is a 75% chance of receiving SLN124 and a 25% chance of receiving placebo in the first part of the study. The odds remain the same when the treatment is chosen again in the second part of the study. The participant or the research team will not know which one the participant has received. However, the Safety Review Committee of the study will know who receives SLN124 or placebo. They will use the two sets of results to compare the effects of SLN124 with placebo.
In total, approximately 128 participants will be enrolled globally (approximately 64 participants from part one and approximately 64 participants from part two).REC name
London - Westminster Research Ethics Committee
REC reference
21/LO/0339
Date of REC Opinion
29 Jun 2021
REC opinion
Further Information Favourable Opinion