Phase Ib study in Adults with Thalassaemia or Myelodysplastic Syndrome
Research type
Research Study
Full title
A Randomized, Single-Blind, Placebo Controlled, Phase 1b Single Ascending (Part A) and Multiple Dose (Part B) First-in-Man Study in Adult Patients with Non-Transfusion Dependent Beta Thalassaemia or Low Risk Myelodysplastic Syndrome to Investigate the Safety, Tolerability, Pharmacokinetic and Pharmacodynamic Response of SLN124
IRAS ID
264655
Contact name
John Brooke Porter
Contact email
Sponsor organisation
Silence Therapeutics plc
Eudract number
2018-004673-27
Duration of Study in the UK
2 years, 3 months, 14 days
Research summary
SLN124 is a new compound that has been designed to work very specifically to increase hepcidin production by the liver. This is expected to treat the hepcidin deficiency seen with β-thalassaemia. By increasing hepcidin, SLN124 should improve the body’s ability to balance iron absorption and safe storage. Giving SLN124 is expected to lower harmful levels of iron in the blood and prevent excess iron damaging growing red blood cells in the bone marrow. In this way, SLN124 is designed to treat both iron overload and anaemia at the same time.
This study investigates the effect of SLN124 which helps to lower the amount of iron that is accumulating in the body due to patient’s condition of having either β-thalassaemia or low-risk myelodysplastic syndrome (MDS). The study is the first to be performed in humans and will assess what the best dose of SLN124 is which should be used for treating iron overload.
During the study the safety of the drug will be checked and how patient’s body deals with the drug (pharmacokinetics) and the effect of the drug on the body (pharmacodynamics) will be monitored.
The study consists of two parts; in the first Part A, 32-40 participants will receive one single dose of SLN124 or placebo per subcutaneous injection. The overall duration of patient’s participation is up to 3 months with up to 6 visits to the hospital.
In Part B, 30 patients will receive up to three separate doses approximately 1 month apart from each other. The overall duration of patient’s participation is up to 7 months with up to 10 visits to the hospital.
The study will be conducted in multiple countries in Europe and the Middle East with up to 14 sites.REC name
East of England - Cambridgeshire and Hertfordshire Research Ethics Committee
REC reference
19/EE/0201
Date of REC Opinion
30 Aug 2019
REC opinion
Further Information Favourable Opinion