drugset / Trial / NCT04718844

A Study Investigate the Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Response of SLN124 in Adults With Alpha/Beta-thalassaemia and Very Low- and Low-risk Myelodysplastic Syndrome

NCT04718844 ↗

Phase 1 Completed 44 enrolled Silence Therapeutics GmbH
RandomizedParallel-groupDouble-blindTreatment

Summary

This study will investigate the safety and tolerability of SLN124 in patients with Thalassaemia or patients with Very Low- and Low-risk Myelodysplastic Syndrome (MDS) after single ascending s.c. doses and multiple doses in healthy male and female subjects. Up to 7 cohorts of 56 patients with Thalassaemia and up to 7 cohorts of 56 patients with MDS will be enrolled. Each subject will receive single or multiple doses of SLN124 or placebo given by subcutaneous (s.c) injection.

Timeline

Start
2021-04-14
Primary completion
2023-05-23
Completion
2023-05-23

Drugs

EvaluationDrugModalityDoseRoute
Subject SLN124 Small interfering RNA (siRNA) 1 mg/kg Subcutaneous
Subject SLN124 Small interfering RNA (siRNA) 3 mg/kg Subcutaneous
Subject SLN124 Small interfering RNA (siRNA) 6 mg/kg Subcutaneous
Subject SLN124 Small interfering RNA (siRNA) 10 mg/kg Subcutaneous