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
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
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| 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 |