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This study will evaluate the safety and tolerability of SLN124 in healthy volunteers.
This first-in-human (FIH) study will investigate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of SLN124 after single ascending s.c. doses in healthy male and female subjects.
Up to 3 cohorts of 24 healthy volunteers will be enrolled. Each subject will receive a single dose of SLN124 or placebo given by subcutaneous (s.c) injection.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 1.0mg/kg | Experimental | Drug: SLN124 |
|
| Placebo | Placebo Comparator |
| |
| 3.0mg/kg | Experimental | Drug: SLN124 |
|
| Optional Cohort | Experimental | An additional dose level may be explored |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SLN124 | Drug | SLN124 for subcutaneous (s.c.) injection |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of treatment-emergent adverse events | safety and tolerability | 8 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Pharmacokinetic: peak plasma concentration (Cmax) | 7 days | |
| Pharmacokinetic: area under the plasma concentration (AUC) | 7 days | |
| Pharmacokinetic: apparent total clearance from plasma after s.c injection (CL/F) |
| Measure | Description | Time Frame |
|---|---|---|
| Pharmacodynamic: change in ferritin | 8 weeks | |
| Pharmacodynamic: change in TSAT | 8 weeks | |
| Pharmacodynamic: change in hepcidin |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Giles Campion, MD | Silence Therapeutics | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hammersmith Medicines Research | London | United Kingdom |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37497888 | Derived | Porter JB, Scrimgeour A, Martinez A, James L, Aleku M, Wilson R, Muckenthaler M, Boyce M, Wilkes D, Schaeper U, Campion GV. SLN124, a GalNAc conjugated 19-mer siRNA targeting tmprss6, reduces plasma iron and increases hepcidin levels of healthy volunteers. Am J Hematol. 2023 Sep;98(9):1425-1435. doi: 10.1002/ajh.27015. Epub 2023 Jul 27. |
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| Placebo | Drug | Sodium chloride for s.c. injection |
|
| 7 days |
| 8 weeks |
| Pharmacodynamic: change in haemoglobin | 8 weeks |