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| Name | Class |
|---|---|
| Arrowhead Pharmaceuticals | INDUSTRY |
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This is a phase 1, randomized, double-blind, placebo-controlled, parallel-group study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics effects of a single dose of VSA001 injection in Chinese healthy adult volunteers. Eligible enrolled participants will initially receive VSA001 injection at the assigned dose level.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| VSA001 injection | Active Comparator | A single dose of active drug (VSA001, low or high dose) will be administered by subcutaneous injection on Day 1. |
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| Placebo | Placebo Comparator | The placebo is normal saline (0.9%) administered subcutaneously; volume matched to the corresponding VSA001 dose volume. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| VSA001 injection | Drug | The active drug is VSA001 injection. The active pharmaceutical ingredient (API) contained in VSA001 is a synthetic, double-stranded, hepatocyte targeted NAG-conjugated RNAi. |
| Measure | Description | Time Frame |
|---|---|---|
| Safety and tolerability | Incidence, frequency, and severity of adverse events (AEs) and serious adverse events (SAEs), and the relationship with VSA-01001. Clinically significant abnormalities in laboratory tests, vital signs, physical examination, 12-lead electrocardiograms (ECG). | 85 days |
| Measure | Description | Time Frame |
|---|---|---|
| Pharmacokinetics parameter: Cmax | Maximum plasma concentration (Cmax) | 48 hours |
| Pharmacokinetics parameter: Tmax | Time to maximum plasma concentration (Tmax) |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Peking University Third Hospital | Beijing | Beijing Municipality | China |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41094680 | Derived | Wang F, You D, Niu X, Shi J, Li Y, Qi L, Li H. Safety, pharmacokinetics and pharmacodynamics of Plozasiran in Chinese healthy volunteers. Cardiovasc Diabetol. 2025 Oct 15;24(1):399. doi: 10.1186/s12933-025-02929-9. |
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| Placebo | Drug | 0.9% Saline, volume matched |
|
| 48 hours |
| Pharmacokinetics parameter: AUC0-t | Area under the plasma concentration-time curve from the time 0 to the last quantifiable time point (AUC0-t). | 48 hours |
| Pharmacokinetics parameter: t1/2 | Half-life (t1/2). | 48 hours |
| Pharmacokinetics parameter: CL/F | Apparent clearance (CL/F). | 48 hours |
| Pharmacokinetics parameter: Vz/F | Apparent volume of distribution (Vz/F). | 48 hours |
| Pharmacodynamic (PD) parameters | Change from baseline over time in fasting serum APOC3 and triglycerides (TGs). | 85 days |