A VSA003 Phase 1 Study in Chinese Adult Healthy Volunteers

A Phase 1 Single Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamic Effects of VSA003 in Chinese Adult Healthy Volunteers

This is a randomized, double blinded, phase 1 study. The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single dose of VSA003 in healthy adult volunteerst

Study Overview

• Status: Completed
• Conditions: Dyslipidemias; Hypertriglyceridemia; Familial Hypercholesterolemia
• Intervention / Treatment: Drug: VSA003; Drug: 0.9% NaCl

• Study Type: Interventional
• Enrollment (Actual): 36
• Phase: Phase 1

Contacts and Locations

Study Locations

• China
  • Beijing Municipality
    • Beijing, Beijing Municipality, China
      • Peking Union Medical College Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult
• Accepts Healthy Volunteers: Yes

Description

Inclusion Criteria:

• Women of child bearing potential must have a negative pregnancy test, cannot be breastfeeding and must be willing to use contraception
• BMI 18.0~28.0 kg/m2
• Willing to provide written informed consent and to comply with study requirements
• On a stable diet for at least 4 weeks with no plans to significantly alter diet or weight over course of study
• TG> 100 mg/dL
• LDL-C> 70 mg/dL

Exclusion Criteria:

• Clinically significant health concerns
• Regular use of alcohol within one month prior to screening
• Recent (within 3 months) use of illicit drugs
• Female with pregnancy or breastfeeding
• QTcF>450 ms in ECG
• Donation or loss of whole blood more than 400 ml prior to administration of the study treatment

Note: additional inclusion/exclusion criteria may apply, per protocol

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: VSA003; single dose of VSA003 by subcutaneous (sc) injections: 50 mg, 100 mg, 200 mg	Drug: VSA003; sequential dosing, SC, single dose: 50 mg, 100 mg, 200 mg
Placebo Comparator: placebo; sterile normal saline (0.9% NaCl) calculated volume to match active treatment	Drug: 0.9% NaCl; placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Frequency and severity of adverse event (AE) and serious adverse event (SAE)	safety and tolerability	Up to 85±3 days post-dose

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Maximum observed concentration (Cmax) of VSA003	pharmacokinetics (PK)	Up to 48 hours post dose
Time of occurrence of Cmax (tmax) of VSA003	PK	Up to 48 hours post dose
Apparent terminal phase half-life (t1/2) of VSA003	PK	Up to 48 hours post dose
Area under the concentration-time curve from time zero (pre-dose) to the last quantifiable concentration (AUC0-t) of VSA003	PK	Up to 48 hours post dose
Apparent clearance (CL/F) of VSA003	PK	Up to 48 hours post dose
Apparent terminal phase volume of distribution (Vz/F) of VSA003	PK	Up to 48 hours post dose
Change of fasting serum ANGPTL3 from pre-dose baseline	PD	Up to 85±3 days post-dose
Anti-drug Antibodies (ADA) to VSA003	immunogenecity	Up to 85±3 days post-dose

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change of low density lipoprotein cholesterol (LDL-C) from pre-dose baseline	Lipid profile	Up to 85±3 days post-dose
Change of total cholesterol (TC) from pre-dose baseline	Lipid profile	Up to 85±3 days post-dose
Change of triglyceride (TG) from pre-dose baseline	Lipid profile	Up to 85±3 days post-dose
Change of high density lipoprotein cholesterol (HDL-C) from pre-dose baseline	Lipid profile	Up to 85±3 days post-dose
Change of fasting glucose from pre-dose baseline	glucose metabolism	Up to 85±3 days post-dose
Change of HbA1c from pre-dose baseline	glucose metabolism	Up to 85±3 days post-dose
Change of fasting C peptide from pre-dose baseline	glucose metabolism	Up to 85±3 days post-dose
Change of fasting insulin from pre-dose baseline	glucose metabolism	Up to 85±3 days post-dose

Collaborators and Investigators

• Sponsor: Visirna Therapeutics HK Limited

Study record dates

Study Major Dates

• Study Start (Actual): June 1, 2023
• Primary Completion (Actual): May 7, 2024
• Study Completion (Actual): May 7, 2024

Study Registration Dates

• First Submitted: April 26, 2023
• First Submitted That Met QC Criteria: May 5, 2023
• First Posted (Actual): May 9, 2023

Study Record Updates

• Last Update Posted (Estimated): September 12, 2025
• Last Update Submitted That Met QC Criteria: September 5, 2025
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases; Hyperlipidemias; Lipid Metabolism Disorders; Lipid Metabolism, Inborn Errors; Hyperlipoproteinemias; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nutritional and Metabolic Diseases; Dyslipidemias; Hyperlipoproteinemia Type II; Hypertriglyceridemia; Pharmaceutical Preparations; Crystalloid Solutions; Isotonic Solutions; Solutions; Saline Solution
• Other Study ID Numbers: VSA003-1001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No