Dyslipidemias, Familial Hypercholesterolemia, Hypertriglyceridemia
Conditions
Brief summary
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
Interventions
DRUGVSA003
sequential dosing, SC, single dose: 50 mg, 100 mg, 200 mg
DRUG0.9% NaCl
placebo
Sponsors
Visirna Therapeutics HK Limited
Study design
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 55 Years
Healthy volunteers
Yes
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/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Frequency and severity of adverse event (AE) and serious adverse event (SAE) | Up to 85±3 days post-dose | safety and tolerability |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change of fasting serum ANGPTL3 from pre-dose baseline | Up to 85±3 days post-dose | PD |
| Anti-drug Antibodies (ADA) to VSA003 | Up to 85±3 days post-dose | immunogenecity |
| Maximum observed concentration (Cmax) of VSA003 | Up to 48 hours post dose | pharmacokinetics (PK) |
| Time of occurrence of Cmax (tmax) of VSA003 | Up to 48 hours post dose | PK |
| Apparent terminal phase half-life (t1/2) of VSA003 | Up to 48 hours post dose | PK |
| Area under the concentration-time curve from time zero (pre-dose) to the last quantifiable concentration (AUC0-t) of VSA003 | Up to 48 hours post dose | PK |
| Apparent clearance (CL/F) of VSA003 | Up to 48 hours post dose | PK |
| Apparent terminal phase volume of distribution (Vz/F) of VSA003 | Up to 48 hours post dose | PK |
Other
| Measure | Time frame | Description |
|---|---|---|
| Change of total cholesterol (TC) from pre-dose baseline | Up to 85±3 days post-dose | Lipid profile |
| Change of triglyceride (TG) from pre-dose baseline | Up to 85±3 days post-dose | Lipid profile |
| Change of high density lipoprotein cholesterol (HDL-C) from pre-dose baseline | Up to 85±3 days post-dose | Lipid profile |
| Change of fasting glucose from pre-dose baseline | Up to 85±3 days post-dose | glucose metabolism |
| Change of HbA1c from pre-dose baseline | Up to 85±3 days post-dose | glucose metabolism |
| Change of fasting C peptide from pre-dose baseline | Up to 85±3 days post-dose | glucose metabolism |
| Change of fasting insulin from pre-dose baseline | Up to 85±3 days post-dose | glucose metabolism |
| Change of low density lipoprotein cholesterol (LDL-C) from pre-dose baseline | Up to 85±3 days post-dose | Lipid profile |
Countries
China
Outcome results
None listed