Significant reduction in ANGPLT3 levels is expected to reduce plasma LDL-C and TG levels and may lead to the reduction of risk of coronary heart disease and cardiovascular events associated with hypertriglyceridemia and LDL-C in subjects with persistent dyslipidemia. In this clinical study, the efficacy and safety of VSA003, an RNAi-based therapeutic targeting ANGPTL3, will be evaluated in subjects with HoFH.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
45
Percent change from baseline in serum LDL-C at month 6
Time frame: At month 6
Percent change and absolute change in serum ANGPTL3 levels from baseline at month 6
Time frame: At month 6
Percent change and absolute change in serum LDL-C, ApoB, non-HDL-C, VLDL-C, TC and TG levels from baseline
Time frame: Up to month 6, Up to month 18
Percent change and absolute change in serum HDL-C, ApoC-III, ApoC-II, ApoA-I, ApoA-V and Lp[a] levels from baseline
Time frame: Up to month 6, Up to month 18
Percentage of patients achieving ≥30% reduction in serum LDL-C from baseline
Time frame: At month 6, At month 18
Percentage of patients achieving ≥50% reduction in serum LDL-C from baseline
Time frame: At month 6, At month 18
Number of Participants with Adverse Events
Time frame: Up to month 6, Up to month 18
Number of Participants with Anti-Drug Antibodies (ADAs) to VSA003 Over Time
Time frame: Up to month 6, Up to month 18
Correlation between VSA003 serum concentrations and QT intervals
Time frame: Up to month 6, Up to month 18
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