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
46
Peking Union Medical College Hospital
Beijing, China
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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