This is a single center, open-label study to evaluate the efficacy of AKCEA-ANGPTL3- LRX for reduction of low density lipoprotein cholesterol (LDL-C) levels in patients with Homozygous Familial Hypercholesterolemia (HoFH).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Single open-label cohort
Clinical Site
Québec, Quebec, Canada
Reduction of low density lipoprotein cholesterol (LDL-C).
Percent change in LDL-C from Baseline to week 14.
Time frame: 14 weeks
Effect of ISIS 703802 on plasma angiopoietin like 3 (ANGPTL3).
Absolute and percentage change in ANGPTL3 protein from Baseline to Week 14 will be summarized.
Time frame: 7 and 14 Weeks
Effect of ISIS 703802 on lipid parameters.
Absolute and percentage change from Baseline to Week 14 will be summarized.
Time frame: 7 and 14 Weeks
Evaluate plasma trough levels of ISIS 703802.
Plasma trough levels of ISIS 703802 during treatment period and those during post-treatment follow up period will be descriptively summarized.
Time frame: 14 Weeks
The safety of ISIS 703802 by the incidence of treatment-emergent adverse events
The safety of ISIS 703802 will be assessed by determining adverse effects.
Time frame: 14 Weeks
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