Participants with documented homozygous familial hypercholesterolemia (HoFH) who have provided informed consent will receive 2 open-label doses of ARO-ANG3 and be evaluated for safety and efficacy parameters through 36 weeks. Participants who complete the first 36 week treatment period may opt to continue in an additional 24-month extension period during which they will receive up to 8 doses open-label doses of ARO-ANG3.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
18
Participants will be randomized to receive ARO-ANG3 SC on Day 1 and Day 84 during the initial 36 Weeks of the study and on Day1 and Months 3, 6, 9, 12, 15, 18, and 21 of the extension period
Research Site 4
Mount Sinai, New York, United States
Research Site 5
Cincinnati, Ohio, United States
Research Site 8
Camperdown, New South Wales, Australia
Research Site 3
Nedlands, Western Australia, Australia
Percent Change from Baseline in Fasting Calculated Low-Density Lipoprotein-Cholesterol (LDL-C) and LDL-C by Preparative Ultracentrifugation (LDL-C [PUC]) up to Week 24
Time frame: Baseline, up to Week 24
Percent Change from Baseline in Fasting LDL-C (PUC) Over Time
Time frame: Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period)
Absolute Change from Baseline in Fasting LDL-C (PUC) Over Time
Time frame: Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period)
Percent Change from Baseline in Fasting Calculated LDL-C Over Time
Time frame: Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period)
Absolute Change from Baseline in Fasting Calculated LDL-C Over Time
Time frame: Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period)
Percent Change from Baseline in Fasting Angiopoietin-like 3 (ANGPTL3) Over Time
Time frame: Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period)
Absolute Change from Baseline in Fasting ANGPTL3 Over Time
Time frame: Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period)
Percent Change from Baseline in Fasting Total Apolipoprotein B (ApoB) Over Time
Time frame: Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period)
Absolute Change from Baseline in Fasting Total ApoB Over Time
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Research Site 2
Chicoutimi, Quebec, Canada
Research Site 1
Québec, Quebec, Canada
Research Site 7
Johannesburg, South Africa
Time frame: Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period)
Percent Change from Baseline in Fasting High-Density Lipoprotein-Cholesterol (HDL-C) Over Time
Time frame: Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period) 36
Absolute Change from Baseline in Fasting HDL-C Over Time
Time frame: Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period)
Percent Change from Baseline in Fasting Non-High-Density Lipoprotein-Cholesterol (Non-HDL-C) Over Time
Time frame: Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period)
Absolute Change from Baseline in Fasting Non-HDL-C Over Time
Time frame: Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period)
Percent Change from Baseline in Fasting Very-Low-Density Lipoprotein-Cholesterol (VLDL-C) Over Time
Time frame: Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period)
Absolute Change from Baseline in VLDL-C Over Time
Time frame: Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period)
Percent Change from Baseline in Fasting Total Cholesterol (TC) Over Time
Time frame: Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period)
Absolute Change from Baseline in Fasting TC Over Time
Time frame: Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period)
Percent Change from Baseline in Fasting Triglycerides (TG) Over Time
Time frame: Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period)
Absolute Change from Baseline in Fasting TG Over Time
Time frame: Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period)
Number of Participants with Treatment Emergent Adverse Events (TEAEs)
Time frame: Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period)
Number of Participants with Anti-Drug Antibodies (ADAs) to ARO-ANG3 Over Time
Time frame: Baseline, up to Week 36 (initial treatment period), up to Month 24 (extension period)
Proportion of Participants meeting United States National Lipid Association Apheresis Eligibility Criteria of LDL-C ≥ 300 mg/dL at Week 24
Time frame: Week 24
Proportion of Participants Meeting European Union (EU) Apheresis Eligibility Criteria per German Apheresis Working Group at Week 24
Time frame: Week 24