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The purpose of this study is to evaluate the safety, tolerability, pharmacokinetcs and pharmacodynamics of single- and multiple doses of ARO-ANG3 in healthy adult volunteers and in dyslipidemic patients including familial hypercholesterolemia and severe hypertriglyceridemia.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ARO-ANG3 | Experimental |
| |
| Placebo | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ARO-ANG3 | Drug | single or multiple doses of ARO-ANG3 by subcutaneous (sc) injections |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants with Adverse Events (AEs) Possibly or Probably Related to Treatment | Up to 113 (+/- 3 days) post-dose |
| Measure | Description | Time Frame |
|---|---|---|
| Pharmacokinetics (PK) of ARO-ANG3: Maximum Observed Plasma Concentration (Cmax) | Single dose phase: Up to 48 hours post-dose | |
| PK of ARO-ANG3: Time to Maximum Plasma Concentration (Tmax) | Single dose phase: Up to 48 hours post-dose |
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Inclusion Criteria:
Exclusion Criteria:
NOTE: additional inclusion/exclusion criteria may apply, per protocol
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Research Site 2 | Camperdown | New South Wales | 2050 | Australia | ||
| Research Site 3 |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41946917 | Derived | Watts GF, Scott R, Sullivan D, Baker J, Clifton P, Hamilton J, Given B, Melquist S, Zhou R, Gaudet D, Goldberg IJ, Knowles JW, Leeper NJ, Hegele RA, Ballantyne CM. Zodasiran for cholesterol and triglyceride lowering in patients with hyperlipidemia: final report of phase 1 basket trial. Nat Med. 2026 Apr;32(4):1432-1443. doi: 10.1038/s41591-026-04307-8. Epub 2026 Apr 7. | |
| 37626170 |
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| ID | Term |
|---|---|
| D050171 | Dyslipidemias |
| D006938 | Hyperlipoproteinemia Type II |
| D015228 | Hypertriglyceridemia |
| ID | Term |
|---|---|
| D052439 | Lipid Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D008052 | Lipid Metabolism, Inborn Errors |
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| sterile normal saline (0.9% NaCl) |
| Drug |
calculated volume to match active treatment |
|
| PK of ARO-ANG3: Terminal Elimination Half-Life (t1/2) | Single dose phase: Up to 48 hours post-dose |
| PK of ARO-ANG3: Area Under the Plasma Concentration Versus Time Curve From Zero to 24 Hours (AUC0-24) | Single dose phase: Up to 48 hours post-dose |
| PK of ARO-ANG3: Area Under the Plasma Concentration Versus Time Curve From Zero to infinity (AUCinf) | Single dose phase: Up to 48 hours post-dose |
| Reduction in Fasting Serum ANGPTL3 from Pre-Dose Baseline | Baseline, Up to Day 113 (+/- 3 days) |
| Adelaide |
| South Australia |
| 5000 |
| Australia |
| Research Site 1 | Nedlands | 6009 | Australia |
| Research Site 4 | Grafton | Auckland | 1010 | New Zealand |
| Research Site 5 | Papatoetoe | Aukland | 2025 | New Zealand |
| Research Site 6 | Christchurch | 8011 | New Zealand |
| Derived |
| Watts GF, Schwabe C, Scott R, Gladding PA, Sullivan D, Baker J, Clifton P, Hamilton J, Given B, Melquist S, Zhou R, Chang T, San Martin J, Gaudet D, Goldberg IJ, Knowles JW, Hegele RA, Ballantyne CM. RNA interference targeting ANGPTL3 for triglyceride and cholesterol lowering: phase 1 basket trial cohorts. Nat Med. 2023 Sep;29(9):2216-2223. doi: 10.1038/s41591-023-02494-2. Epub 2023 Aug 25. |
| 33900278 | Derived | Ying Q, Chan DC, Watts GF. Angiopoietin-like protein 3 inhibitors and contemporary unmet needs in lipid management. Curr Opin Lipidol. 2021 Jun 1;32(3):210-212. doi: 10.1097/MOL.0000000000000747. No abstract available. |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D006951 | Hyperlipoproteinemias |
| D006949 | Hyperlipidemias |