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The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple doses of ARO-APOC3 in healthy adult volunteers and in patients with severe hypertriglyceridemia and familial chylomicronemia syndrome (FCS).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ARO-APOC3 | Experimental |
| |
| Placebo | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ARO-APOC3 | Drug | single or multiple doses of ARO-APOC3 by subcutaneous (sc) injections |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants with Adverse Events (AEs) Possibly or Probably Related to Treatment | Up to Day 113 (+/- 3 days) |
| Measure | Description | Time Frame |
|---|---|---|
| Pharmacokinetics (PK) of ARO-APOC3 in Normal Healthy Volunteers (NHVs): Maximum Observed Plasma Concentration (Cmax) | Single dose phase: Up to 48 hours post-dose | |
| PK of ARO-APOC3 in NHVs: 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 5 |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 38320498 | Derived | Gaudet D, Clifton P, Sullivan D, Baker J, Schwabe C, Thackwray S, Scott R, Hamilton J, Given B, Melquist S, Zhou R, Chang T, San Martin J, Watts GF, Goldberg IJ, Knowles JW, Hegele RA, Ballantyne CM. RNA Interference Therapy Targeting Apolipoprotein C-III in Hypertriglyceridemia. NEJM Evid. 2023 Dec;2(12):EVIDoa2200325. doi: 10.1056/EVIDoa2200325. Epub 2023 Nov 17. |
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| sterile normal saline (0.9% NaCl) |
| Drug |
calculated volume to match active treatment |
|
| PK of ARO-APOC3 in NHVs: Terminal Elimination Half-Life (t1/2) | Single dose phase: Up to 48 hours post-dose |
| PK of ARO-APOC3 in NHVs: 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-APOC3 in NHVs: 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 APOC3 from Pre-Dose Baseline | Up to Day 113 (+/- 3 days) |
| Sippy Downs |
| Queensland |
| Australia |
| Research Site 3 | Adelaide | South Australia | 5000 | Australia |
| Research Site 4 | Perth | Washington | 6009 | Australia |
| Research Site 7 | London | Ontario | N6A 5B7 | Canada |
| Research Site 6 | Chicoutimi | Quebec | G7H 7K9 | Canada |
| Research Site 8 | Montreal | Quebec | H2W 1R7 | Canada |
| Research Site 9 | Grafton | Auckland | 1010 | New Zealand |
| Research Site 10 | Papatoetoe | Auckland | 2025 | New Zealand |
| Research Site 11 | Christchurch | 8011 | New Zealand |
| ID | Term |
|---|---|
| D015228 | Hypertriglyceridemia |
| D008072 | Hyperlipoproteinemia Type I |
| ID | Term |
|---|---|
| D006949 | Hyperlipidemias |
| D050171 | Dyslipidemias |
| D052439 | Lipid Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D008052 | Lipid Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D006951 | Hyperlipoproteinemias |
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