Phase 2
N=229
Study to Evaluate ARO-APOC3 in Adults With Severe Hypertriglyceridemia
Severe Hypertriglyceridemia
Bottom Line
View on ClinicalTrials.gov: NCT04720534 ↗Enrolled (actual)
229
Serious AEs
10.2%
Results posted
Mar 2026
Primary outcome: Primary: Percent Change From Baseline at Week 24 in Fasting Triglycerides (TG) — -17.2; -66.0; -70.2; -74.2 percentage change — p=< 0.0001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- ARO-APOC3 (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Arrowhead Pharmaceuticals
- Primary completion
- Mar 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change From Baseline at Week 24 in Fasting Triglycerides (TG) |
-17.2; -66.0; -70.2; -74.2 | < 0.0001 sig |
| SECONDARY Percent Change From Baseline Over Time Through Week 48 in Fasting TG |
7.2; -63.3; -79.5; -77.6; -0.7; -54.0 | < 0.0001 sig |
| SECONDARY Percent Change From Baseline at Week 24 in Apolipoprotein (Apo)C-III |
114.2; -69.6; -73.7; -79.4 | 0.076 |
| SECONDARY Percent Change From Baseline Over Time Through Week 48 in ApoC-III |
209.3; -67.3; -84.4; -84.6; 146.3; -59.8 | 0.0078 sig |
| SECONDARY Percent Change From Baseline at Week 24 in Fasting Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) |
-1.5; -29.4; -28.4; -21.7 | < 0.0001 sig |
| SECONDARY Percent Change From Baseline Over Time Through Week 48 in Fasting Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) |
6.3; -27.0; -34.7; -26.1; 4.5; -19.7 | <0.0001 sig |
| SECONDARY Percent Change From Baseline at Week 24 in Fasting High-Density Lipoprotein Cholesterol (HDL-C) |
10.6; 54.2; 62.8; 67.6 | < 0.0001 sig |
| SECONDARY Percent Change From Baseline Over Time Through Week 48 in Fasting HDL-C |
4.3; 53.4; 76.2; 88.1; 6.2; 45.4 | <0.0001 sig |
| SECONDARY Percent Change From Baseline at Week 24 in Fasting Total Apolipoprotein B (ApoB) |
8.0; 6.0; -5.3; 0.7 | 0.8155 |
| SECONDARY Percent Change From Baseline Over Time Through Week 48 in Fasting Total ApoB |
16.2; -2.3; -9.0; -1.9; 16.0; -0.8 | 0.0587 |
| SECONDARY Percent Change From Baseline at Week 24 in Fasting Low-Density Lipoprotein-Cholesterol (LDL-C) |
-5.5; 301.9; -15.4; 2.0; 18.0; 49.0 | 0.0737 |
| SECONDARY Percent Change From Baseline Over Time Through Week 48 in Fasting LDL-C |
-29.3; 267.3; -13.5; 0.6; -11.3; 297.4 | 0.0843 |
| SECONDARY Change From Baseline in Plasma Concentrations of ARO-APOC3 Over TimeThrough Week 12 |
0.000; 0.000; 0.000; 10.258; 21.207; 34.352 | — |
| SECONDARY Number of Participants With Treatment-Emergent Adverse Events (TEAEs) |
43; 43; 36; 49; 8; 13 | — |
Summary
The purpose of AROAPOC3-2001 is to evaluate the efficacy and safety of ARO-APOC3 in participants with severe hypertriglyceridemia. Participants will receive 2 subcutaneous injections of ARO-APOC3.
Eligibility Criteria
Inclusion Criteria
- Based on medical history, evidence of triglycerides (TG) ≥ 500 mg/dL and ≤ 4000 mg/dL at Screening
- Fasting TG ≥ 500 mg/dL at Screening
- Willing to follow diet counseling per Investigator judgment based on local standard of care
- Women of childbearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception
- Willing to provide written informed consent and to comply with study requirements
Exclusion Criteria
- Active pancreatitis within 12 weeks prior to first dose
- Any planned bariatric surgery or similar procedures to induce weight loss from consent to end of study
- Acute coronary syndrome event within 24 weeks of first dose
- Major surgery within 12 weeks of first dose
- Planned coronary intervention (e.g., stent placement or heart bypass) or any non-cardiac major surgical procedure throughout the study
- Uncontrolled hypertension
- Human immunodeficiency virus (HIV) infection, seropositive for Hepatitis B (HBV), seropositive for Hepatitis C (HCV)
- Uncontrolled hypothyroidism or hyperthyroidism
- Hemorrhagic stroke within 24 weeks of first dose
- Malignancy within the last 2 years prior to date of consent requiring systemic treatment (some exceptions apply)
Note: additional inclusion/exclusion criteria may apply per protocol
Data sourced from ClinicalTrials.gov (NCT04720534). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.