Study to Evaluate ARO-APOC3 in Adults With Severe Hypertriglyceridemia

NCT04720534 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: AROAPOC3-2001
• Study Type: interventional
• Target: 229
• Locations: 8 countries
• Sites: 74

Brief 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.

Conditions

Severe Hypertriglyceridemia

Outcome Measures

Primary Outcomes (1)

• Percent Change From Baseline at Week 24 in Fasting Triglycerides (TG)

  Baseline, Week 24

Secondary Outcomes (13)

• Percent Change From Baseline Over Time Through Week 48 in Fasting TG

  Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 36, 48/early termination (ET)

• Percent Change From Baseline at Week 24 in Apolipoprotein (Apo)C-III

  Baseline, Week 24

• Percent Change From Baseline Over Time Through Week 48 in ApoC-III

  Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 36, 48/early termination (ET)

• Percent Change From Baseline at Week 24 in Fasting Non-High-Density Lipoprotein Cholesterol (Non-HDL-C)

  Baseline, Week 24

• Percent Change From Baseline Over Time Through Week 48 in Fasting Non-High-Density Lipoprotein Cholesterol (Non-HDL-C)

  Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 36, 48/early termination (ET)

Study Arms (4)

Placebo, Day 1 and Week 12

EXPERIMENTAL

Participants received a total of 2 placebo SC injections on Day 1 and Week 12 for a total of 2 injections.

Drug: Placebo

ARO-APOC3 10 mg

EXPERIMENTAL

Participants received a total of 2 ARO-APOC3 10 mg SC injections on Day 1 and Week 12 for a total of 2 injections.

Drug: ARO-APOC3

ARO-APOC3 25 mg

EXPERIMENTAL

Participants received a total of 2 ARO-APOC3 25 mg SC injections on Day 1 and Week 12 for a total of 2 injections.

Drug: ARO-APOC3

ARO-APOC3 50 mg

EXPERIMENTAL

Participants received a total of 2 ARO-APOC3 50 mg SC injections on Day 1 and Week 12 for a total of 2 injections.

Drug: ARO-APOC3

Interventions

ARO-APOC3 DRUG

2 doses of ARO-APOC3 by subcutaneous (sc) injection

ARO-APOC3 10 mg; ARO-APOC3 25 mg; ARO-APOC3 50 mg

Placebo DRUG

calculated volume to match active treatment by sc injection

Placebo, Day 1 and Week 12

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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)

Sponsors & Collaborators

• Arrowhead Pharmaceuticals: lead

Study Sites (74)

Research Site 2

Beverly Hills, California, 90211, United States

Location

Research Site 4

Palm Springs, California, 92292, United States

Location

Research Site 5

Boca Raton, Florida, 33434, United States

Location

Research Site 6

Fort Lauderdale, Florida, 33308, United States

Location

Research Site 10

Miami, Florida, 33144, United States

Location

Research Site 9

Miami, Florida, 33144, United States

Location

Research Site 11

Miami, Florida, 33155, United States

Location

Research Site 8

Miami, Florida, 33173, United States

Location

Research Site 12

Miami Springs, Florida, 33166, United States

Location

Research Site 14

Port Orange, Florida, 32127, United States

Location

Research Site 16

Dunwoody, Georgia, 30338, United States

Location

Research Site 17

Arlington Heights, Illinois, 60005, United States

Location

Research Site 18

Lexington, Kentucky, 40503, United States

Location

Research Site 20

Minneapolis, Minnesota, 55455, United States

Location

Research Site 21

Tupelo, Mississippi, 38801, United States

Location

Research Site 22

Kalispell, Montana, 59901, United States

Location

Research Site 23

Omaha, Nebraska, 68114, United States

Location

Research Site 24

Las Vegas, Nevada, 89121, United States

Location

Research Site 25

Long Island City, New York, 11106, United States

Location

Research Site 26

New Windsor, New York, 12553, United States

Location

Research Site 27

New York, New York, 10029, United States

Location

Research Site 29

Morehead City, North Carolina, 28557, United States

Location

Research Site 30

Fargo, North Dakota, 58104, United States

Location

Research Site 31

Dayton, Ohio, 45419, United States

Location

Research Site 32

Franklin, Ohio, 45005, United States

Location

Research Site 33

Maumee, Ohio, 43537, United States

Location

Research Site 34

Oklahoma City, Oklahoma, 73135, United States

Location

Research Site 35

Greenville, South Carolina, 29607, United States

Location

Research Site 36

Chattanooga, Tennessee, 37421, United States

Location

Research Site 38

Houston, Texas, 77030, United States

Location

Research Site 39

Houston, Texas, 77099, United States

Location

Research Site 40

San Antonio, Texas, 78249, United States

Location

Research Site 41

Manassas, Virginia, 20110, United States

Location

Research Site 43

Perth, Nedlands, 6009, Australia

Location

Research Site 42

Camperdown, New South Wales, 2050, Australia

Location

Research Site 44

Brisbane, Queensland, 4064, Australia

Location

Research Site 45

Sippy Downs, Queensland, 4556, Australia

Location

Research Site 46

Adelaide, South Australia, 5000, Australia

Location

Research Site 47

Clayton, Victoria, 3168, Australia

Location

Research Site 49

Concord, Ontario, L4K 4M2, Canada

Location

Research Site 50

London, Ontario, N6A 5A5, Canada

Location

Research Site 51

Toronto, Ontario, M4G 3E8, Canada

Location

Research Site 52

Chicoutimi, Quebec, G7H5H6, Canada

Location

Research Site 53

Greenfield, Quebec, J4V2G8, Canada

Location

Research Site 54

Montreal, Quebec, H2W2T2, Canada

Location

Research Site 55

Aachen, 52074, Germany

Location

Research Site 56

Leipzig, 0 4103, Germany

Location

Research Site 57

Baja, 6500, Hungary

Location

Research Site 58

Baja, 6500, Hungary

Location

Research Site 59

Balatonföldvár, H-8230, Hungary

Location

Research Site 60

Békéscsaba, 5600, Hungary

Location

Research Site 61

Debrecen, 4032, Hungary

Location

Research Site 62

Gyöngyös, 3200, Hungary

Location

Research Site 63

Komárom, 2921, Hungary

Location

Research Site 65

Nyíregyháza, 4400, Hungary

Location

Research Site 64

Nyíregyháza, 4405, Hungary

Location

Research Site 66

Amsterdam, 1105 AZ, Netherlands

Location

Research Site 67

Dordrecht, 3318 AT, Netherlands

Location

Research Site 68

Sneek, 08601, Netherlands

Location

Research Site 73

Grafton, Auckland, 1010, New Zealand

Location

Research Site 69

Christchurch, 8011, New Zealand

Location

Research Site 70

Hamilton, 3200, New Zealand

Location

Research Site 71

Papatoetoe, 2025, New Zealand

Location

Research Site 72

Rotorua, 3010, New Zealand

Location

Research Site 74

Bydgoszcz, 85-605, Poland

Location

Research Site 75

Elblag, 82-300, Poland

Location

Research Site 76

Gdynia, 81-157, Poland

Location

Research Site 83

Lodz, 94-048, Poland

Location

Research Site 78

Oświęcim, 32-600, Poland

Location

Research Site 79

Poznan, 61-655, Poland

Location

Research Site 80

Puławy, 24-100, Poland

Location

Research Site 81

Rzeszów, 35-055, Poland

Location

Research Site 82

Wołomin, 05-200, Poland

Location

Research Site 77

Łęczyca, 99-100, Poland

Location

Related Publications (1)

• Gaudet D, Pall D, Watts GF, Nicholls SJ, Rosenson RS, Modesto K, San Martin J, Hellawell J, Ballantyne CM. Plozasiran (ARO-APOC3) for Severe Hypertriglyceridemia: The SHASTA-2 Randomized Clinical Trial. JAMA Cardiol. 2024 Jul 1;9(7):620-630. doi: 10.1001/jamacardio.2024.0959.

  PMID: 38583092 DERIVED

MeSH Terms

Conditions

Hypertriglyceridemia

Condition Hierarchy (Ancestors)

Hyperlipidemias; Dyslipidemias; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Chief Operating Officer
• Organization: Arrowhead Pharmaceuticals, Inc.

info@arrowheadpharma.com

1 (626) 304-3400

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 19, 2021
• First Posted: January 22, 2021
• Study Start: May 31, 2021
• Primary Completion: March 9, 2023
• Study Completion: August 31, 2023
• Last Updated: March 24, 2026
• Results First Posted: March 24, 2026

Record last verified: 2026-03

Locations

AU (6); NZ (5); NL (3); US (33); DE (2); CA (6); HU (9); PL (10)