Study of ARO-ANG3 in Adults With Mixed Dyslipidemia

NCT04832971 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: AROANG3-2001
• Study Type: interventional
• Target: 204
• Locations: 4 countries
• Sites: 24

Brief Summary

The purpose of AROANG3-2001 is to evaluate the efficacy and safety of ARO-ANG3 in participants with mixed dyslipidemia. Participants will initially receive 2 subcutaneous injections of ARO-ANG3 or placebo. Participants who complete the double-blind treatment period may opt to continue in an open-label extension during which they will receive up to 8 doses of ARO-ANG3.

Conditions

Mixed Dyslipidemia

Outcome Measures

Primary Outcomes (1)

• Percent Change From Baseline in Fasting TG at Week 24

  Baseline, Week 24

Secondary Outcomes (21)

• Percent Change From Baseline in Fasting TG Over Time

  Baseline, up to Week 36 (double-blind treatment period)

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

  Baseline, Week 24

• Percent Change From Baseline in Fasting Non-HDL-C Over Time

  Baseline, up to Week 36 (double-blind treatment period)

• Percent Change From Baseline in Fasting Total Apolipoprotein B (ApoB) at Week 24

  Baseline, Week 24

• Percent Change From Baseline in Fasting Total ApoB Over Time

  Baseline, up to Week 36 (double-blind treatment period)

Study Arms (4)

ARO-ANG3 50 mg

EXPERIMENTAL

Two doses of ARO-ANG3 by subcutaneous (sc) injection at Day 1 and Week 12 during double-blind treatment period. Up to 8 doses of ARO-ANG3 by sc injection during the open-label extension period.

Drug: ARO-ANG3

ARO-ANG3 100 mg

EXPERIMENTAL

Two doses of ARO-ANG3 bysc injection at Day 1 and Week 12 during double-blind treatment period. Up to 8 doses of ARO-ANG3 by sc injection during the open-label extension period.

Drug: ARO-ANG3

ARO-ANG3 200 mg

EXPERIMENTAL

Two doses of ARO-ANG3 by sc injection at Day 1 and Week 12 during double-blind treatment period. Up to 8 doses of ARO-ANG3 by sc injection during the open-label extension period.

Drug: ARO-ANG3

Placebo

PLACEBO COMPARATOR

Calculated volume to match active treatment by sc injection at Day 1 and Week 12 during the double-blind treatment period. Up to 8 doses of ARO-ANG3 by sc injection during the open-label extension period.

Drug: ARO-ANG3; Drug: Placebo

Interventions

ARO-ANG3 DRUG

ARO-ANG3 Injection

ARO-ANG3 100 mg; ARO-ANG3 200 mg; ARO-ANG3 50 mg; Placebo

Placebo DRUG

Sterile Normal Saline (0.9% NaCl)

Placebo

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) ≥ 150 mg/dL but ≤ 499 mg/dL
• Fasting levels at Screening of LDL-C ≥ 70 mg/dL OR non-HDL-C ≥ 100 mg/dL after at least 4 weeks of stable diet and stable optimal statin therapy
• Mean fasting TG ≥ 150 mg/dL and ≤ 499 mg/dL during Screening collected at two separate and consecutive visits and at least 7 days apart and not more than 17 days apart
• Willing to follow diet counseling and maintain a stable diet per Investigator judgment based on local standard of care
• Participants of childbearing potential must agree to use highly-effective contraception during the study and for at least 24 weeks from last dose of study medication
• Women of childbearing potential must have a negative pregnancy test and cannot be breastfeeding
• Women of childbearing potential on hormonal contraceptives must be stable on the medication for ≥ 2 menstrual cycles prior to Day 1
• Men must not donate sperm during the study and for at least 24 weeks following the last dose of study medication
• Able and willing to provide written informed consent and to comply with study requirements

You may not qualify if:

• Current use or use within 365 days from Day 1 of any hepatocyte targeted siRNA or antisense oligonucleotide molecule
• Active pancreatitis within 12 weeks prior to Day 1
• 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 Day 1
• Major surgery within 12 weeks of Day 1 or planned surgery during the study
• Planned coronary intervention (e.g., stent placement or heart bypass) during 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 Day 1
• History of bleeding diathesis or coagulopathy
• Current diagnosis of nephrotic syndrome
• Systemic use of corticosteroids or anabolic steroids within 4 weeks prior to Day 1 or planned use during the study
• Malignancy within the last 2 years prior to date of consent requiring systemic treatment (some exceptions apply)

Sponsors & Collaborators

• Arrowhead Pharmaceuticals: lead

Study Sites (24)

Research Site 5

Huntington Park, California, 90255, United States

Location

Research Site 7

Hialeah, Florida, 33012, United States

Location

Research Site 17

Miami, Florida, 33144, United States

Location

Research Site 8

Port Orange, Florida, 32127, United States

Location

Research Site 24

Minneapolis, Minnesota, 55455, United States

Location

Research Site 10

Omaha, Nebraska, 68114, United States

Location

Research Site 23

Las Vegas, Nevada, 89121, United States

Location

Research Site 22

New York, New York, 10029, United States

Location

Research Site 15

Greensboro, North Carolina, 27408, United States

Location

Research Site 2

Morehead City, North Carolina, 28557, United States

Location

Research Site 1

Marion, Ohio, 43302, United States

Location

Research Site 4

Camp Hill, Pennsylvania, 17011, United States

Location

Research Site 11

Houston, Texas, 77030, United States

Location

Research Site 6

Blacktown, New South Wales, 2148, Australia

Location

Research Site 21

Sippy Downs, Queensland, 4556, Australia

Location

Research Site 18

Nedlands, 6009, Australia

Location

Research Site 25

London, Ontario, N6A 5A5, Canada

Location

Research Site 16

Chicoutimi, Quebec, G7H 7K9, Canada

Location

Research Site 12

Québec, G1G 3Z4, Canada

Location

Research Site 9

Québec, H7T2P5, Canada

Location

Research Site 19

Birkenhead, 0626, New Zealand

Location

Research Site 13

Christchurch, 8011, New Zealand

Location

Research Site 20

Hamilton, 3200, New Zealand

Location

Research Site 14

Rotorua, 3010, New Zealand

Location

Related Publications (2)

• Rosenson RS, Gaudet D, Hegele RA, Ballantyne CM, Nicholls SJ, Lucas KJ, San Martin J, Zhou R, Muhsin M, Chang T, Hellawell J, Watts GF; ARCHES-2 Trial Team. Zodasiran, an RNAi Therapeutic Targeting ANGPTL3, for Mixed Hyperlipidemia. N Engl J Med. 2024 Sep 12;391(10):913-925. doi: 10.1056/NEJMoa2404147. Epub 2024 May 29.

  PMID: 38809174 DERIVED

• Dimitriadis K, Theofilis P, Iliakis P, Pyrpyris N, Dri E, Sakalidis A, Soulaidopoulos S, Tsioufis P, Fragkoulis C, Chrysohoou C, Tsiachris D, Tsioufis K. Management of dyslipidemia in coronary artery disease: the present and the future. Coron Artery Dis. 2024 Sep 1;35(6):516-524. doi: 10.1097/MCA.0000000000001375. Epub 2024 Apr 29.

  PMID: 38682459 DERIVED

Results Point of Contact

• Title: Patrick O'Brien, Chief Operating Officer
• Organization: Arrowhead Pharmaceuticals, Inc.

PObrien@arrowheadpharma.com

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: April 2, 2021
• First Posted: April 6, 2021
• Study Start: June 28, 2021
• Primary Completion: August 30, 2022
• Study Completion: September 25, 2024
• Last Updated: December 3, 2025
• Results First Posted: January 16, 2024

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

NZ (4); US (13); AU (3); CA (4)