Study to Evaluate ARO-APOC3 in Adults With Severe Hypertriglyceridemia (SHASTA-2)

March 3, 2026 updated by: Arrowhead Pharmaceuticals

A Double-Blind, Placebo-Controlled Phase 2b Study to Evaluate the Efficacy and Safety of ARO-APOC3 in Adults With Severe Hypertriglyceridemia

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
229

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • Nedlands
      • Perth, Nedlands, Australia, 6009
        • Research Site 43
    • New South Wales
      • Camperdown, New South Wales, Australia, 2050
        • Research Site 42
    • Queensland
      • Brisbane, Queensland, Australia, 4064
        • Research Site 44
      • Sippy Downs, Queensland, Australia, 4556
        • Research Site 45
    • South Australia
      • Adelaide, South Australia, Australia, 5000
        • Research Site 46
    • Victoria
      • Clayton, Victoria, Australia, 3168
        • Research Site 47
  • Canada
    • Ontario
      • Concord, Ontario, Canada, L4K 4M2
        • Research Site 49
      • London, Ontario, Canada, N6A 5A5
        • Research Site 50
      • Toronto, Ontario, Canada, M4G 3E8
        • Research Site 51
    • Quebec
      • Chicoutimi, Quebec, Canada, G7H5H6
        • Research Site 52
      • Greenfield, Quebec, Canada, J4V2G8
        • Research Site 53
      • Montreal, Quebec, Canada, H2W2T2
        • Research Site 54
  • Germany
      • Aachen, Germany, 52074
        • Research Site 55
      • Leipzig, Germany, 0 4103
        • Research Site 56
  • Hungary
      • Baja, Hungary, 6500
        • Research Site 57
      • Baja, Hungary, 6500
        • Research Site 58
      • Balatonföldvár, Hungary, H-8230
        • Research Site 59
      • Békéscsaba, Hungary, 5600
        • Research Site 60
      • Debrecen, Hungary, 4032
        • Research Site 61
      • Gyöngyös, Hungary, 3200
        • Research Site 62
      • Komárom, Hungary, 2921
        • Research Site 63
      • Nyíregyháza, Hungary, 4400
        • Research Site 65
      • Nyíregyháza, Hungary, 4405
        • Research Site 64
  • Netherlands
      • Amsterdam, Netherlands, 1105 AZ
        • Research Site 66
      • Dordrecht, Netherlands, 3318 AT
        • Research Site 67
      • Sneek, Netherlands, 08601
        • Research Site 68
  • New Zealand
      • Christchurch, New Zealand, 8011
        • Research Site 69
      • Hamilton, New Zealand, 3200
        • Research Site 70
      • Papatoetoe, New Zealand, 2025
        • Research Site 71
      • Rotorua, New Zealand, 3010
        • Research Site 72
    • Auckland
      • Grafton, Auckland, New Zealand, 1010
        • Research Site 73
  • Poland
      • Bydgoszcz, Poland, 85-605
        • Research Site 74
      • Elblag, Poland, 82-300
        • Research Site 75
      • Gdynia, Poland, 81-157
        • Research Site 76
      • Lodz, Poland, 94-048
        • Research Site 83
      • Oświęcim, Poland, 32-600
        • Research Site 78
      • Poznan, Poland, 61-655
        • Research Site 79
      • Puławy, Poland, 24-100
        • Research Site 80
      • Rzeszów, Poland, 35-055
        • Research Site 81
      • Wołomin, Poland, 05-200
        • Research Site 82
      • Łęczyca, Poland, 99-100
        • Research Site 77
  • United States
    • California
      • Beverly Hills, California, United States, 90211
        • Research Site 2
      • Palm Springs, California, United States, 92292
        • Research Site 4
    • Florida
      • Boca Raton, Florida, United States, 33434
        • Research Site 5
      • Fort Lauderdale, Florida, United States, 33308
        • Research Site 6
      • Miami, Florida, United States, 33144
        • Research Site 10
      • Miami, Florida, United States, 33144
        • Research Site 9
      • Miami, Florida, United States, 33155
        • Research Site 11
      • Miami, Florida, United States, 33173
        • Research Site 8
      • Miami Springs, Florida, United States, 33166
        • Research Site 12
      • Port Orange, Florida, United States, 32127
        • Research Site 14
    • Georgia
      • Dunwoody, Georgia, United States, 30338
        • Research Site 16
    • Illinois
      • Arlington Heights, Illinois, United States, 60005
        • Research Site 17
    • Kentucky
      • Lexington, Kentucky, United States, 40503
        • Research Site 18
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • Research Site 20
    • Mississippi
      • Tupelo, Mississippi, United States, 38801
        • Research Site 21
    • Montana
      • Kalispell, Montana, United States, 59901
        • Research Site 22
    • Nebraska
      • Omaha, Nebraska, United States, 68114
        • Research Site 23
    • Nevada
      • Las Vegas, Nevada, United States, 89121
        • Research Site 24
    • New York
      • Long Island City, New York, United States, 11106
        • Research Site 25
      • New Windsor, New York, United States, 12553
        • Research Site 26
      • New York, New York, United States, 10029
        • Research Site 27
    • North Carolina
      • Morehead City, North Carolina, United States, 28557
        • Research Site 29
    • North Dakota
      • Fargo, North Dakota, United States, 58104
        • Research Site 30
    • Ohio
      • Dayton, Ohio, United States, 45419
        • Research Site 31
      • Franklin, Ohio, United States, 45005
        • Research Site 32
      • Maumee, Ohio, United States, 43537
        • Research Site 33
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73135
        • Research Site 34
    • South Carolina
      • Greenville, South Carolina, United States, 29607
        • Research Site 35
    • Tennessee
      • Chattanooga, Tennessee, United States, 37421
        • Research Site 36
    • Texas
      • Houston, Texas, United States, 77030
        • Research Site 38
      • Houston, Texas, United States, 77099
        • Research Site 39
      • San Antonio, Texas, United States, 78249
        • Research Site 40
    • Virginia
      • Manassas, Virginia, United States, 20110
        • Research Site 41

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Placebo, Day 1 and Week 12
Participants received a total of 2 placebo SC injections on Day 1 and Week 12 for a total of 2 injections.
Drug: Placebo
calculated volume to match active treatment by sc injection
Experimental: ARO-APOC3 10 mg
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
2 doses of ARO-APOC3 by subcutaneous (sc) injection
Experimental: ARO-APOC3 25 mg
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
2 doses of ARO-APOC3 by subcutaneous (sc) injection
Experimental: ARO-APOC3 50 mg
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
2 doses of ARO-APOC3 by subcutaneous (sc) injection

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Percent Change From Baseline at Week 24 in Fasting Triglycerides (TG)
Time Frame: Baseline, Week 24
Baseline, Week 24

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Percent Change From Baseline Over Time Through Week 48 in Fasting TG
Time Frame: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 36, 48/early termination (ET)
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
Time Frame: Baseline, Week 24
Baseline, Week 24
Percent Change From Baseline Over Time Through Week 48 in ApoC-III
Time Frame: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 36, 48/early termination (ET)
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)
Time Frame: Baseline, Week 24
Baseline, Week 24
Percent Change From Baseline Over Time Through Week 48 in Fasting Non-High-Density Lipoprotein Cholesterol (Non-HDL-C)
Time Frame: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 36, 48/early termination (ET)
Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 36, 48/early termination (ET)
Percent Change From Baseline at Week 24 in Fasting High-Density Lipoprotein Cholesterol (HDL-C)
Time Frame: Baseline, Week 24
Baseline, Week 24
Percent Change From Baseline Over Time Through Week 48 in Fasting HDL-C
Time Frame: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 36, 48/early termination (ET)
Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 36, 48/early termination (ET)
Percent Change From Baseline at Week 24 in Fasting Total Apolipoprotein B (ApoB)
Time Frame: Baseline, Week 24
Baseline, Week 24
Percent Change From Baseline Over Time Through Week 48 in Fasting Total ApoB
Time Frame: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 36, 48/early termination (ET)
Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 36, 48/early termination (ET)
Percent Change From Baseline at Week 24 in Fasting Low-Density Lipoprotein-Cholesterol (LDL-C)
Time Frame: Baseline, Week 24
LDL-C analyses used both Martin-Hopkins methodology and ultracentrifugation. The Martin-Hopkins formula is a method for calculating LDL-C that improves accuracy by using an adjustable factor for estimating very-low-density lipoprotein (VLDL) cholesterol cholesterol based on triglyceride and non-HDL cholesterol levels.
Baseline, Week 24
Percent Change From Baseline Over Time Through Week 48 in Fasting LDL-C
Time Frame: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 36, 48/early termination (ET)
LDL-C analyses used both Martin-Hopkins methodology (MHM) and ultracentrifugation (UC). The Martin-Hopkins formula is a method for calculating LDL-C that improves accuracy by using an adjustable factor for estimating very-low-density lipoprotein (VLDL) cholesterol cholesterol based on triglyceride and non-HDL cholesterol levels.
Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 36, 48/early termination (ET)
Change From Baseline in Plasma Concentrations of ARO-APOC3 Over TimeThrough Week 12
Time Frame: Day 1: pre-dose, 15 minutes, 1, 3, 6, 24 hours post-dose; Week 12: pre-dose, 15 minutes, 1, 3, 6, 24 hours post-dose
Day 1: pre-dose, 15 minutes, 1, 3, 6, 24 hours post-dose; Week 12: pre-dose, 15 minutes, 1, 3, 6, 24 hours post-dose
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Time Frame: From first dose of study drug up to Week 48
An adverse event (AE) is any untoward medical occurrence, which does not necessarily have to have a causal relationship with this treatment. A serious AE (SAE) is an AE that: results in death; is life-threatening; requires inpatient hospitalization or prolongation of an existing hospitalization; results in persistent or significant disability/incapacity; is a congenital anomaly/birth defect; or is a medically important event or reaction. TEAEs are AEs that occur following investigational product (IP) administration or a pre-existing condition exacerbated following IP administration.
From first dose of study drug up to Week 48

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Arrowhead Pharmaceuticals

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
May 31, 2021

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
March 9, 2023

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
August 31, 2023

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
January 19, 2021

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 19, 2021

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
January 22, 2021

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 24, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 3, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
March 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • AROAPOC3-2001

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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