Study of ARO-APOC3 (Plozasiran) in Adults With Familial Chylomicronemia Syndrome (FCS) (PALISADE)

May 18, 2026 updated by: Arrowhead Pharmaceuticals

A Phase 3 Study to Evaluate the Efficacy and Safety of ARO-APOC3 in Adults With Familial Chylomicronemia Syndrome

The purpose of AROAPOC3-3001 is to evaluate the efficacy and safety of ARO-APOC3 (plozasiran) in adult participants with familial chylomicronemia syndrome (FCS). Participants who have met all eligibility criteria will be randomized to receive 4 doses of plozasiran or matching placebo administered subcutaneously. Participants who complete the randomized period will continue in a 2-year open-label extension period where all participants will receive plozasiran.

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.
75

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 3

Expanded Access

Expanded Access

A way for patients with serious diseases or conditions who cannot participate in a clinical trial to gain access to a medical product that has not been approved by the U.S. Food and Drug Administration (FDA). Also called compassionate use. There are different expanded access types.
Available

Available

  • Available: Expanded access is currently available for this investigational treatment, and patients who are not participants in the clinical study may be able to gain access to the drug, biologic, or medical device being studied.
  • No longer available: Expanded access was available for this intervention previously but is not currently available and will not be available in the future.
  • Temporarily not available: Expanded access is not currently available for this intervention but is expected to be available in the future.
  • Approved for marketing: The intervention has been approved by the U.S. Food and Drug Administration for use by the public.
 outside the clinical trial. See expanded access record.

Contacts and Locations

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

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Argentina
      • Córdoba, Argentina, X5003DCE
        • Clinical Site 10
      • Formosa, Argentina, 3600
        • Clinical Site 11
  • Australia
      • Melbourne, Australia, 3081
        • Clinical Site 12
      • Nedlands, Australia, 6009
        • Clinical Site 13
    • New South Wales
      • Camperdown, New South Wales, Australia, 2050
        • Clinical Site 14
      • St Leonards, New South Wales, Australia, 2065
        • Clinical Site 15
    • Victoria
      • Melbourne, Victoria, Australia, 3004
        • Clinical Site 16
  • Austria
      • Graz, Austria, 8036
        • Clinical Site 17
  • Belgium
      • Edegem, Belgium, 2650
        • Clinical Site 18
      • Ghent, Belgium, 9000
        • Clinical Site 19
      • Leuven, Belgium, 3000
        • Clinical Site 20
      • Liège, Belgium, 4000
        • Clinical Site 21
  • Canada
    • Ontario
      • London, Ontario, Canada, N6A 5B7
        • Clinical Site 22
      • Toronto, Ontario, Canada, M5G 2C4
        • Clinical Site 23
    • Quebec
      • Chicoutimi, Quebec, Canada, G7H 7K9
        • Clinical Site 24
      • Montreal, Quebec, Canada, H2W 1R7
        • Clinical Site 25
      • Québec, Quebec, Canada, G1V 4W2
        • Clinical Site 26
  • Croatia
      • Zagreb, Croatia, 10000
        • Clinical Site 27
  • France
      • Paris, France, 75013
        • Clinical Site 28
    • Cedez 05
      • Marseille, Cedez 05, France, 13385
        • Clinical Site 29
  • Germany
      • Jena, Germany, 7740
        • Clinical Site 30
      • Leipzig, Germany, 4103
        • Clinical Site 31
  • Ireland
      • Galway, Ireland, H91 YR71
        • Clinical Site 32
  • Israel
      • Jerusalem, Israel, 9112001
        • Clinical Site 33
  • Japan
      • Chiba, Japan, 260-8677
        • Clinical Site 34
      • Ishikawa, Japan, 920-8641
        • Clinical Site 35
      • Osaka, Japan, 598-0048
        • Clinical Site 36
      • Tochigi, Japan, 329-0498
        • Clinical Site 37
      • Tokyo, Japan, 113-8655
        • Clinical Site 38
      • Tokyo, Japan
        • Clinical Site 39
  • Mexico
      • Mexico City, Mexico, 11650
        • Clinical Site 40
    • Mexico DF
      • Tlalpan, Mexico DF, Mexico, 14000
        • Clinical Site 41
    • Morelos
      • Cuernavaca, Morelos, Mexico, 62250
        • Clinical Site 42
  • New Zealand
      • Auckland, New Zealand, 1010
        • Clinical Site 44
      • Auckland, New Zealand, 2025
        • Clinical Site 43
      • Christchurch, New Zealand, 8011
        • Clinical Site 45
  • Oman
      • Muscat, Oman
        • Clinical Site 46
  • Poland
      • Lodz, Poland, 93-338
        • Clinical Site 47
  • Serbia
      • Belgrade, Serbia, 11000
        • Clinical Site 48
      • Niš, Serbia, 18000
        • Clinical Site 49
  • Singapore
      • Singapore, Singapore, 119074
        • Clinical Site 50
  • South Korea
      • Gwangju, South Korea, 61469
        • Clinical Site 51
      • Seoul, South Korea, 03080
        • Clinical Site 52
  • Spain
      • A Coruña, Spain, 15001
        • Clinical Site 53
      • Granada, Spain, 18012
        • Clinical Site 54
      • Madrid, Spain, 28007
        • Clinical Site 55
      • Santiago de Compostela, Spain, 15706
        • Clinical Site 56
  • Turkey (Türkiye)
      • Izmir, Turkey (Türkiye), 35100
        • Clinical Site 57
    • Kayseri
      • Melikgazi, Kayseri, Turkey (Türkiye), 38030
        • Clinical Site 58
  • United States
    • Florida
      • Boca Raton, Florida, United States, 33434
        • Clinical Site 1
    • Georgia
      • Suwanee, Georgia, United States, 30024
        • Clinical Site 2
    • Indiana
      • Indianapolis, Indiana, United States, 46290
        • Clinical Site 3
    • Maryland
      • Elkridge, Maryland, United States, 21075
        • Clinical Site 4
    • Missouri
      • St Louis, Missouri, United States, 63110
        • Clinical Site 5
    • New York
      • New York, New York, United States, 10016
        • Clinical Site 7
      • New York, New York, United States, 10029
        • Clinical Site 6
    • Texas
      • Austin, Texas, United States, 78731
        • Clinical Site 8
    • Virginia
      • Norfolk, Virginia, United States, 23510
        • Clinical Site 9

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:

  • Fasting triglycerides (TG) ≥ 10 mmol/L (≥ 880 mg/dL) at screening refractory to standard lipid lowering therapy
  • Diagnosis of FCS
  • Willing to follow dietary counseling as per investigator judgement based on local standard of care
  • Participants of childbearing potential (males & females) must use highly-effective contraception during the study and for at least 24 weeks following the last dose of study medication. Males must not donate sperm during the study and for at least 24 weeks following the last dose of study medication
  • Women of childbearing potential must have a negative pregnancy test at Screening 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

Exclusion Criteria:

  • Current use or use within the last 365 Days from Day 1 of any hepatocyte-targeted siRNA or antisense oligonucleotide molecule
  • Diabetes mellitus newly diagnosed within 12 weeks of Screening or where HbA1c ≥ 9.0% at Screening
  • Active pancreatitis within 12 weeks before Day 1
  • History of acute coronary syndrome event within 24 weeks of Day 1
  • History of major surgery within 12 weeks of Day 1
  • Uncontrolled hypertension
  • On treatment with human immunodeficiency virus (HIV) antiretroviral therapy
  • Seropositive for hepatitis B virus (HBV) or hepatitis C virus (HCV)
  • New York Heart Association (NYHA) Clas II, III, or IV heart failure

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: ARO-APOC3 (Plozasiran) 25 mg

Randomized Period: plozasiran 25 mg Q3M for a total of 4 doses.

Open-label Period (Parts A and B): plozasiran 25 mg Q3M for a total of 8 doses.
Drug: Plozasiran
ARO-APOC3 subcutaneous (SC) injection
Other Names:
  • ARO-APOC3
Placebo Comparator: Placebo for ARO-APOC3 (Plozasiran) 25 mg

Randomized Period: volume-matched placebo every 3 months (Q3M) for a total of 4 doses.

Open-label Period (Parts A and B): plozasiran 25 mg Q3M for a total of 8 doses.
Drug: Plozasiran
ARO-APOC3 subcutaneous (SC) injection
Other Names:
  • ARO-APOC3
Drug: Placebo
sterile normal saline (0.9% NaCl) SC injection
Experimental: ARO-APOC3 (Plozasiran) 50 mg

Randomized Period: plozasiran 25 mg Q3M for a total of 4 doses.

Open-label Period: plozasiran 50 mg (Part A), then 25 mg (Part B) Q3M for a total of 8 doses.
Drug: Plozasiran
ARO-APOC3 subcutaneous (SC) injection
Other Names:
  • ARO-APOC3
Placebo Comparator: Placebo for ARO-APOC3 (Plozasiran) 50 mg

Randomized Period: volume-matched placebo every 3 months (Q3M) for a total of 4 doses.

Open-label Period: plozasiran 50 mg (Part A), then 25 mg (Part B) Q3M for a total of 8 doses.
Drug: Plozasiran
ARO-APOC3 subcutaneous (SC) injection
Other Names:
  • ARO-APOC3
Drug: Placebo
sterile normal saline (0.9% NaCl) 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 Month 10 in Fasting Triglycerides (TG)
Time Frame: Baseline, Month 10
Baseline, Month 10

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 in Fasting TG at Month 10 and Month 12 (Averaged)
Time Frame: Baseline, Month 10, Month 12
Baseline, Month 10, Month 12
Percent Change From Baseline in Apolipoprotein C-III (APOC3) at Month 10
Time Frame: Baseline, Month 10
Baseline, Month 10
Percent Change From Baseline in Fasting APOC3 at Month 12
Time Frame: Baseline, Month 12
Baseline, Month 12
Percentage of Participants With Positively Adjudicated Events of Acute Pancreatitis (Randomized Period)
Time Frame: From first dose of study drug through Month 12 (Randomized Period)

All adverse events (AEs) and serious adverse events (SAEs) reported by the Investigator during the study that are consistent with an event of acute pancreatitis will be adjudicated by a blinded, independent committee according to the 2013 Atlanta definition meeting 2 of the following 3 criteria:

  1. Abdominal pain consistent with acute pancreatitis (acute onset of a persistent, severe, epigastric pain often radiating to the back)
  2. Serum lipase activity (or amylase activity) ≥3 times the upper limit of normal (×ULN)
  3. Characteristic findings of acute pancreatitis on contrast-enhanced computed tomography (CECT), magnetic resonance imaging (MRI), or transabdominal ultrasonography.
From first dose of study drug through Month 12 (Randomized Period)
Percentage of Participants With Positively Adjudicated Events of Acute Pancreatitis (Open-Label Period)
Time Frame: From first dose of study drug through Month 36 (Open-Label Period)

All adverse events (AEs) and serious adverse events (SAEs) reported by the Investigator during the study that are consistent with an event of acute pancreatitis will be adjudicated by a blinded, independent committee according to the 2013 Atlanta definition meeting 2 of the following 3 criteria:

  1. Abdominal pain consistent with acute pancreatitis (acute onset of a persistent, severe, epigastric pain often radiating to the back)
  2. Serum lipase activity (or amylase activity) ≥3 times the upper limit of normal (×ULN)
  3. Characteristic findings of acute pancreatitis on contrast-enhanced computed tomography (CECT), magnetic resonance imaging (MRI), or transabdominal ultrasonography.
From first dose of study drug through Month 36 (Open-Label Period)
Percent Change From Baseline in Non-High Density Lipoprotein Cholesterol (Non-HDL-C) at Month 10
Time Frame: Baseline, Month 10
Baseline, Month 10
Percent Change From Baseline in Non-HDL-C at Month 12
Time Frame: Baseline, Month 12
Baseline, Month 12
Percent Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Month 10
Time Frame: Baseline, Month 10
Baseline, Month 10
Percent Change From Baseline in HDL-C at Month 12
Time Frame: Baseline, Month 12
Baseline, Month 12
Percent Change From Baseline in Fasting Triglycerides (TG) at Month 12
Time Frame: Baseline, Month 12
Baseline, Month 12
Percentage of Participants Achieving Fasting TG of <500, 880, and 1000 mg/dL at Month 10
Time Frame: Month 10
Month 10
Percentage of Participants Achieving Fasting TG of <500, 880, and 1000 mg/dL at Month 12
Time Frame: Month 12
Month 12
Percentage of Participants Achieving ≥40% and ≥70% Reduction From Baseline in Fasting TG at Month 10
Time Frame: Baseline, Month 10
Baseline, Month 10
Change From Baseline in Fasting TG Over Time
Time Frame: Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12
Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12
Percent Change From Baseline in Fasting TG Over Time
Time Frame: Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12
Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12
Number of Participants With Treatment-Emergent Adverse Events (AEs) and/or Serious Adverse Events (SAEs; Randomized Period)
Time Frame: From first dose of study drug through Month 12 (Randomized Period)
AE: any untoward medical occurrence which does not necessarily have to have a causal relationship with treatment. Treatment-emergent AEs (TEAEs): AEs with onset after administration of the study drug, or when a preexisting medical condition increases in severity or frequency after study drug administration. SAE: AE that fulfills one or more of the following: results in death; is immediately life-threatening; requires in-patient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability or incapacity; results in a congenital abnormality or birth defect; is an important medical event that may jeopardize the patient or may require medical intervention to prevent one of the outcomes listed above. Severity was reported as: mild, moderate, severe, life threatening, death.
From first dose of study drug through Month 12 (Randomized Period)
Number of Participants With Treatment-Emergent AEs and/or SAEs (Open-Label Period)
Time Frame: From first dose of open-label study drug through Month 36 (Open-Label Period)
AE: any untoward medical occurrence which does not necessarily have to have a causal relationship with treatment. Treatment-emergent AEs (TEAEs): AEs with onset after administration of the study drug, or when a preexisting medical condition increases in severity or frequency after study drug administration. SAE: AE that fulfills one or more of the following: results in death; is immediately life-threatening; requires in-patient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability or incapacity; results in a congenital abnormality or birth defect; is an important medical event that may jeopardize the patient or may require medical intervention to prevent one of the outcomes listed above. Severity was reported as: mild, moderate, severe, life threatening, death.
From first dose of open-label study drug through Month 36 (Open-Label Period)

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

Helpful Links

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.
December 14, 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.
April 29, 2024

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.
April 21, 2026

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).
October 11, 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.
October 11, 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.
October 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.
June 5, 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.
May 18, 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.
May 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.
  • AROAPOC3-3001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

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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