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

NCT ID: NCT05089084

Last Updated: 2025-04-30

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

75 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-01-11

Study Completion Date

2026-04-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Familial Chylomicronemia

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

ARO-APOC3 (plozasiran)

4 doses of plozasiran by subcutaneous (sc) injection (randomized period)

8 doses of plozasiran by sc injection (open-label period)

Group Type EXPERIMENTAL

Plozasiran

Intervention Type DRUG

ARO-APOC3 injection

Placebo

calculated volume to match active treatment by sc injection (randomized period)

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

sterile normal saline (0.9% NaCl)

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Plozasiran

ARO-APOC3 injection

Intervention Type DRUG

Placebo

sterile normal saline (0.9% NaCl)

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

ARO-APOC3

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Fasting 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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Arrowhead Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Excel Medical Clinical Trials, LLC

Boca Raton, Florida, United States

Site Status

Herman Clinical Research, LLC

Suwanee, Georgia, United States

Site Status

Ascension St. Vincent Cardiovascular Research Institute

Indianapolis, Indiana, United States

Site Status

Centennial Medical Group

Elkridge, Maryland, United States

Site Status

Washington University School of Medicine, Division of Endocrinology, Metabolism and Lipid Research

St Louis, Missouri, United States

Site Status

New York University Langone Medical Center

New York, New York, United States

Site Status

Icahn School of Medicine at Mt. Sinai

New York, New York, United States

Site Status

Texas Diabetes and Endocrinology

Austin, Texas, United States

Site Status

York Clinical Research, LLC

Norfolk, Virginia, United States

Site Status

Instituto Medico DAMIC

Córdoba, , Argentina

Site Status

Instituto Modelo de Gastroenterologia

Formosa, , Argentina

Site Status

Royal Prince Alfred Hospital

Camperdown, New South Wales, Australia

Site Status

Royal North Shore Hospital

St Leonards, New South Wales, Australia

Site Status

Baker Heart and Diabetes Institute

Melbourne, Victoria, Australia

Site Status

Austin Health

Melbourne, , Australia

Site Status

Linear Clinical Research Ltd

Nedlands, , Australia

Site Status

Medizinische Universitaet Graz

Graz, , Austria

Site Status

Universitair Ziekenhuis Antwerpen (UZA)

Edegem, , Belgium

Site Status

University Hospital Ghent

Ghent, , Belgium

Site Status

Universitaire Ziekenhuizen Leuven

Leuven, , Belgium

Site Status

Centre Hospitalier Universitaire (CHU) de Liege

Liège, , Belgium

Site Status

Robarts Research Institute

London, Ontario, Canada

Site Status

Toronto General Hospital

Toronto, Ontario, Canada

Site Status

Ecogene-21

Chicoutimi, Quebec, Canada

Site Status

Institute de Recherches Cliniques de Montreal

Montreal, Quebec, Canada

Site Status

Clinique des Maladies Lipidiques de Quebec Inc.

Québec, Quebec, Canada

Site Status

University Hospital Center Zagreb -Rebro, Department for Metabolic Diseases

Zagreb, , Croatia

Site Status

AP-HM-Hopital de La Conception

Marseille, Cedez 05, France

Site Status

AP-HP Hopital Pitie-Salpetriere

Paris, , France

Site Status

Universitaetsklinikum Jena

Jena, , Germany

Site Status

Universitaetsklinikum Leipzig

Leipzig, , Germany

Site Status

University Hospital Galway

Galway, , Ireland

Site Status

Hadassah Medical Center Ein Karem

Jerusalem, , Israel

Site Status

Chiba University Hospital

Chiba, , Japan

Site Status

Kanazawa University Hospital

Ishikawa, , Japan

Site Status

Rinku General Medical Center

Osaka, , Japan

Site Status

Jichi Medical University Hospital

Tochigi, , Japan

Site Status

Tokyo University Hospital

Tokyo, , Japan

Site Status

Nippon Medical School Hospital

Tokyo, , Japan

Site Status

National Institute of Medical Sciences and Nutrition - Salvador Zubiran (INCMNSZ)

Tlalpan, Mexico DF, Mexico

Site Status

Instituto de Diabetes Obesidad y Nutrición S.C.

Cuernavaca, Morelos, Mexico

Site Status

Centro Especializado en Diabetes Obesidad y Prevención de entermedades Cardiovasculares (CEDOPEC)

Mexico City, , Mexico

Site Status

NZCR OPCO Ltd

Auckland, , New Zealand

Site Status

Middlemore Clinical Trials

Auckland, , New Zealand

Site Status

New Zealand Clinical Research

Christchurch, , New Zealand

Site Status

Sultan Qaboos University Hospital

Muscat, , Oman

Site Status

Instytut Centrum Zdrowia Matki Polki

Lodz, , Poland

Site Status

Clinical Center of Serbia, Institute of Endocrinology, Diabetes and Metabolic Diseases

Belgrade, , Serbia

Site Status

Clinical Centre Nis

Niš, , Serbia

Site Status

National University Hospital

Singapore, , Singapore

Site Status

Chonnam National University Hospital

Gwangju, , South Korea

Site Status

Seoul National University Hospital

Seoul, , South Korea

Site Status

Hospital Abente y Lago

A Coruña, , Spain

Site Status

Complejo Hospitalario Universitario de Granada - Hospital Universitario Virgen de las Nieves

Granada, , Spain

Site Status

Hospital General Universitario Gregorio Maranon

Madrid, , Spain

Site Status

Hospital Clinico Universitario de Santiago

Santiago de Compostela, , Spain

Site Status

Erciyes University Faculty of Medicine

Melikgazi, Kayseri, Turkey (Türkiye)

Site Status

Ege University Hospital Department Of Infectious Diseases

Izmir, , Turkey (Türkiye)

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States Argentina Australia Austria Belgium Canada Croatia France Germany Ireland Israel Japan Mexico New Zealand Oman Poland Serbia Singapore South Korea Spain Turkey (Türkiye)

References

Explore related publications, articles, or registry entries linked to this study.

Watts GF, Rosenson RS, Hegele RA, Goldberg IJ, Gallo A, Mertens A, Baass A, Zhou R, Muhsin M, Hellawell J, Leeper NJ, Gaudet D; PALISADE Study Group. Plozasiran for Managing Persistent Chylomicronemia and Pancreatitis Risk. N Engl J Med. 2025 Jan 9;392(2):127-137. doi: 10.1056/NEJMoa2409368. Epub 2024 Sep 2.

Reference Type DERIVED
PMID: 39225259 (View on PubMed)

Related Links

Access external resources that provide additional context or updates about the study.

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

AROAPOC3-3001

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

CS-121 APOC3 Base Editing in FCS
NCT07176923 NOT_YET_RECRUITING EARLY_PHASE1
Safety & Efficacy of AlloRx SC® in PTHS Patients
NCT05165017 UNKNOWN PHASE1/PHASE2
Senicapoc and Dehydrated Stomatocytosis
NCT04372498 COMPLETED PHASE1/PHASE2