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

June 9, 2023 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 or AROAPOC3-3001 is to evaluate the efficacy and safety of ARO-APOC3 in adult participants with familial chylomicronemia syndrome (FCS). Participants who have met all eligibility criteria will be randomized to receive 4 doses of ARO-APOC3 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 ARO-APOC3.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

72

Phase

  • Phase 3

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 Locations

  • Argentina
      • Córdoba, Argentina, X5003DCE
        • Instituto Médico DAMIC
      • Formosa, Argentina, 3600
        • Instituto Modelo de Gastroenterologia
  • Australia
      • Melbourne, Australia, 3081
        • Austin Health
      • Nedlands, Australia, 6009
        • Linear Clinical Research Ltd
    • New South Wales
      • Camperdown, New South Wales, Australia, 2050
        • Royal Prince Alfred Hospital
      • St. Leonards, New South Wales, Australia, 2065
        • Royal North Shore Hospital
    • Victoria
      • Melbourne, Victoria, Australia, 3004
        • Baker Heart and Diabetes Institute
  • Austria
      • Graz, Austria, 8036
        • Medizinische Universitaet Graz
  • Belgium
      • Edegem, Belgium, 2650
        • Universitair Ziekenhuis Antwerpen (Uza)
      • Ghent, Belgium, 9000
        • University Hospital Ghent
      • Leuven, Belgium, 3000
        • Universitaire Ziekenhuizen Leuven
      • Liège, Belgium, 4000
        • Centre Hospitalier Universitaire (Chu) de Liege
  • Canada
    • Ontario
      • London, Ontario, Canada, N6A 5B7
        • Robarts Research Institute
      • Toronto, Ontario, Canada, M5G 2C4
        • Toronto General Hospital
    • Quebec
      • Chicoutimi, Quebec, Canada, G7H 7K9
        • Ecogene-21
      • Montreal, Quebec, Canada, H2W 1R7
        • Institute de Recherches Cliniques de Montreal
      • Québec, Quebec, Canada, G1V 4W2
        • Clinique des Maladies Lipidiques de Quebec Inc.
  • Croatia
      • Zagreb, Croatia, 10000
        • University Hospital Center Zagreb -Rebro, Department for Metabolic Diseases
  • France
      • Paris, France, 75013
        • AP-HP Hôpital Pitié-Salpêtrière
    • Cedez 05
      • Marseille, Cedez 05, France, 13385
        • AP-HM-Hopital de La Conception
  • Germany
      • Jena, Germany, 7740
        • Universitaetsklinikum Jena
      • Leipzig, Germany, 4103
        • Universitaetsklinikum Leipzig
  • Ireland
      • Galway, Ireland, H91 YR71
        • University Hospital Galway
  • Israel
      • Jerusalem, Israel, 9112001
        • Hadassah Medical Center Ein Karem
  • Japan
      • Chiba, Japan, 260-8677
        • Chiba University Hospital
      • Ishikawa, Japan, 920-8641
        • Kanazawa University Hospital
      • Osaka, Japan, 598-0048
        • Rinku General Medical Center
      • Tochigi, Japan, 329-0498
        • Jichi Medical University Hospital
      • Tokyo, Japan
        • Nippon Medical School Hospital
      • Tokyo, Japan, 113-8655
        • Tokyo University Hospital
  • Korea, Republic of
      • Gwangju, Korea, Republic of, 61469
        • Chonnam National University Hospital
      • Seoul, Korea, Republic of, 03080
        • Seoul National University Hospital
  • Mexico
      • Mexico City, Mexico, 11650
        • Centro Especializado en Diabetes Obesidad y Prevención de entermedades Cardiovasculares (CEDOPEC)
    • Mexico DF
      • Tlalpan, Mexico DF, Mexico, 14000
        • National Institute of Medical Sciences and Nutrition - Salvador Zubiran (INCMNSZ)
    • Morelos,
      • Cuernavaca, Morelos,, Mexico, 62250
        • Instituto de Diabetes Obesidad y Nutricion S.C.
  • New Zealand
      • Auckland, New Zealand, 2025
        • Middlemore Clinical Trials
      • Auckland, New Zealand, 1010
        • NZCR OpCo Ltd
      • Christchurch, New Zealand, 8011
        • New Zealand Clinical Research
  • Oman
      • Muscat, Oman
        • Sultan Qaboos University Hospital
  • Poland
      • Łódź, Poland, 93-338
        • Instytut Centrum Zdrowia Matki Polki
  • Serbia
      • Belgrade, Serbia, 11000
        • Clinical Center of Serbia, Institute of Endocrinology, Diabetes and Metabolic Diseases
      • Niš, Serbia, 18000
        • Clinical Centre Nis
  • Singapore
      • Singapore, Singapore, 119074
        • National University Hospital
  • Spain
      • A Coruña, Spain, 15001
        • Hospital Abente y Lago
      • Granada, Spain, 18012
        • Complejo Hospitalario Universitario de Granada - Hospital Universitario Virgen de las Nieves
      • Madrid, Spain, 28007
        • Hospital General Universitario Gregorio Marañon
      • Santiago De Compostela, Spain, 15706
        • Hospital Clinico Universitario de Santiago
  • Turkey
      • İzmir, Turkey, 35100
        • Ege University Hospital Department Of Infectious Diseases
    • Kayseri
      • Melikgazi, Kayseri, Turkey, 38030
        • Erciyes University Faculty of Medicine
  • United States
    • Florida
      • Boca Raton, Florida, United States, 33434
        • Excel Medical Clinical Trials, LLC
    • Georgia
      • Suwanee, Georgia, United States, 30024
        • Herman Clinical Research, LLC
    • Indiana
      • Indianapolis, Indiana, United States, 46290
        • Ascension St. Vincent Cardiovascular Research Institute
    • Maryland
      • Elkridge, Maryland, United States, 21075
        • Centennial Medical Group
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Washington University School of Medicine, Division of Endocrinology, Metabolism and Lipid Research
    • New York
      • New York, New York, United States, 10016
        • New York University Langone Medical Center
      • New York, New York, United States, 10029
        • Icahn School of Medicine at Mt. Sinai
    • Texas
      • Austin, Texas, United States, 78731
        • Texas Diabetes and Endocrinology
    • Virginia
      • Norfolk, Virginia, United States, 23510
        • York Clinical Research, LLC

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

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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

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
Intervention / Treatment
Experimental: ARO-APOC3

4 doses of ARO-APOC3 by subcutaneous (sc) injection (randomized period)

8 doses of ARO-APOC3 by sc injection (open-label period)
Drug: ARO-APOC3
ARO-APOC3 injection
Placebo Comparator: Placebo
calculated volume to match active treatment by sc injection (randomized period)
Drug: Placebo
sterile normal saline (0.9% NaCl)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Percent Change from Baseline in Fasting Triglycerides (TG) at Month 10
Time Frame: Baseline, Month 10
Baseline, Month 10

Secondary Outcome Measures

Outcome Measure
Time Frame
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 Non-High Density Lipoprotein Cholesterol (Non-HDL-C) at Month 10
Time Frame: Baseline, Month 10
Baseline, Month 10
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 Fasting TG at Month 12
Time Frame: Baseline, Month 12
Baseline, Month 12
Percent Change from Baseline in Fasting APOC3 at Month 12
Time Frame: Baseline, Month 12
Baseline, Month 12
Percent Change from Baseline in Fasting Non-HDL-C at Month 12
Time Frame: Baseline, Month 12
Baseline, Month 12
Percent Change from Baseline in Fasting HDL-C at Month 12
Time Frame: Baseline, Month 12
Baseline, Month 12
Proportion of Patients Achieving TG of < 500 mg/dL at Month 10
Time Frame: Month 10
Month 10
Proportion of Patients Achieving TG of < 500 mg/dL at Month 12
Time Frame: Month 12
Month 12
Change from Baseline in Fasting TG Over Time
Time Frame: Baseline, up through Month 12
Baseline, up through Month 12
Percent Change from Baseline in Fasting TG Over Time
Time Frame: Baseline, up through Month 12
Baseline, up through Month 12
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
Number of Participants with Treatment-Emergent Adverse Events (AEs) and/or Serious Adverse Events (SAEs)
Time Frame: From first dose of study drug through Month 12 (Randomized Period) and through Month 36 (Open-label Period)
From first dose of study drug through Month 12 (Randomized Period) and through Month 36 (Open-label Period)
Number of Participants with Positively Adjudicated Events of Acute Pancreatitis
Time Frame: From first dose of study drug through Month 12 (Randomized Period) and through Month 36 (Open-label Period)
From first dose of study drug through Month 12 (Randomized Period) and through Month 36 (Open-label Period)

Collaborators and Investigators

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

Sponsor

Arrowhead Pharmaceuticals

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)

January 11, 2022

Primary Completion (Estimated)

April 1, 2024

Study Completion (Estimated)

April 1, 2026

Study Registration Dates

First Submitted

October 11, 2021

First Submitted That Met QC Criteria

October 11, 2021

First Posted (Actual)

October 22, 2021

Study Record Updates

Last Update Posted (Actual)

June 12, 2023

Last Update Submitted That Met QC Criteria

June 9, 2023

Last Verified

June 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AROAPOC3-3001

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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