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

Primary Purpose

Familial Chylomicronemia

Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
ARO-APOC3
Placebo
Sponsored by
Arrowhead Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Familial Chylomicronemia

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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
  • Known history of human immunodeficiency virus (HIV) infection
  • 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

Sites / Locations

  • Excel Medical Clinical Trials, LLC
  • Herman Clinical Research, LLC
  • Ascension St. Vincent Cardiovascular Research Institute
  • Centennial Medical Group
  • Washington University School of Medicine, Division of Endocrinology, Metabolism and Lipid Research
  • New York University Langone Medical Center
  • Icahn School of Medicine at Mt. Sinai
  • Texas Diabetes and Endocrinology
  • York Clinical Research, LLC
  • Instituto Medico DAMIC
  • Instituto Modelo de Gastroenterologia
  • Royal Prince Alfred Hospital
  • Royal North Shore Hospital
  • Baker Heart and Diabetes Institute
  • Austin Health
  • Linear Clinical Research Ltd
  • Medizinische Universitaet Graz
  • Universitair Ziekenhuis Antwerpen (UZA)
  • University Hospital Ghent
  • Universitaire Ziekenhuizen Leuven
  • Centre Hospitalier Universitaire (CHU) de Liege
  • Robarts Research Institute
  • Toronto General Hospital
  • Ecogene-21
  • Institute de Recherches Cliniques de Montreal
  • Clinique des Maladies Lipidiques de Quebec Inc.
  • University Hospital Center Zagreb -Rebro, Department for Metabolic Diseases
  • AP-HM-Hopital de La Conception
  • AP-HP Hopital Pitie-Salpetriere
  • Universitaetsklinikum Jena
  • Universitaetsklinikum Leipzig
  • University Hospital Galway
  • Hadassah Medical Center Ein Karem
  • Chiba University Hospital
  • Kanazawa University Hospital
  • Rinku General Medical Center
  • Jichi Medical University Hospital
  • Tokyo University Hospital
  • Nippon Medical School Hospital
  • Chonnam National University Hospital
  • Seoul National University Hospital
  • National Institute of Medical Sciences and Nutrition - Salvador Zubiran (INCMNSZ)
  • Instituto de Diabetes Obesidad y Nutrición S.C.
  • Centro Especializado en Diabetes Obesidad y Prevención de entermedades Cardiovasculares (CEDOPEC)
  • NZCR OPCO Ltd
  • Middlemore Clinical Trials
  • New Zealand Clinical Research
  • Sultan Qaboos University Hospital
  • Instytut Centrum Zdrowia Matki Polki
  • Clinical Center of Serbia, Institute of Endocrinology, Diabetes and Metabolic Diseases
  • Clinical Centre Nis
  • National University Hospital
  • Hospital Abente y Lago
  • Complejo Hospitalario Universitario de Granada - Hospital Universitario Virgen de las Nieves
  • Hospital General Universitario Gregorio Maranon
  • Hospital Clinico Universitario de Santiago
  • Erciyes University Faculty of Medicine
  • Ege University Hospital Department Of Infectious Diseases

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

ARO-APOC3

Placebo

Arm Description

4 doses of ARO-APOC3 by subcutaneous (sc) injection (randomized period) 8 doses of ARO-APOC3 by sc injection (open-label period)

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

Outcomes

Primary Outcome Measures

Percent Change from Baseline in Fasting Triglycerides (TG) at Month 10

Secondary Outcome Measures

Percent Change from Baseline in Fasting TG at Month 10 and Month 12 (Averaged)
Percent Change from Baseline in Apolipoprotein C-III (APOC3) at Month 10
Percent Change from Baseline in Fasting APOC3 at Month 12
Percent Change from Baseline in Non-High Density Lipoprotein Cholesterol (Non-HDL-C) at Month 10
Percent Change from Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Month 10
Percent Change from Baseline in Fasting TG at Month 12
Percent Change from Baseline in Fasting Non-HDL-C at Month 12
Percent Change from Baseline in Fasting HDL-C at Month 12
Proportion of Patients Achieving TG of < 500 mg/dL at Month 10
Proportion of Patients Achieving TG of < 500 mg/dL at Month 12
Change from Baseline in Fasting TG Over Time
Percent Change from Baseline in Fasting TG Over Time
Number of Participants with Treatment-Emergent Adverse Events (AEs) and/or Serious Adverse Events (SAEs)
Number of Participants with Positively Adjudicated Events of Acute Pancreatitis

Full Information

First Posted
October 11, 2021
Last Updated
June 9, 2023
Sponsor
Arrowhead Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT05089084
Brief Title
Study of ARO-APOC3 in Adults With Familial Chylomicronemia Syndrome (FCS)
Acronym
PALISADE
Official Title
A Phase 3 Study to Evaluate the Efficacy and Safety of ARO-APOC3 in Adults With Familial Chylomicronemia Syndrome
Study Type
Interventional

2. Study Status

Record Verification Date
June 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
January 11, 2022 (Actual)
Primary Completion Date
April 2024 (Anticipated)
Study Completion Date
April 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Arrowhead Pharmaceuticals

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Familial Chylomicronemia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
72 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
ARO-APOC3
Arm Type
Experimental
Arm Description
4 doses of ARO-APOC3 by subcutaneous (sc) injection (randomized period) 8 doses of ARO-APOC3 by sc injection (open-label period)
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
calculated volume to match active treatment by sc injection (randomized period)
Intervention Type
Drug
Intervention Name(s)
ARO-APOC3
Intervention Description
ARO-APOC3 injection
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
sterile normal saline (0.9% NaCl)
Primary Outcome Measure Information:
Title
Percent Change from Baseline in Fasting Triglycerides (TG) at Month 10
Time Frame
Baseline, Month 10
Secondary Outcome Measure Information:
Title
Percent Change from Baseline in Fasting TG at Month 10 and Month 12 (Averaged)
Time Frame
Baseline, Month 10, Month 12
Title
Percent Change from Baseline in Apolipoprotein C-III (APOC3) at Month 10
Time Frame
Baseline, Month 10
Title
Percent Change from Baseline in Fasting APOC3 at Month 12
Time Frame
Baseline, Month 12
Title
Percent Change from Baseline in Non-High Density Lipoprotein Cholesterol (Non-HDL-C) at Month 10
Time Frame
Baseline, Month 10
Title
Percent Change from Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Month 10
Time Frame
Baseline, Month 10
Title
Percent Change from Baseline in Fasting TG at Month 12
Time Frame
Baseline, Month 12
Title
Percent Change from Baseline in Fasting Non-HDL-C at Month 12
Time Frame
Baseline, Month 12
Title
Percent Change from Baseline in Fasting HDL-C at Month 12
Time Frame
Baseline, Month 12
Title
Proportion of Patients Achieving TG of < 500 mg/dL at Month 10
Time Frame
Month 10
Title
Proportion of Patients Achieving TG of < 500 mg/dL at Month 12
Time Frame
Month 12
Title
Change from Baseline in Fasting TG Over Time
Time Frame
Baseline, up through Month 12
Title
Percent Change from Baseline in Fasting TG Over Time
Time Frame
Baseline, up through Month 12
Title
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)
Title
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)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Facility Information:
Facility Name
Excel Medical Clinical Trials, LLC
City
Boca Raton
State/Province
Florida
ZIP/Postal Code
33434
Country
United States
Facility Name
Herman Clinical Research, LLC
City
Suwanee
State/Province
Georgia
ZIP/Postal Code
30024
Country
United States
Facility Name
Ascension St. Vincent Cardiovascular Research Institute
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46290
Country
United States
Facility Name
Centennial Medical Group
City
Elkridge
State/Province
Maryland
ZIP/Postal Code
21075
Country
United States
Facility Name
Washington University School of Medicine, Division of Endocrinology, Metabolism and Lipid Research
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
New York University Langone Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10016
Country
United States
Facility Name
Icahn School of Medicine at Mt. Sinai
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
Texas Diabetes and Endocrinology
City
Austin
State/Province
Texas
ZIP/Postal Code
78731
Country
United States
Facility Name
York Clinical Research, LLC
City
Norfolk
State/Province
Virginia
ZIP/Postal Code
23510
Country
United States
Facility Name
Instituto Medico DAMIC
City
Córdoba
ZIP/Postal Code
X5003DCE
Country
Argentina
Facility Name
Instituto Modelo de Gastroenterologia
City
Formosa
ZIP/Postal Code
3600
Country
Argentina
Facility Name
Royal Prince Alfred Hospital
City
Camperdown
State/Province
New South Wales
ZIP/Postal Code
2050
Country
Australia
Facility Name
Royal North Shore Hospital
City
St. Leonards
State/Province
New South Wales
ZIP/Postal Code
2065
Country
Australia
Facility Name
Baker Heart and Diabetes Institute
City
Melbourne
State/Province
Victoria
ZIP/Postal Code
3004
Country
Australia
Facility Name
Austin Health
City
Melbourne
ZIP/Postal Code
3081
Country
Australia
Facility Name
Linear Clinical Research Ltd
City
Nedlands
ZIP/Postal Code
6009
Country
Australia
Facility Name
Medizinische Universitaet Graz
City
Graz
ZIP/Postal Code
8036
Country
Austria
Facility Name
Universitair Ziekenhuis Antwerpen (UZA)
City
Edegem
ZIP/Postal Code
2650
Country
Belgium
Facility Name
University Hospital Ghent
City
Ghent
ZIP/Postal Code
9000
Country
Belgium
Facility Name
Universitaire Ziekenhuizen Leuven
City
Leuven
ZIP/Postal Code
3000
Country
Belgium
Facility Name
Centre Hospitalier Universitaire (CHU) de Liege
City
Liège
ZIP/Postal Code
4000
Country
Belgium
Facility Name
Robarts Research Institute
City
London
State/Province
Ontario
ZIP/Postal Code
N6A 5B7
Country
Canada
Facility Name
Toronto General Hospital
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 2C4
Country
Canada
Facility Name
Ecogene-21
City
Chicoutimi
State/Province
Quebec
ZIP/Postal Code
G7H 7K9
Country
Canada
Facility Name
Institute de Recherches Cliniques de Montreal
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H2W 1R7
Country
Canada
Facility Name
Clinique des Maladies Lipidiques de Quebec Inc.
City
Québec
State/Province
Quebec
ZIP/Postal Code
G1V 4W2
Country
Canada
Facility Name
University Hospital Center Zagreb -Rebro, Department for Metabolic Diseases
City
Zagreb
ZIP/Postal Code
10000
Country
Croatia
Facility Name
AP-HM-Hopital de La Conception
City
Marseille
State/Province
Cedez 05
ZIP/Postal Code
13385
Country
France
Facility Name
AP-HP Hopital Pitie-Salpetriere
City
Paris
ZIP/Postal Code
75013
Country
France
Facility Name
Universitaetsklinikum Jena
City
Jena
ZIP/Postal Code
7740
Country
Germany
Facility Name
Universitaetsklinikum Leipzig
City
Leipzig
ZIP/Postal Code
4103
Country
Germany
Facility Name
University Hospital Galway
City
Galway
ZIP/Postal Code
H91 YR71
Country
Ireland
Facility Name
Hadassah Medical Center Ein Karem
City
Jerusalem
ZIP/Postal Code
9112001
Country
Israel
Facility Name
Chiba University Hospital
City
Chiba
ZIP/Postal Code
260-8677
Country
Japan
Facility Name
Kanazawa University Hospital
City
Ishikawa
ZIP/Postal Code
920-8641
Country
Japan
Facility Name
Rinku General Medical Center
City
Osaka
ZIP/Postal Code
598-0048
Country
Japan
Facility Name
Jichi Medical University Hospital
City
Tochigi
ZIP/Postal Code
329-0498
Country
Japan
Facility Name
Tokyo University Hospital
City
Tokyo
ZIP/Postal Code
113-8655
Country
Japan
Facility Name
Nippon Medical School Hospital
City
Tokyo
Country
Japan
Facility Name
Chonnam National University Hospital
City
Gwangju
ZIP/Postal Code
61469
Country
Korea, Republic of
Facility Name
Seoul National University Hospital
City
Seoul
ZIP/Postal Code
03080
Country
Korea, Republic of
Facility Name
National Institute of Medical Sciences and Nutrition - Salvador Zubiran (INCMNSZ)
City
Tlalpan
State/Province
Mexico DF
ZIP/Postal Code
14000
Country
Mexico
Facility Name
Instituto de Diabetes Obesidad y Nutrición S.C.
City
Cuernavaca
State/Province
Morelos,
ZIP/Postal Code
62250
Country
Mexico
Facility Name
Centro Especializado en Diabetes Obesidad y Prevención de entermedades Cardiovasculares (CEDOPEC)
City
Mexico City
ZIP/Postal Code
11650
Country
Mexico
Facility Name
NZCR OPCO Ltd
City
Auckland
ZIP/Postal Code
1010
Country
New Zealand
Facility Name
Middlemore Clinical Trials
City
Auckland
ZIP/Postal Code
2025
Country
New Zealand
Facility Name
New Zealand Clinical Research
City
Christchurch
ZIP/Postal Code
8011
Country
New Zealand
Facility Name
Sultan Qaboos University Hospital
City
Muscat
Country
Oman
Facility Name
Instytut Centrum Zdrowia Matki Polki
City
Łódź
ZIP/Postal Code
93-338
Country
Poland
Facility Name
Clinical Center of Serbia, Institute of Endocrinology, Diabetes and Metabolic Diseases
City
Belgrade
ZIP/Postal Code
11000
Country
Serbia
Facility Name
Clinical Centre Nis
City
Niš
ZIP/Postal Code
18000
Country
Serbia
Facility Name
National University Hospital
City
Singapore
ZIP/Postal Code
119074
Country
Singapore
Facility Name
Hospital Abente y Lago
City
A Coruña
ZIP/Postal Code
15001
Country
Spain
Facility Name
Complejo Hospitalario Universitario de Granada - Hospital Universitario Virgen de las Nieves
City
Granada
ZIP/Postal Code
18012
Country
Spain
Facility Name
Hospital General Universitario Gregorio Maranon
City
Madrid
ZIP/Postal Code
28007
Country
Spain
Facility Name
Hospital Clinico Universitario de Santiago
City
Santiago De Compostela
ZIP/Postal Code
15706
Country
Spain
Facility Name
Erciyes University Faculty of Medicine
City
Melikgazi
State/Province
Kayseri
ZIP/Postal Code
38030
Country
Turkey
Facility Name
Ege University Hospital Department Of Infectious Diseases
City
İzmir
ZIP/Postal Code
35100
Country
Turkey

12. IPD Sharing Statement

Learn more about this trial

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

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