We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 1 for:    NCT05089084
Previous Study | Return to List | Next Study

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05089084
Recruitment Status : Recruiting
First Posted : October 22, 2021
Last Update Posted : October 24, 2022
Sponsor:
Information provided by (Responsible Party):
Arrowhead Pharmaceuticals

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 protocol eligibility criteria will be randomized to receive 4 total doses of ARO-APOC3 or matching placebo administered subcutaneously.

Condition or disease Intervention/treatment Phase
Familial Chylomicronemia Drug: ARO-APOC3 Drug: Placebo Phase 3

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 3 Study to Evaluate the Efficacy and Safety of ARO-APOC3 in Adults With Familial Chylomicronemia Syndrome
Actual Study Start Date : January 11, 2022
Estimated Primary Completion Date : December 2023
Estimated Study Completion Date : December 2023


Arm Intervention/treatment
Experimental: ARO-APOC3
4 doses or ARO-APOC3 by subcutaneous (sc) injection
Drug: ARO-APOC3
ARO-APOC3 injection

Placebo Comparator: Placebo
calculated volume to match active treatment by sc injection
Drug: Placebo
sterile normal saline (0.9% NaCl)




Primary Outcome Measures :
  1. Percent Change from Baseline in Fasting Triglycerides (TG) at Month 10 [ Time Frame: Baseline, Month 10 ]

Secondary Outcome Measures :
  1. Percent Change from Baseline in Apolipoprotein C-III (APOC3) at Month 10 [ Time Frame: Baseline, Month 10 ]
  2. Percent Change from Baseline in Non-High Density Lipoprotein Cholesterol (Non-HDL-C) at Month 10 [ Time Frame: Baseline, Month 10 ]
  3. Percent Change from Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Month 10 [ Time Frame: Baseline, Month 10 ]
  4. Percent Change from Baseline in Fasting TG at Month 12 [ Time Frame: Baseline, Month 12 ]
  5. Percent Change from Baseline in Fasting APOC3 at Month 12 [ Time Frame: Baseline, Month 12 ]
  6. Percent Change from Baseline in Fasting Non-HDL-C at Month 12 [ Time Frame: Baseline, Month 12 ]
  7. Percent Change from Baseline in Fasting HDL-C at Month 12 [ Time Frame: Baseline, Month 12 ]
  8. Proportion of Patients Achieving TG of < 500 mg/dL at Month 10 [ Time Frame: Month 10 ]
  9. Proportion of Patients Achieving TG of < 500 mg/dL at Month 12 [ Time Frame: Month 12 ]
  10. Change from Baseline in Fasting TG Over Time [ Time Frame: Baseline, up through Month 12 ]
  11. Percent Change from Baseline in Fasting TG Over Time [ Time Frame: Baseline, up through Month 12 ]
  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 ]
  13. Number of Participants with Positively Adjudicated Events of Acute Pancreatitis [ Time Frame: From first dose of study drug through Month 12 ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
  • 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


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05089084


Contacts
Layout table for location contacts
Contact: Clinical Operations Lead 626-304-3400 AROAPOC@arrowheadpharma.com

Locations
Show Show 35 study locations
Sponsors and Collaborators
Arrowhead Pharmaceuticals
Layout table for additonal information
Responsible Party: Arrowhead Pharmaceuticals
ClinicalTrials.gov Identifier: NCT05089084    
Other Study ID Numbers: AROAPOC3-3001
First Posted: October 22, 2021    Key Record Dates
Last Update Posted: October 24, 2022
Last Verified: October 2022

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
Layout table for MeSH terms
Hyperlipoproteinemia Type I
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Hyperlipoproteinemias
Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases