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

Completed · Phase 3 · Has a placebo group

Conditions studied: Familial Chylomicronemia

In brief

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.

Key facts

Study ID

NCT05089084

Run by

Arrowhead Pharmaceuticals

People needed

75

Starts

2021-12-14

Expected to finish

2026-04-21

Last updated by the study team

2026-06-05

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Clinical Site 2 — Suwanee, Georgia, United States
• Clinical Site 3 — Indianapolis, Indiana, United States
• Clinical Site 4 — Elkridge, Maryland, United States
• Clinical Site 5 — St Louis, Missouri, United States
• Clinical Site 7 — New York, New York, United States
• Clinical Site 6 — New York, New York, United States
• Clinical Site 8 — Austin, Texas, United States
• Clinical Site 9 — Norfolk, Virginia, United States
• Clinical Site 10 — Córdoba, Argentina
• Clinical Site 11 — Formosa, Argentina
• Clinical Site 14 — Camperdown, New South Wales, Australia
• Clinical Site 15 — St Leonards, New South Wales, Australia
• Clinical Site 16 — Melbourne, Victoria, Australia
• Clinical Site 12 — Melbourne, Australia
• Clinical Site 13 — Nedlands, Australia
• Clinical Site 17 — Graz, Austria
• Clinical Site 18 — Edegem, Belgium
• Clinical Site 19 — Ghent, Belgium
• Clinical Site 20 — Leuven, Belgium
• Clinical Site 21 — Liège, Belgium
• Clinical Site 22 — London, Ontario, Canada
• Clinical Site 23 — Toronto, Ontario, Canada
• Clinical Site 24 — Chicoutimi, Quebec, Canada
• Clinical Site 25 — Montreal, Quebec, Canada
• Clinical Site 1 — Boca Raton, Florida, United States

Full record on ClinicalTrials.gov

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