Overview

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

Status:
Recruiting
Trial end date:
2023-12-01
Target enrollment:
0
Participant gender:
All
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.
Phase:
Phase 3
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Arrowhead Pharmaceuticals
Criteria
Inclusion Criteria:

- Fasting TG ≥ 10 mmol/L 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