NCT05089084

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

A Phase 3 Study to Evaluate the Efficacy and Safety of ARO-APOC3 in Adults With Familial Chylomicronemia Syndrome

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 75 (actual)
• Sponsor: Arrowhead Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• Familial Chylomicronemia

Interventions

Type	Name	Description
DRUG	Plozasiran	ARO-APOC3 subcutaneous (SC) injection
DRUG	Placebo	sterile normal saline (0.9% NaCl) SC injection

Timeline

• Start date: 2021-12-14
• Primary completion: 2024-04-29
• Completion: 2026-04-01
• First posted: 2021-10-22
• Last updated: 2026-02-04
• Results posted: 2026-02-04

Locations

58 sites across 21 countries: United States, Argentina, Australia, Austria, Belgium, Canada, Croatia, France, Germany, Ireland, Israel, Japan, Mexico, New Zealand, Oman, Poland, Serbia, Singapore, South Korea, Spain, Turkey (Türkiye)

Regulatory

• FDA-regulated drug study