Phase 3 Study to Evaluate the Efficacy and Safety of ARO-APOC3 in Adults with FCS
Research type
Research Study
Full title
A Phase 3 Study to Evaluate the Efficacy and Safety of ARO-APOC3 in Adults with Familial Chylomicronemia Syndrome
IRAS ID
1004639
Contact name
Jesse Ho
Contact email
Sponsor organisation
Arrowhead Pharmaceuticals, Inc.
Eudract number
2021-003680-10
ISRCTN Number
ISRCTN12904794
Research summary
Familial chylomicronemia syndrome (FCS) is a severe and ultrarare genetic disease, with a prevalence of approximately 1 in 1,000,000. FCS is caused by various gene mutations which leads to extremely high fasting triglyceride (TG) levels. Such severe elevations in TG lead to various serious signs and symptoms including acute pancreatitis, chronic daily abdominal pain, type 2 diabetes mellitus, hepatic steatosis, and cognitive issues. Currently, the only effective treatment is a diet extremely low in fat, the therapeutic options that can adequately treat FCS are very limited.
AROAPOC3-3001 is a phase 3, randomised, double blind, multicentre study evaluating subcutaneous injection of ARO-APOC3 compared to placebo in adults with FCS. The study drug, ARO-APOC3, has been developed by Arrowhead Pharmaceuticals Inc. as a treatment for dyslipidemias (including FCS and severe hypertriglyceridemia). It works as an RNA inhibitor and is thought to lower serum TG by preventing lipoprotein lipase inhibition. A previously completed Phase 1 study has shown ARO-APOC3 is well tolerated in healthy volunteers.
The primary objective of the study is to evaluate the efficacy and safety of ARO-APOC3 in adults with FCS. Approximately 60 eligible patients globally will be randomised 1 of 4 treatment groups (ARO-APOC3 25mg, volume matched placebo, ARO-APOC3 50mg, volume matched placebo) in a 2:1:2:1 ratio. Participants will receive 4 total doses of ARO-APOC3 or placebo once every 3 months. The total duration for participation is approximately 56 weeks from screening to the Month 12 end of study visit.
The study is sponsored by Arrowhead Pharmaceuticals, Inc.
REC name
East Midlands - Leicester Central Research Ethics Committee
REC reference
22/EM/0055
Date of REC Opinion
5 Apr 2022
REC opinion
Further Information Favourable Opinion