A Phase 3 Study of AKCEA-APOCIIILRx Administered to Patients With FCS
Research type
Research Study
Full title
A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of AKCEA‑APOCIII‑LRX Administered Subcutaneously to Patients with Familial Chylomicronemia Syndrome (FCS)
IRAS ID
289484
Contact name
Ekaterina Gramadnikova
Contact email
Sponsor organisation
Ionis Pharmaceuticals, Inc.
Eudract number
2020-002536-67
Duration of Study in the UK
1 years, 0 months, 29 days
Research summary
Familial Chylomicronemia Syndrome (FCS)is a disease passed on through families. People with FCS have high amounts of fats called triglycerides (TG) and chylomicrons (small drops of fat) in their blood. People with FCS may get fat deposits on their skin called xanthomas. They often experience frequent and severe pain in the stomach area, over and over again that starts and stops quickly and are at a higher risk of developing pancreatitis. Pancreatitis is a condition where the pancreas (an organ in the stomach area that helps with the break down and regulation of blood sugar) becomes inflamed (swollen) over a short period of time. Pancreatitis can cause severe pain in the stomach area and often requires long stays in the hospital.
Apolipoprotein C-III (apoC-III) is a protein (amino acids, which are compounds made of carbon, hydrogen, niyrogen, oxygen, or sulfur) found in blood that increases TG blood levels. The study medication, ISIS 678354, is designed to reduce the amount of apoC-III in the blood. Reducing the amount of apoC-III in the blood may help people lower the amount of TG and chylomicrons (small drops of fat) and hopefully relieve some of the symptoms they may experience with high TG levels. This may help lower the risk of developing pancreatitis and to improve other symptoms of FCS. This research study is being conducted to determine the safety, tolerability and efficacy of the study medication, ISIS 678354.
This is a multi-centre (many sites), randomized (people are allocated at random) , double-blind (neither the patients nor the study doctor know who is getting a placebo (and who is getting the treatment), placebo-controlled study. Placebo is a drug that has no medical effect.REC name
East Midlands - Leicester South Research Ethics Committee
REC reference
21/EM/0023
Date of REC Opinion
24 Mar 2021
REC opinion
Further Information Favourable Opinion