ISIS 678354-CS13
Research type
Research Study
Full title
An Open-Label Extension Study of AKCEA-APOCIII-LRX Administered Subcutaneously to Patients with Familial Chylomicronemia Syndrome (FCS)
IRAS ID
1006497
Contact name
Stacy Woeppel
Contact email
Sponsor organisation
Ionis Pharmaceuticals, Inc.
Eudract number
2021-003280-95
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 in their blood. People with FCS may get fat deposits on their skin called xanthomas. They often experience frequent and repetitive severe abdominal pain (stomach area) and are at a higher risk of developing pancreatitis. Pancreatitis is inflammation of the pancreas (an organ in the stomach area that helps with digestion and regulates blood sugar) that can cause severe pain in the stomach area and often requires long stays in the hospital.
Apolipoprotein C-III (apoC-III) is a protein 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 and hopefully reduce some of the symptoms you 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 open-label extension (OLE) study of FCS Participants rolling-over from ISIS 678354-CS3 (the index study).REC name
Wales REC 5
REC reference
23/WA/0023
Date of REC Opinion
16 Jun 2023
REC opinion
Further Information Favourable Opinion