ISIS 304801-CS7 - The APPROACH Open-Label Extension
Research type
Research Study
Full title
An Open-Label Extension Study of Volanesorsen Administered Subcutaneously to Patients with Familial Chylomicronemia Syndrome (FCS)
IRAS ID
195010
Contact name
Alan Jones
Contact email
Sponsor organisation
Ionis Pharmaceuticals, Inc.
Eudract number
2015-003755-21
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 8 months, 1 days
Research summary
Familial Chylomicronemia Syndrome (FCS) is a rare genetic disease characterised by severely high levels of triglycerides (fatty substances) and chylomicrons (proteins that transport triglycerides) in the blood. The disease is caused by a mutation (error) in the lipoprotein lipase (LPL) gene and therefore cannot produce LPL. Without LPL, the body is unable to break down fats correctly.
Patients often experience severe stomach pain, enlargement of the liver, eye abnormalities, and yellow deposits of fatty material on the skin (xanthomas). One of the major risks of high levels of triglycerides and chylomicrons is acute pancreatitis (inflammation of the pancreas) causing life threatening complications.
There is currently no cure for FCS. Current treatments for FCS focus on a low-fat, alcohol-free diet to reduce symptoms of the disease.
Ionis Pharmaceuticals have developed a new drug, volanesorsen, that has been designed to reduce the amount of apoC-III protein in the blood which is a key regulator of triglyceride levels.
This study is being done to evaluate the safety and effects of extended use of volanesorsen in patients with FCS who have already participated in study ISIS 304801-CS6 or ISIS 304801-CS16.
Approximately 70 participants (men and women) aged 18 years and over who have completed study ISIS 304801-CS6 or ISIS 304801-CS16 will take part in this open-label extension study. Participants will receive 300 mg volanesorsen by injection once a week for 52 weeks.REC name
West Midlands - Edgbaston Research Ethics Committee
REC reference
16/WM/0170
Date of REC Opinion
6 Jun 2016
REC opinion
Further Information Favourable Opinion