Investigating the Burden of Illness associated with FCS
Research type
Research Study
Full title
Investigation of Findings and Observations Captured in Burden of Illness Survey in FCS Patients
IRAS ID
213671
Contact name
Louis St. Laurence O'Dea
Contact email
Sponsor organisation
Akcea Therapeutics
Duration of Study in the UK
0 years, 3 months, 1 days
Research summary
The IN-FOCUS study (Investigation of Findings and Observations Captured in Burden of Illness Survey in FCS Patients) is an anonymous web-based patient survey. The purpose of the study is to help doctors and others better understand the effects of a condition called familial chylomicronemia syndrome (FCS), also known as lipoprotein lipase deficiency (LPLD) or type I hyperlipoproteinemia. FCS is a very rare cardiometabolic lipid disorder that affects roughly 1 person in 1 million and is the cause of a great deal of pain - both acute and chronic - as well as general morbidity.
As such, the results of this study will be published in a medical journal and will be used to increase awareness and understanding of FCS among physicians, healthcare payers, and the public. This information may also be helpful to many people living with FCS as well as their families and care partners.
The IN-FOCUS study is funded by Akcea Therapeutics, a biopharmaceutical company based in the United States, and is being conducted by Trinity Partners, a life-sciences strategy consulting firm also based in the United States.
REC name
East Midlands - Derby Research Ethics Committee
REC reference
16/EM/0387
Date of REC Opinion
1 Sep 2016
REC opinion
Favourable Opinion