MODE
Research type
Research Study
Full title
Modifying Orphan Disease Evaluation (MODE) Study: A Multicenter, Open-Label Study of the Effects of CER-001 on Plaque Volume in Subjects with Homozygous Familial Hypercholesterolemia (HoFH)
IRAS ID
87887
Contact name
Handrean Soran
Sponsor organisation
Cerenis Therapeutics
Eudract number
2011-003998-28
Clinicaltrials.gov Identifier
Research summary
CER-001 is being studied as an intravenously (into the vein) delivered disease modifying therapy for patients with homozgous familial hypercholesterolemia (HoFH). Familial hypercholesterolemia (FH) is an autosomal dominant disorder of low-density lipoprotein (LDL) receptors. Absence or gross malfunctioning of the LDL receptors impair the normal processing of circulating LDL in the body , leading to severe rise of total cholesterol and LDL-cholesterol. The homozygous form of FH affects approximately 1 in 1 million persons worldwide and causes an absent to minimal functioning of these LDL receptors. This causes severe and widespread atherosclorosis affecting all major arteries. The risk for early heart disease events such as heart attack and sudden death starts at an early stage, with events occurring in children as young as 1-2 years of age. Currently without major intervention, patients with HoFH do not survive past early adulthood. In patients who retain some degree of LDL receptor function, diet control and aggressive treatment with statins and other cholesterol lowering medication can be effective. In patients who have complete absence of LDL receptor functioning, LDL apheresis (a form of dialysis to eliminate the LDL in the blood), liver transplantation or further surgery are necessary. The available medication used to treat HoFH are targeted at reducing circulating levels of total and LDL-cholesterol. These measures can slow the progression of cardiovascular disease, however they are unlikely to help undo the existing disease due to years of cholesterol accumulation in the vessels walls and an unmet medical therapy to adequately reduce this and the chance of resulting ischemic events exists. CER-001 has the potential to help in the treatment of reversing the cholesterol accumulation by mimicking the action of High density lipoprotein in the reverse lipid transport (RLT) pathway which helps expel the cholesterol back to the liver to be eliminated.
REC name
London - Brighton & Sussex Research Ethics Committee
REC reference
11/LO/1789
Date of REC Opinion
24 Jan 2012
REC opinion
Further Information Favourable Opinion