Exploratory Study of Plaque Regression

Phase 2

Completed

Brief Summary: Despite the availability of several classes of very effective drugs available to treat heterozygous Familial Hypercholesterolemia (HeFH), there remains a large unmet medical need for new, effective and well tolerated therapies. There are a number of therapies given on a chronic basis to reduce long term risk, such as statins, fibrates, niacin, omega 3 fatty acids, resins, cholesterol absorption inhibitors and antiplatelet or anticoagulant drugs, but subjects with heterozygous Familial Hypercholesterolemia remain at high risk for cardiovascular events. There is still a need for acute therapies that can lead to rapid pacification of unstable plaque in order to reduce the risk of these events. This study will assess the effects of CER-001 , a recombinant human Apo-A-1 based HDL mimetic, on indices of atherosclerotic plaque progression and regression as assessed by 3Tesla MRI (3TMRI)and intravascular ultrasound (IVUS) evaluations in patients with HeFH.

Recruitment & Eligibility

Inclusion Criteria

Male or Female subjects at least 18 years old
Subject presents heterozygous FH, known CHD and receiving maximally tolerated lipid modifying therapy, at stable doses for at least 3 months
LDL-C of > 110 mg/dl
Angiographic evidence of coronary artery disease with suitable "target" coronary artery for IVUS

Exclusion Criteria

Confirmed diagnosis of homozygous FH
Significant health problems (other than cardiovascular disease) in the recent past including blood disorders, cancer, or digestive problems
Female subjects not meeting the study definition of non child-bearing potential
Use of an investigational agent within 30 days of the first CER-001 dose
Receiving current lipid apheresis

Arm && Interventions

Group	Intervention	Description
Open label single arm study of CER-001	CER-001	Open label single arm study of CER-001

Primary Outcome Measures

Name	Time	Method
Change in Total Plaque Volume	Baseline and 3 weeks post final dose	Nominal change in total plaque volume (ACTPV), as assessed by 3D IVUS, from the baseline measurement to the follow-up taken \~3 weeks following the final dose of study medication (approximately 10 weeks after the baseline assessment)

Secondary Outcome Measures

Name	Time	Method
Percent Change in Plaque Volume	Baseline and 3 weeks post final dose	Percent change in total plaque volume (PCTPV), as assessed by IVUS, from the baseline measurement to the follow up taken approximately 3 weeks following the final dose of study medication (approximately 10 weeks after the baseline assessment)
Change in carotid plaque volume	Baseline and 3 weeks post final dose	Percent change in total carotid plaque volume, as assessed by 3TMRI, from the baseline measurement to the follow up taken approximately 3 weeks following the final dose of study medication