Exploratory Study of Plaque Regression

Study Overview
=================
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.

Official Title
-----------------
EXPLORATORY STUDY OF PLAQUE REGRESSION:A Phase II Single Center Open-Label Exploratory Trial of the Effect of CER 001 in Subjects With Familial Hypercholesterolemia

Conditions
-----------------
Heterozygous Familial Hypercholesterolemia

Intervention / Treatment
-----------------
* Drug: CER-001


Participation Criteria
=================
Eligibility Criteria
-----------------
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

Ages Eligible for Study
-----------------
Minimum Age: 18 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: Open label single arm study of CER-001<br>Open label single arm study of CER-001 | Drug: CER-001<br>* Weekly injection<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Total Plaque Volume | 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) | Baseline and 3 weeks post final dose | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percent Change in Plaque Volume | 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) | Baseline and 3 weeks post final dose | 
| Change in carotid plaque volume | 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 | Baseline and 3 weeks post final dose | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
ApoA-1, Heterozygous Familial Hypercholesterolemia, Familial Hypercholesterolemia, HDL mimetic