Effect of CER-001 on Plaque Volume in Homozygous Familial Hypercholesterolemia (HoFH) Subjects

Study Overview
=================
Brief Summary
-----------------
The available medications used to treat HoFH are targeted at reducing circulating levels of total and LDL-cholesterol. These measures can retard the progression of cardiovascular disease, however, they are unlikely to regress existing disease due to years of cholesterol accumulation in the vessel walls and therefore cannot adequately reduce the existing risk for an ischemic event. HDL has multiple actions that could lead to plaque stabilization and regression, such as rapid removal of large quantities of cholesterol from the vasculature, improvement in endothelial function, protection against oxidative damage and reduction in inflammation. 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 measurements in patients with HoFH.

Official 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)

Conditions
-----------------
Homozygous Familial Hypercholesterolemia

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


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Male or female subject 12 years or older Subject presents with Homozygous FH Exclusion Criteria: Weight >100 kg Subjects with significant health problems in the recent past including blood disorders, cancer, or digestive problems Female subjects of child-bearing potential Known major hematologic, renal , hepatic, metabolic, gastrointestinal or endocrine dysfunction Contraindication to MRI scanning that would preclude the use of contrast-enhanced 3TMRI

Ages Eligible for Study
-----------------
Minimum Age: 12 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 |
| --- | --- |
| Experimental: CER-001<br>Open label single arm study of CER-001 | Drug: CER-001<br>* Biweekly infusion<br>|

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

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percent change from baseline to follow-up in carotid mean vessel wall area | Percent change from baseline to follow-up in carotid mean vessel wall area | Baseline then 6 months and/or  2 weeks post final dose | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in carotid vessel wall volume | Percent change in carotid vessel wall volume , as assessed by 3TMRI, from the baseline measurement to the follow up taken  2 weeks following the final dose of study medication. | Baseline then 6 months and/or  2 weeks post final dose | 

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