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

Phase 2CompletedNCT01412034

Cerenis Therapeutics, SA23 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Homozygous Familial Hypercholesterolemia

Interventions

Eligibility

Sex: ALLMin age: 12 Years

Medical Language ↔ Plain English

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

Locations (9)

Clinical Research Facility

Hartford, Connecticut, United States

Clinical Research Facility

North Massapequa, New York, United States

Clinical Research Facility

Chicoutimi, Quebec, Canada

Clinical Research Facility

Québec, Quebec, Canada

Clinical Research Facility

Rome, Italy

Clinical Research Facility

Amsterdam, Netherlands

Clinical Research Facility

Maastricht, Netherlands

Clinical Research Facility

Nijmegen, Netherlands

Clinical Research Facility

Manchester, United Kingdom

Outcomes

Primary Outcomes

Percent change from baseline to follow-up in carotid mean vessel wall area

Time frame: Baseline then 6 months and/or ~2 weeks post final dose

Secondary Outcomes

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.

Time frame: Baseline then 6 months and/or ~2 weeks post final dose