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

Cerenis Therapeutics

Status and phase

Completed

Phase 2

Conditions

Homozygous Familial Hypercholesterolemia

Treatments

Drug: CER-001

Study type

Interventional

Funder types

Industry

Identifiers

NCT01412034

CER-001-CLIN-003

Details and patient eligibility

About

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.

Enrollment

23 patients

Sex

All

Ages

12+ years old

Volunteers

No Healthy Volunteers

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