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)
Trial Snapshot
- Phase
- Phase 2
- Status
- Completed
- Sponsor
- Enrollment
- 23
- Locations
- 1
- Primary Endpoint
- Percent change from baseline to follow-up in carotid mean vessel wall area
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.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 12 Years to — (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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
Arms & Interventions
CER-001
Open label single arm study of CER-001
Intervention: CER-001 (Drug)
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
Percent change from baseline to follow-up in carotid mean vessel wall area
Secondary Outcomes
- Change in carotid vessel wall volume(Baseline then 6 months and/or ~2 weeks post final dose)