Homozygous Familial Hypercholesterolemia
Conditions
Keywords
Homozygous Familial Hypercholesterolemia, Familial Hypercholesterolemia, HDL mimetic, ApoA-1
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.
Interventions
DRUGCER-001
Biweekly infusion
Sponsors
Cerenis Therapeutics, SA
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percent change from baseline to follow-up in carotid mean vessel wall area | 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
| Measure | Time frame | Description |
|---|---|---|
| Change in carotid vessel wall volume | Baseline then 6 months and/or ~2 weeks post final dose | 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. |
Countries
Canada, Italy, Netherlands, United Kingdom, United States
Outcome results
None listed