Heterozygous Familial Hypercholesterolemia
Conditions
Keywords
Heterozygous Familial Hypercholesterolemia, Familial Hypercholesterolemia, HDL mimetic, ApoA-1
Brief summary
Despite the availability of several classes of very effective drugs available to treat heterozygous Familial Hypercholesterolemia (HeFH), there remains a large unmet medical need for new, effective and well tolerated therapies. There are a number of therapies given on a chronic basis to reduce long term risk, such as statins, fibrates, niacin, omega 3 fatty acids, resins, cholesterol absorption inhibitors and antiplatelet or anticoagulant drugs, but subjects with heterozygous Familial Hypercholesterolemia remain at high risk for cardiovascular events. There is still a need for acute therapies that can lead to rapid pacification of unstable plaque in order to reduce the risk of these events. 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 (3TMRI)and intravascular ultrasound (IVUS) evaluations in patients with HeFH.
Interventions
DRUGCER-001
Weekly injection
Sponsors
Cerenis Therapeutics, SA
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Male or Female subjects at least 18 years old * Subject presents heterozygous FH, known CHD and receiving maximally tolerated lipid modifying therapy, at stable doses for at least 3 months * LDL-C of \> 110 mg/dl * Angiographic evidence of coronary artery disease with suitable target coronary artery for IVUS
Exclusion criteria
* Confirmed diagnosis of homozygous FH * Significant health problems (other than cardiovascular disease) in the recent past including blood disorders, cancer, or digestive problems * Female subjects not meeting the study definition of non child-bearing potential * Use of an investigational agent within 30 days of the first CER-001 dose * Receiving current lipid apheresis
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Total Plaque Volume | Baseline and 3 weeks post final dose | Nominal change in total plaque volume (ACTPV), as assessed by 3D IVUS, from the baseline measurement to the follow-up taken \ 3 weeks following the final dose of study medication (approximately 10 weeks after the baseline assessment) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percent Change in Plaque Volume | Baseline and 3 weeks post final dose | Percent change in total plaque volume (PCTPV), as assessed by IVUS, from the baseline measurement to the follow up taken approximately 3 weeks following the final dose of study medication (approximately 10 weeks after the baseline assessment) |
| Change in carotid plaque volume | Baseline and 3 weeks post final dose | Percent change in total carotid plaque volume, as assessed by 3TMRI, from the baseline measurement to the follow up taken approximately 3 weeks following the final dose of study medication |
Countries
Canada
Outcome results
None listed