Clinical trial NCT01515241: Exploratory Study of Plaque Regression
Official title: EXPLORATORY STUDY OF PLAQUE REGRESSION:A Phase II Single Center Open-Label Exploratory Trial of the Effect of CER 001 in Subjects With Familial Hypercholesterolemia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Heterozygous Familial Hypercholesterolemia
Keywords: Heterozygous Familial Hypercholesterolemia
Familial Hypercholesterolemia
HDL mimetic
ApoA-1
Lead sponsor: Cerenis Therapeutics, SA
Sponsor class: INDUSTRY
First posted: 2012-01-24
Enrollment: 10
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Change in Total Plaque Volume
timeFrame: Baseline and 3 weeks post final dose
Secondary outcomes:
measure: Percent Change in Plaque Volume
timeFrame: Baseline and 3 weeks post final dose
measure: Change in carotid plaque volume
timeFrame: Baseline and 3 weeks post final dose
Other outcomes:
Locations:
city: Montreal
country: Canada
facility: Montreal Heart Institute
state: Quebec
Study officials:
affiliation: Montreal Heart Institute
name: Jean-Claude Tardif, MD
role: PRINCIPAL_INVESTIGATOR
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