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

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.

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Key information

Conditions

Homozygous Familial Hypercholesterolemia Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dyslipidemias Genetic Diseases, Inborn Hyperlipidemias Hyperlipoproteinemia Type II Hyperlipoproteinemias Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases

Age range

12 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Clinical Research Facility, Chicoutimi, Quebec, Canada

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

CER-001

Drug

Biweekly infusion

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

Time frame: 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.

Sponsors and collaborators

Lead sponsor

Cerenis Therapeutics, SA

Industry

Registry information

Official study title

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)

Acronym: MODE

Important dates

Study start: 2011
Primary completion: 2013
Study completion: 2014
First posted: Aug 8, 2011
Registry last updated: Aug 18, 2015

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed