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

This study has been completed.
Information provided by (Responsible Party):
Cerenis Therapeutics, SA Identifier:
First received: August 5, 2011
Last updated: July 28, 2015
Last verified: July 2015
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.

Condition Intervention Phase
Homozygous Familial Hypercholesterolemia Drug: CER-001 Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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)

Resource links provided by NLM:

Further study details as provided by Cerenis Therapeutics, SA:

Primary Outcome Measures:
  • 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 Outcome Measures:
  • 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.

Enrollment: 23
Study Start Date: November 2011
Study Completion Date: August 2014
Primary Completion Date: October 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: CER-001
Open label single arm study of CER-001
Drug: CER-001
Biweekly infusion


Ages Eligible for Study:   12 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   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
  Contacts and Locations
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Please refer to this study by its identifier: NCT01412034

United States, Connecticut
Clinical Research Facility
Hartford, Connecticut, United States, 06102
United States, New York
Clinical Research Facility
N. Massapequa, New York, United States, 11758
Canada, Quebec
Clinical Research Facility
Chicoutimi, Quebec, Canada, G7H 7P2
Clinical Research Facility
Quebec, Canada, G1V4M6
Clinical Research Facility
Rome, Italy, 100161
Clinical Research Facility
Amsterdam, Netherlands, 1105AZ
Clinical Research Facility
Maastricht, Netherlands, 6229 HX
Clinical Research Facility
Nijmegen, Netherlands, 6500 HB
United Kingdom
Clinical Research Facility
Manchester, United Kingdom, M13 9WL
Sponsors and Collaborators
Cerenis Therapeutics, SA
Principal Investigator: John J. Kastelein, MD PhD Amsterdam Medical Center
  More Information


Responsible Party: Cerenis Therapeutics, SA Identifier: NCT01412034     History of Changes
Other Study ID Numbers: CER-001-CLIN-003
Study First Received: August 5, 2011
Last Updated: July 28, 2015

Keywords provided by Cerenis Therapeutics, SA:
Homozygous Familial Hypercholesterolemia
Familial Hypercholesterolemia
HDL mimetic

Additional relevant MeSH terms:
Hyperlipoproteinemia Type II
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Hyperlipoproteinemias processed this record on September 19, 2017