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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01412034
Recruitment Status : Completed
First Posted : August 8, 2011
Last Update Posted : August 18, 2015
Information provided by (Responsible Party):
Cerenis Therapeutics, SA

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.

Condition or disease Intervention/treatment Phase
Homozygous Familial Hypercholesterolemia Drug: CER-001 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 23 participants
Allocation: N/A
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)
Study Start Date : November 2011
Actual Primary Completion Date : October 2013
Actual Study Completion Date : August 2014

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: CER-001
Open label single arm study of CER-001
Drug: CER-001
Biweekly infusion

Primary Outcome Measures :
  1. 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 :
  1. 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.

Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01412034

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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
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Principal Investigator: John J. Kastelein, MD PhD VU University Medical Center
Publications of Results:
Other Publications:

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Responsible Party: Cerenis Therapeutics, SA Identifier: NCT01412034    
Other Study ID Numbers: CER-001-CLIN-003
First Posted: August 8, 2011    Key Record Dates
Last Update Posted: August 18, 2015
Last Verified: July 2015
Keywords provided by Cerenis Therapeutics, SA:
Homozygous Familial Hypercholesterolemia
Familial Hypercholesterolemia
HDL mimetic
Additional relevant MeSH terms:
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Hyperlipoproteinemia Type II
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn