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Trial record 2 of 4 for:    17387133 [PUBMED-IDS]

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

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ClinicalTrials.gov Identifier: NCT01412034
Recruitment Status : Completed
First Posted : August 8, 2011
Last Update Posted : August 18, 2015
Sponsor:
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

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 23 participants
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

U.S. FDA Resources

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.



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Ages Eligible for Study:   12 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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 ClinicalTrials.gov identifier (NCT number): NCT01412034


Locations
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
Canada
Clinical Research Facility
Quebec, Canada, G1V4M6
Italy
Clinical Research Facility
Rome, Italy, 100161
Netherlands
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
Investigators
Principal Investigator: John J. Kastelein, MD PhD Amsterdam Medical Center

Publications of Results:
Other Publications:

Responsible Party: Cerenis Therapeutics, SA
ClinicalTrials.gov Identifier: NCT01412034     History of Changes
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
ApoA-1

Additional relevant MeSH terms:
Hypercholesterolemia
Hyperlipoproteinemia Type II
Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Hyperlipoproteinemias