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

This study is currently recruiting participants.
Verified March 2013 by Cerenis Therapeutics, SA
Sponsor:
Information provided by (Responsible Party):
Cerenis Therapeutics, SA
ClinicalTrials.gov Identifier:
NCT01412034
First received: August 5, 2011
Last updated: March 21, 2013
Last verified: March 2013
History of Changes
  Purpose

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: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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:

MedlinePlus related topics: Cholesterol
U.S. FDA Resources

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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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.


Estimated Enrollment: 30
Study Start Date: November 2011
Estimated Study Completion Date: December 2013
Estimated Primary Completion Date: November 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

  Eligibility

Ages Eligible for Study:   12 Years and older
Genders Eligible for Study:   Both
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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01412034

Contacts
Contact: MODE Project Manager Medpace +1.513.579.9911 ext 2415 a.steinreide@medpace.com

Locations
United States, Connecticut
Clinical Research Facility Completed
Hartford, Connecticut, United States, 06102
United States, New York
Clinical Research Facility Completed
N. Massapequa, New York, United States, 11758
Canada, Quebec
Clinical Research Facility Recruiting
Chicoutimi, Quebec, Canada, G7H 7P2
Canada
Clinical Research Facility Completed
Quebec, Canada, G1V4M6
Italy
Clinical Research Facility Active, not recruiting
Rome, Italy, 100161
Netherlands
Clinical Research Facility Recruiting
Amsterdam, Netherlands, 1105AZ
Clinical Research Facility Recruiting
Maastricht, Netherlands, 6229 HX
Clinical Research Facility Recruiting
Nijmegen, Netherlands, 6500 HB
United Kingdom
Clinical Research Facility Recruiting
Manchester, United Kingdom, M13 9WL
Sponsors and Collaborators
Cerenis Therapeutics, SA
Investigators
Principal Investigator: John J.P. Kastelein, MD PhD
  More Information

Publications:

Responsible Party: Cerenis Therapeutics, SA
ClinicalTrials.gov Identifier: NCT01412034     History of Changes
Other Study ID Numbers: CER-001-CLIN-003
Study First Received: August 5, 2011
Last Updated: March 21, 2013
Health Authority: United States: Food and Drug Administration
Canada: Health Canada
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Italy: The Italian Medicines Agency
Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)
United Kingdom: Medicines and Healthcare Products Regulatory Agency

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

ClinicalTrials.gov processed this record on May 21, 2013