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

July 28, 2015 updated by: Cerenis Therapeutics, SA

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)

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

23

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
      • Quebec, Canada, G1V4M6
        • Clinical Research Facility
    • Quebec
      • Chicoutimi, Quebec, Canada, G7H 7P2
        • Clinical Research Facility
  • Italy
      • Rome, Italy, 100161
        • Clinical Research Facility
  • Netherlands
      • Amsterdam, Netherlands, 1105AZ
        • Clinical Research Facility
      • Maastricht, Netherlands, 6229 HX
        • Clinical Research Facility
      • Nijmegen, Netherlands, 6500 HB
        • Clinical Research Facility
  • United Kingdom
      • Manchester, United Kingdom, M13 9WL
        • Clinical Research Facility
  • United States
    • Connecticut
      • Hartford, Connecticut, United States, 06102
        • Clinical Research Facility
    • New York
      • N. Massapequa, New York, United States, 11758
        • Clinical Research Facility

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

12 years and older (ADULT, OLDER_ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
Baseline then 6 months and/or ~2 weeks post final dose

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Baseline then 6 months and/or ~2 weeks post final dose

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Cerenis Therapeutics, SA

Investigators

  • Principal Investigator: John J. Kastelein, MD PhD, Amsterdam UMC, location VUmc

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

November 1, 2011

Primary Completion (Actual)

October 1, 2013

Study Completion (Actual)

August 1, 2014

Study Registration Dates

First Submitted

August 5, 2011

First Submitted That Met QC Criteria

August 5, 2011

First Posted (Estimate)

August 8, 2011

Study Record Updates

Last Update Posted (Estimate)

August 18, 2015

Last Update Submitted That Met QC Criteria

July 28, 2015

Last Verified

July 1, 2015

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CER-001-CLIN-003

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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