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

This study is ongoing, but not recruiting participants.

Sponsor:

Information provided by (Responsible Party):

Cerenis Therapeutics, SA

ClinicalTrials.gov Identifier:

NCT01412034

First received: August 5, 2011

Last updated: June 10, 2013

Last verified: June 2013

History of Changes

Tracking Information

First Received Date ^ICMJE

August 5, 2011

Last Updated Date

June 10, 2013

Start Date ^ICMJE

November 2011

Estimated Primary Completion Date

November 2013 (final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

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

Original Primary Outcome Measures ^ICMJE

Percent change in total carotid plaque volume [ Time Frame: Baseline and ~2 weeks post final dose ] [ Designated as safety issue: No ]

Percent change in total carotid plaque volume, as assessed by 3TMRI, from the baseline measurement to the follow up taken ~2 weeks following the final dose of study medication.

Change History

Complete list of historical versions of study NCT01412034 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

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.

Original Secondary Outcome Measures ^ICMJE

Change in carotid plaque volume [ Time Frame: Baseline and ~2 weeks post final dose ] [ Designated as safety issue: No ]

Absolute change in carotid plaque volume, as assessed by 3TMRI, from the baseline measurement to the follow up taken ~2 weeks following the final dose of study medication.

Current Other Outcome Measures ^ICMJE

Not Provided

Original Other Outcome Measures ^ICMJE

Not Provided

Descriptive Information

Brief Title ^ICMJE

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

Official Title ^ICMJE

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)

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.

Detailed Description

Not Provided

Study Type ^ICMJE

Interventional

Study Phase

Phase 2

Study Design ^ICMJE

Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment

Condition ^ICMJE

Homozygous Familial Hypercholesterolemia

Intervention ^ICMJE

Drug: CER-001

Biweekly infusion

Study Arm (s)

Experimental: CER-001

Open label single arm study of CER-001

Intervention: Drug: CER-001

Publications *

* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Active, not recruiting

Estimated Enrollment ^ICMJE

Estimated Completion Date

December 2013

Estimated Primary Completion Date

November 2013 (final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

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

Gender

Both

Ages

12 Years and older

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact information is only displayed when the study is recruiting subjects

Location Countries ^ICMJE

United States, Canada, Italy, Netherlands, United Kingdom

Administrative Information

NCT Number ^ICMJE

NCT01412034

Other Study ID Numbers ^ICMJE

CER-001-CLIN-003

Has Data Monitoring Committee

Responsible Party

Cerenis Therapeutics, SA

Study Sponsor ^ICMJE

Cerenis Therapeutics, SA

Collaborators ^ICMJE

Not Provided

Investigators ^ICMJE

Principal Investigator:

John J.P. Kastelein, MD PhD

Information Provided By

Cerenis Therapeutics, SA

Verification Date

June 2013

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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