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

Sponsor

Cerenis Therapeutics, SA (Industry)

Overall Status

Completed

CT.gov ID

NCT01412034

Collaborator

(none)

Enrollment

Locations

Arm

Duration (Months)

2.6

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

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 Design

Study Type:

Interventional

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 :

Nov 1, 2011

Actual Primary Completion Date :

Oct 1, 2013

Actual Study Completion Date :

Aug 1, 2014

Arms and Interventions

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

Arm

Intervention/Treatment

Experimental: CER-001

Open label single arm study of CER-001

Drug: CER-001

Biweekly infusion

Outcome Measures

Primary Outcome Measures

Percent change from baseline to follow-up in carotid mean vessel wall area [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

Change in carotid vessel wall volume [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.

Eligibility Criteria

Criteria

Ages Eligible for Study:

12 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Locations

	Site	City	State	Country	Postal Code
1	Clinical Research Facility	Hartford	Connecticut	United States	06102
2	Clinical Research Facility	N. Massapequa	New York	United States	11758
3	Clinical Research Facility	Chicoutimi	Quebec	Canada	G7H 7P2
4	Clinical Research Facility	Quebec		Canada	G1V4M6
5	Clinical Research Facility	Rome		Italy	100161
6	Clinical Research Facility	Amsterdam		Netherlands	1105AZ
7	Clinical Research Facility	Maastricht		Netherlands	6229 HX
8	Clinical Research Facility	Nijmegen		Netherlands	6500 HB
9	Clinical Research Facility	Manchester		United Kingdom	M13 9WL