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

Phase 2

Completed

• Conditions: Homozygous Familial Hypercholesterolemia
• Interventions: Drug: CER-001

• Registration Number: NCT01412034
• Lead Sponsor: 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2011-08-05
• Study First Submitted QC: 2011-08-05
• Study First Posted: 2011-08-08
• Study Start: 2011-11
• Primary Completion: 2013-10
• Study Completion: 2014-08
• Status Verified: 2015-07
• Disposition First Submitted: 2015-07-28
• Disposition First Submitted QC: 2015-07-28
• Last Update Submitted: 2015-07-28
• Disposition First Posted: 2015-08-18
• Last Update Posted: 2015-08-18

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 23

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
CER-001	CER-001	Open label single arm study of CER-001

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
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.

Trial Locations

Locations (1)

Clinical Research Facility; 🇬🇧 Manchester, United Kingdom