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: January 29, 2014
Last verified: January 2014

August 5, 2011
January 29, 2014
November 2011
October 2013   (final data collection date for primary outcome measure)
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
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.
Complete list of historical versions of study NCT01412034 on ClinicalTrials.gov Archive Site
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.
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.
Not Provided
Not Provided
 
Effect of CER-001 on Plaque Volume in Homozygous Familial Hypercholesterolemia (HoFH) Subjects
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.

Not Provided
Interventional
Phase 2
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Homozygous Familial Hypercholesterolemia
Drug: CER-001
Biweekly infusion
Experimental: CER-001
Open label single arm study of CER-001
Intervention: Drug: CER-001

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
23
April 2014
October 2013   (final data collection date for primary outcome measure)

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
Both
12 Years and older
No
Contact information is only displayed when the study is recruiting subjects
Netherlands,   United States,   Canada,   Italy,   United Kingdom
 
NCT01412034
CER-001-CLIN-003
No
Cerenis Therapeutics, SA
Cerenis Therapeutics, SA
Not Provided
Principal Investigator: John J.P. Kastelein, MD PhD
Cerenis Therapeutics, SA
January 2014

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