Back to resultsEffect of CER-001 on Plaque Volume in Homozygous Familial Hypercholesterolemia (HoFH) Subjects (MODE)
Primary Purpose
Homozygous Familial Hypercholesterolemia
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
CER-001
Sponsored by
About this trial
This is an interventional treatment trial for Homozygous Familial Hypercholesterolemia focused on measuring Homozygous Familial Hypercholesterolemia, Familial Hypercholesterolemia, HDL mimetic, ApoA-1
Eligibility Criteria
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
Sites / Locations
- Clinical Research Facility
- Clinical Research Facility
- Clinical Research Facility
- Clinical Research Facility
- Clinical Research Facility
- Clinical Research Facility
- Clinical Research Facility
- Clinical Research Facility
- Clinical Research Facility
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
CER-001
Arm Description
Open label single arm study of CER-001
Outcomes
Primary Outcome Measures
Percent change from baseline to follow-up in carotid mean vessel wall area
Percent change from baseline to follow-up in carotid mean vessel wall area
Secondary Outcome Measures
Change in carotid vessel wall volume
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.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01412034
Brief Title
Effect of CER-001 on Plaque Volume in Homozygous Familial Hypercholesterolemia (HoFH) Subjects
Acronym
MODE
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 Type
Interventional
2. Study Status
Record Verification Date
July 2015
Overall Recruitment Status
Completed
Study Start Date
November 2011 (undefined)
Primary Completion Date
October 2013 (Actual)
Study Completion Date
August 2014 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Cerenis Therapeutics, SA
4. Oversight
Data Monitoring Committee
No
5. 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Homozygous Familial Hypercholesterolemia
Keywords
Homozygous Familial Hypercholesterolemia, Familial Hypercholesterolemia, HDL mimetic, ApoA-1
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
23 (Actual)
8. Arms, Groups, and Interventions
Arm Title
CER-001
Arm Type
Experimental
Arm Description
Open label single arm study of CER-001
Intervention Type
Drug
Intervention Name(s)
CER-001
Intervention Description
Biweekly infusion
Primary Outcome Measure Information:
Title
Percent change from baseline to follow-up in carotid mean vessel wall area
Description
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
Secondary Outcome Measure Information:
Title
Change in carotid vessel wall volume
Description
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.
Time Frame
Baseline then 6 months and/or ~2 weeks post final dose
10. Eligibility
Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
John J. Kastelein, MD PhD
Organizational Affiliation
Amsterdam UMC, location VUmc
Official's Role
Principal Investigator
Facility Information:
Facility Name
Clinical Research Facility
City
Hartford
State/Province
Connecticut
ZIP/Postal Code
06102
Country
United States
Facility Name
Clinical Research Facility
City
N. Massapequa
State/Province
New York
ZIP/Postal Code
11758
Country
United States
Facility Name
Clinical Research Facility
City
Chicoutimi
State/Province
Quebec
ZIP/Postal Code
G7H 7P2
Country
Canada
Facility Name
Clinical Research Facility
City
Quebec
ZIP/Postal Code
G1V4M6
Country
Canada
Facility Name
Clinical Research Facility
City
Rome
ZIP/Postal Code
100161
Country
Italy
Facility Name
Clinical Research Facility
City
Amsterdam
ZIP/Postal Code
1105AZ
Country
Netherlands
Facility Name
Clinical Research Facility
City
Maastricht
ZIP/Postal Code
6229 HX
Country
Netherlands
Facility Name
Clinical Research Facility
City
Nijmegen
ZIP/Postal Code
6500 HB
Country
Netherlands
Facility Name
Clinical Research Facility
City
Manchester
ZIP/Postal Code
M13 9WL
Country
United Kingdom
12. IPD Sharing Statement
Citations:
PubMed Identifier
10441095
Citation
Eriksson M, Carlson LA, Miettinen TA, Angelin B. Stimulation of fecal steroid excretion after infusion of recombinant proapolipoprotein A-I. Potential reverse cholesterol transport in humans. Circulation. 1999 Aug 10;100(6):594-8. doi: 10.1161/01.cir.100.6.594.
Results Reference
background
PubMed Identifier
10195926
Citation
Nanjee MN, Doran JE, Lerch PG, Miller NE. Acute effects of intravenous infusion of ApoA1/phosphatidylcholine discs on plasma lipoproteins in humans. Arterioscler Thromb Vasc Biol. 1999 Apr;19(4):979-89. doi: 10.1161/01.atv.19.4.979.
Results Reference
background
PubMed Identifier
18389214
Citation
Nieuwdorp M, Vergeer M, Bisoendial RJ, op 't Roodt J, Levels H, Birjmohun RS, Kuivenhoven JA, Basser R, Rabelink TJ, Kastelein JJ, Stroes ES. Reconstituted HDL infusion restores endothelial function in patients with type 2 diabetes mellitus. Diabetologia. 2008 Jun;51(6):1081-4. doi: 10.1007/s00125-008-0975-2. Epub 2008 Apr 4. No abstract available.
Results Reference
background
PubMed Identifier
18832751
Citation
Shaw JA, Bobik A, Murphy A, Kanellakis P, Blombery P, Mukhamedova N, Woollard K, Lyon S, Sviridov D, Dart AM. Infusion of reconstituted high-density lipoprotein leads to acute changes in human atherosclerotic plaque. Circ Res. 2008 Nov 7;103(10):1084-91. doi: 10.1161/CIRCRESAHA.108.182063. Epub 2008 Oct 2.
Results Reference
background
PubMed Identifier
14600188
Citation
Nissen SE, Tsunoda T, Tuzcu EM, Schoenhagen P, Cooper CJ, Yasin M, Eaton GM, Lauer MA, Sheldon WS, Grines CL, Halpern S, Crowe T, Blankenship JC, Kerensky R. Effect of recombinant ApoA-I Milano on coronary atherosclerosis in patients with acute coronary syndromes: a randomized controlled trial. JAMA. 2003 Nov 5;290(17):2292-300. doi: 10.1001/jama.290.17.2292.
Results Reference
background
PubMed Identifier
17387133
Citation
Tardif JC, Gregoire J, L'Allier PL, Ibrahim R, Lesperance J, Heinonen TM, Kouz S, Berry C, Basser R, Lavoie MA, Guertin MC, Rodes-Cabau J; Effect of rHDL on Atherosclerosis-Safety and Efficacy (ERASE) Investigators. Effects of reconstituted high-density lipoprotein infusions on coronary atherosclerosis: a randomized controlled trial. JAMA. 2007 Apr 18;297(15):1675-82. doi: 10.1001/jama.297.15.jpc70004. Epub 2007 Mar 26.
Results Reference
background
PubMed Identifier
20538165
Citation
Waksman R, Torguson R, Kent KM, Pichard AD, Suddath WO, Satler LF, Martin BD, Perlman TJ, Maltais JA, Weissman NJ, Fitzgerald PJ, Brewer HB Jr. A first-in-man, randomized, placebo-controlled study to evaluate the safety and feasibility of autologous delipidated high-density lipoprotein plasma infusions in patients with acute coronary syndrome. J Am Coll Cardiol. 2010 Jun 15;55(24):2727-35. doi: 10.1016/j.jacc.2009.12.067.
Results Reference
background
PubMed Identifier
11914243
Citation
Spieker LE, Sudano I, Hurlimann D, Lerch PG, Lang MG, Binggeli C, Corti R, Ruschitzka F, Luscher TF, Noll G. High-density lipoprotein restores endothelial function in hypercholesterolemic men. Circulation. 2002 Mar 26;105(12):1399-402. doi: 10.1161/01.cir.0000013424.28206.8f.
Results Reference
result
Learn more about this trial
Effect of CER-001 on Plaque Volume in Homozygous Familial Hypercholesterolemia (HoFH) Subjects
We'll reach out to this number within 24 hrs