Ignite Creation Date:
2025-12-25 @ 2:30 AM
Ignite Modification Date:
2026-03-07 @ 4:00 PM
Study NCT ID:
NCT01412034
Status:
COMPLETED
Last Update Posted:
2015-08-18
First Post:
2011-08-05
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Effect of CER-001 on Plaque Volume in Homozygous Familial Hypercholesterolemia (HoFH) Subjects
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000090542', 'term': 'Homozygous Familial Hypercholesterolemia'}, {'id': 'D006938', 'term': 'Hyperlipoproteinemia Type II'}], 'ancestors': [{'id': 'D008052', 'term': 'Lipid Metabolism, Inborn Errors'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D006951', 'term': 'Hyperlipoproteinemias'}, {'id': 'D006949', 'term': 'Hyperlipidemias'}, {'id': 'D050171', 'term': 'Dyslipidemias'}, {'id': 'D052439', 'term': 'Lipid Metabolism Disorders'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000591544', 'term': 'CER-001'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 23}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2011-11'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2015-07', 'dispFirstSubmitDate': '2015-07-28', 'completionDateStruct': {'date': '2014-08', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2015-07-28', 'studyFirstSubmitDate': '2011-08-05', 'dispFirstSubmitQcDate': '2015-07-28', 'studyFirstSubmitQcDate': '2011-08-05', 'dispFirstPostDateStruct': {'date': '2015-08-18', 'type': 'ESTIMATED'}, 'lastUpdatePostDateStruct': {'date': '2015-08-18', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2011-08-08', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2013-10', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Percent change from baseline to follow-up in carotid mean vessel wall area', 'timeFrame': 'Baseline then 6 months and/or ~2 weeks post final dose', 'description': 'Percent change from baseline to follow-up in carotid mean vessel wall area'}], 'secondaryOutcomes': [{'measure': 'Change in carotid vessel wall volume', 'timeFrame': 'Baseline then 6 months and/or ~2 weeks post final dose', '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.'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['Homozygous Familial Hypercholesterolemia', 'Familial Hypercholesterolemia', 'HDL mimetic', 'ApoA-1'], 'conditions': ['Homozygous Familial Hypercholesterolemia']}, 'referencesModule': {'references': [{'pmid': '10441095', 'type': 'BACKGROUND', '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.'}, {'pmid': '10195926', 'type': 'BACKGROUND', '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.'}, {'pmid': '18389214', 'type': 'BACKGROUND', '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."}, {'pmid': '18832751', 'type': 'BACKGROUND', '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.'}, {'pmid': '14600188', 'type': 'BACKGROUND', '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.'}, {'pmid': '17387133', 'type': 'BACKGROUND', '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."}, {'pmid': '20538165', 'type': 'BACKGROUND', '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.'}, {'pmid': '11914243', 'type': 'RESULT', '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.'}]}, 'descriptionModule': {'briefSummary': '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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '12 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Male or female subject 12 years or older\n* Subject presents with Homozygous FH\n\nExclusion Criteria:\n\n* Weight \\>100 kg\n* Subjects with significant health problems in the recent past including blood disorders, cancer, or digestive problems\n* Female subjects of child-bearing potential\n* Known major hematologic, renal , hepatic, metabolic, gastrointestinal or endocrine dysfunction\n* Contraindication to MRI scanning that would preclude the use of contrast-enhanced 3TMRI'}, 'identificationModule': {'nctId': 'NCT01412034', 'acronym': 'MODE', 'briefTitle': 'Effect of CER-001 on Plaque Volume in Homozygous Familial Hypercholesterolemia (HoFH) Subjects', 'organization': {'class': 'INDUSTRY', 'fullName': 'Cerenis Therapeutics, SA'}, 'officialTitle': '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)', 'orgStudyIdInfo': {'id': 'CER-001-CLIN-003'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'CER-001', 'description': 'Open label single arm study of CER-001', 'interventionNames': ['Drug: CER-001']}], 'interventions': [{'name': 'CER-001', 'type': 'DRUG', 'description': 'Biweekly infusion', 'armGroupLabels': ['CER-001']}]}, 'contactsLocationsModule': {'locations': [{'zip': '06102', 'city': 'Hartford', 'state': 'Connecticut', 'country': 'United States', 'facility': 'Clinical Research Facility', 'geoPoint': {'lat': 41.76371, 'lon': -72.68509}}, {'zip': '11758', 'city': 'North Massapequa', 'state': 'New York', 'country': 'United States', 'facility': 'Clinical Research Facility', 'geoPoint': {'lat': 40.70093, 'lon': -73.46207}}, {'zip': 'G7H 7P2', 'city': 'Chicoutimi', 'state': 'Quebec', 'country': 'Canada', 'facility': 'Clinical Research Facility', 'geoPoint': {'lat': 48.41963, 'lon': -71.06369}}, {'zip': 'G1V4M6', 'city': 'Québec', 'state': 'Quebec', 'country': 'Canada', 'facility': 'Clinical Research Facility', 'geoPoint': {'lat': 46.81228, 'lon': -71.21454}}, {'zip': '100161', 'city': 'Rome', 'country': 'Italy', 'facility': 'Clinical Research Facility', 'geoPoint': {'lat': 41.89193, 'lon': 12.51133}}, {'zip': '1105AZ', 'city': 'Amsterdam', 'country': 'Netherlands', 'facility': 'Clinical Research Facility', 'geoPoint': {'lat': 52.37403, 'lon': 4.88969}}, {'zip': '6229 HX', 'city': 'Maastricht', 'country': 'Netherlands', 'facility': 'Clinical Research Facility', 'geoPoint': {'lat': 50.84833, 'lon': 5.68889}}, {'zip': '6500 HB', 'city': 'Nijmegen', 'country': 'Netherlands', 'facility': 'Clinical Research Facility', 'geoPoint': {'lat': 51.8425, 'lon': 5.85278}}, {'zip': 'M13 9WL', 'city': 'Manchester', 'country': 'United Kingdom', 'facility': 'Clinical Research Facility', 'geoPoint': {'lat': 53.48095, 'lon': -2.23743}}], 'overallOfficials': [{'name': 'John J. Kastelein, MD PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Amsterdam UMC, location VUmc'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Cerenis Therapeutics, SA', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}