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

NCT ID: NCT01412034

Last Updated: 2015-08-18

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

23 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-11-30

Study Completion Date

2014-08-31

Brief Summary

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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

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Conditions

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Homozygous Familial Hypercholesterolemia

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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CER-001

Open label single arm study of CER-001

Group Type EXPERIMENTAL

CER-001

Intervention Type DRUG

Biweekly infusion

Interventions

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CER-001

Biweekly infusion

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Cerenis Therapeutics, SA

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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John J. Kastelein, MD PhD

Role: PRINCIPAL_INVESTIGATOR

Amsterdam UMC, location VUmc

Locations

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Clinical Research Facility

Hartford, Connecticut, United States

Site Status

Clinical Research Facility

North Massapequa, New York, United States

Site Status

Clinical Research Facility

Chicoutimi, Quebec, Canada

Site Status

Clinical Research Facility

Québec, Quebec, Canada

Site Status

Clinical Research Facility

Rome, , Italy

Site Status

Clinical Research Facility

Amsterdam, , Netherlands

Site Status

Clinical Research Facility

Maastricht, , Netherlands

Site Status

Clinical Research Facility

Nijmegen, , Netherlands

Site Status

Clinical Research Facility

Manchester, , United Kingdom

Site Status

Countries

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United States Canada Italy Netherlands United Kingdom

References

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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.

Reference Type BACKGROUND
PMID: 10441095 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 10195926 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 18389214 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 18832751 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 14600188 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 17387133 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 20538165 (View on PubMed)

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.

Reference Type RESULT
PMID: 11914243 (View on PubMed)

Other Identifiers

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CER-001-CLIN-003

Identifier Type: -

Identifier Source: org_study_id

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