NCT01515241

Brief Summary

Despite the availability of several classes of very effective drugs available to treat heterozygous Familial Hypercholesterolemia (HeFH), there remains a large unmet medical need for new, effective and well tolerated therapies. There are a number of therapies given on a chronic basis to reduce long term risk, such as statins, fibrates, niacin, omega 3 fatty acids, resins, cholesterol absorption inhibitors and antiplatelet or anticoagulant drugs, but subjects with heterozygous Familial Hypercholesterolemia remain at high risk for cardiovascular events. There is still a need for acute therapies that can lead to rapid pacification of unstable plaque in order to reduce the risk of these events. 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 (3TMRI)and intravascular ultrasound (IVUS) evaluations in patients with HeFH.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
10

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Jan 2012

Shorter than P25 for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 1, 2012

Completed
17 days until next milestone

First Submitted

Initial submission to the registry

January 18, 2012

Completed
6 days until next milestone

First Posted

Study publicly available on registry

January 24, 2012

Completed
2 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2012

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2012

Completed
Last Updated

March 3, 2014

Status Verified

January 1, 2014

Enrollment Period

3 months

First QC Date

January 18, 2012

Last Update Submit

January 29, 2014

Conditions

Heterozygous Familial Hypercholesterolemia

Keywords

Heterozygous Familial HypercholesterolemiaFamilial HypercholesterolemiaHDL mimeticApoA-1

Outcome Measures

Primary Outcomes (1)

  • Change in Total Plaque Volume

    Nominal change in total plaque volume (ACTPV), as assessed by 3D IVUS, from the baseline measurement to the follow-up taken \~3 weeks following the final dose of study medication (approximately 10 weeks after the baseline assessment)

    Baseline and 3 weeks post final dose

Secondary Outcomes (2)

  • Percent Change in Plaque Volume

    Baseline and 3 weeks post final dose

  • Change in carotid plaque volume

    Baseline and 3 weeks post final dose

Study Arms (1)

Open label single arm study of CER-001

OTHER

Open label single arm study of CER-001

Drug: CER-001

Interventions

CER-001DRUG

Weekly injection

Open label single arm study of CER-001

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Male or Female subjects at least 18 years old
  • Subject presents heterozygous FH, known CHD and receiving maximally tolerated lipid modifying therapy, at stable doses for at least 3 months
  • LDL-C of \> 110 mg/dl
  • Angiographic evidence of coronary artery disease with suitable "target" coronary artery for IVUS

You may not qualify if:

  • Confirmed diagnosis of homozygous FH
  • Significant health problems (other than cardiovascular disease) in the recent past including blood disorders, cancer, or digestive problems
  • Female subjects not meeting the study definition of non child-bearing potential
  • Use of an investigational agent within 30 days of the first CER-001 dose
  • Receiving current lipid apheresis

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Montreal Heart Institute

Montreal, Quebec, H1T1C8, Canada

Location

Related Publications (8)

  • 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: 10441095BACKGROUND

  • 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: 10195926BACKGROUND

  • 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.

    PMID: 11914243BACKGROUND

  • 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: 18389214BACKGROUND

  • 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: 18832751BACKGROUND

  • 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: 14600188BACKGROUND

  • 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: 17387133BACKGROUND

  • 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: 20538165BACKGROUND

MeSH Terms

Conditions

Hyperlipoproteinemia Type II

Interventions

CER-001

Condition Hierarchy (Ancestors)

Lipid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesHyperlipoproteinemiasHyperlipidemiasDyslipidemiasLipid Metabolism DisordersMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Jean-Claude Tardif, MD

    Montreal Heart Institute

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 18, 2012

First Posted

January 24, 2012

Study Start

January 1, 2012

Primary Completion

April 1, 2012

Study Completion

May 1, 2012

Last Updated

March 3, 2014

Record last verified: 2014-01

Locations

CA(1)