The Role of the Coagulation Pathway at the Synapse in Prion Diseases

NCT ID: NCT02480725

Last Updated: 2015-06-24

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

UNKNOWN

Total Enrollment

50 participants

Study Classification

OBSERVATIONAL

Study Start Date

2015-06-30

Study Completion Date

2025-07-31

Brief Summary

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The study hypothesis is that that the deleterious effect of prions on the brain may be mediated (at least partially) by activation of serine proteases involved in the coagulation system. If this is true, then measurement of the activity of the coagulation system may be a marker of disease onset (in at higher risk individuals such as E200K\* carriers) and for disease progression or activity in affected individuals. In addition, modulation of the coagulation system activity may be a potential tool for therapeutic intervention.

\*E200K- E200K mutation (Glu to Lys substitution) in the prion protein gene

Detailed Description

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We plan to collect Cerebrospinal fluid (CSF) samples for thrombin activity assay in order to test whether there is a difference in thrombin activity in the CSF between CJD (Creutzfeldt-Jakob disease) and non-CJD patients. CSF samples will be obtained from two sources 1. Patients with familial or sporadic CJD and control patients with other neurodegenerative disorders (e.g. SDAT\*\*, NPH) that will be evaluated in Sheba Medical Center 2. From our collaborating group of Prof. Zerr in the German Prion Referral Center at the University of Gottingen which has a collection of thousands of CSF samples from patients with familial and sporadic CJD as well as ideal controls with other degenerative brain disease.

The study has 2 sections:

1. Prospective part in which we plan to recruit 25 patients with CJD and 25 patients with other types of dementia from Sheba Medical Center (SMC). Prior to inclusion in the study a senior neurologist will interview the patient and will verify that he fully understands the objectives of the study and he is mentally qualified to sign the informed consent form (severely demented patients who will not be able to adequately consider the participation in the study will be excluded).

Cognitive performance will be evaluated using the Mini-mental Status Examination and Frontal Assessment Battery scales.

No clinical data other than the cognitive assessment and those needed for the clinical work up will be especially collected for this study.
2. Retrospective part in which CSF samples from CJD patients and patients with other type of dementia will be shipped to us from our collaborators in Germany ans will be assayed for Thrombin activity. We plan to recruit to this part of the study 100-200 CJD patient CSF samples and a same number of samples from age matched controls.

Thrombin activity (for samples from both parts of the study) will be assayed as follows: CSF sample will be placed in a black 96 well dish (10 per well). Thrombin activity will be measured by a fluorometric assay, quantifying the cleavage of the synthetic peptide substrate Boc-Asp(OBzl)-Pro-Arg-AMC\*\*\* (I-1560, Bachem, Switzerland, 13 molar final concentration). Measurements will be performed by the Infinite 2000 microplate reader (Tecan, infinite 200, Switzerland) with excitation and emission filters of 360±35 and 460±35 nm, respectively. CSF testing for thrombin activity will be conducted in Professor Chapman's laboratory in Sheba. This laboratory is actively engaged in research on the role of thrombin and PAR-1 in diseases of the nervous system and is fully equipped to perform the biochemical and protein levels experiments.

The assay has the potential for commercialization as a diagnostic test for CJD. In addition, there is the potential to develop therapeutic agents targeting excessive thrombin activation.

\*\*SDAT=Senile Dementia of Alzheimer Type

\*\*\*AMC= Amino Methyl Coumarin

Conditions

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Creutzfeldt-Jakob Syndrome

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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CJD (Creutzfeldt-Jakob disease) patients

Prior to inclusion in the study a senior neurologist will interview the patient and will verify that he fully understands the objectives of the study and he is mentally qualified to sign the informed consent form (severely demented patients who will not be able to adequately consider the participation in the study will be excluded).

Cognitive performance will be evaluated using the Mini-mental Status Examination and Frontal Assessment Battery scales.

No clinical data other than the cognitive assessment and those needed for the clinical work up will be especially collected for this study.

We plan to collect CSF samples for thrombin activity assay.

No interventions assigned to this group

non-CJD patients with a type of dementia

Prior to inclusion in the study a senior neurologist will interview the patient and will verify that he fully understands the objectives of the study and he is mentally qualified to sign the informed consent form (severely demented patients who will not be able to adequately consider the participation in the study will be excluded).

Cognitive performance will be evaluated using the Mini-mental Status Examination and Frontal Assessment Battery scales.

No clinical data other than the cognitive assessment and those needed for the clinical work up will be especially collected for this study.

We plan to collect CSF samples for thrombin activity assay.

collect CSF (Cerebrospinal fluid) sample

Intervention Type PROCEDURE

collecting CSF samples for thrombin activity assay

CSF samples of non-CJD patient used as control

CSF samples : Thrombin activity will be assayed.

No interventions assigned to this group

Interventions

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collect CSF (Cerebrospinal fluid) sample

collecting CSF samples for thrombin activity assay

Intervention Type PROCEDURE

Eligibility Criteria

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Inclusion Criteria

* Patient undergoing lumbar puncture test as part of the investigation of cognitive decline.

Exclusion Criteria

* Patients on anticoagulation or those who have contraindication for undergoing lumbar puncture.
Minimum Eligible Age

35 Years

Maximum Eligible Age

90 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Prof. Zerr , Prion Referral Center , University of Gottingen, Germany

UNKNOWN

Sponsor Role collaborator

Sheba Medical Center

OTHER_GOV

Sponsor Role lead

Responsible Party

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Dr. Oren Cohen

MD

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Oren Cohen, Dr. (MD)

Role: PRINCIPAL_INVESTIGATOR

Cheba Medical Center

Central Contacts

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Oren Cohen, Dr. (MD)

Role: CONTACT

+972-52-6667584

References

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Deininger MH, Trautmann K, Magdolen V, Luther T, Schluesener HJ, Meyermann R. Cortical neurons of Creutzfeldt-Jakob disease patients express the urokinase-type plasminogen activator receptor. Neurosci Lett. 2002 May 10;324(1):80-2. doi: 10.1016/s0304-3940(02)00168-4.

Reference Type BACKGROUND
PMID: 11983300 (View on PubMed)

Zerr I, Bodemer M, Kaboth U, Kretzschmar H, Oellerich M, Armstrong VW. Plasminogen activities and concentrations in patients with sporadic Creutzfeldt-Jakob disease. Neurosci Lett. 2004 Nov 23;371(2-3):163-6. doi: 10.1016/j.neulet.2004.08.063.

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Almeida LM, Basu U, Khaniya B, Taniguchi M, Williams JL, Moore SS, Guan LL. Gene expression in the medulla following oral infection of cattle with bovine spongiform encephalopathy. J Toxicol Environ Health A. 2011;74(2-4):110-26. doi: 10.1080/15287394.2011.529061.

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Chapman J. Coagulation in inflammatory diseases of the central nervous system. Semin Thromb Hemost. 2013 Nov;39(8):876-80. doi: 10.1055/s-0033-1357482. Epub 2013 Oct 9.

Reference Type BACKGROUND
PMID: 24108468 (View on PubMed)

Maggio N, Shavit E, Chapman J, Segal M. Thrombin induces long-term potentiation of reactivity to afferent stimulation and facilitates epileptic seizures in rat hippocampal slices: toward understanding the functional consequences of cerebrovascular insults. J Neurosci. 2008 Jan 16;28(3):732-6. doi: 10.1523/JNEUROSCI.3665-07.2008.

Reference Type BACKGROUND
PMID: 18199772 (View on PubMed)

Maggio N, Itsekson Z, Dominissini D, Blatt I, Amariglio N, Rechavi G, Tanne D, Chapman J. Thrombin regulation of synaptic plasticity: implications for physiology and pathology. Exp Neurol. 2013 Sep;247:595-604. doi: 10.1016/j.expneurol.2013.02.011. Epub 2013 Feb 27.

Reference Type BACKGROUND
PMID: 23454608 (View on PubMed)

Maggio N, Cavaliere C, Papa M, Blatt I, Chapman J, Segal M. Thrombin regulation of synaptic transmission: implications for seizure onset. Neurobiol Dis. 2013 Feb;50:171-8. doi: 10.1016/j.nbd.2012.10.017. Epub 2012 Oct 25.

Reference Type BACKGROUND
PMID: 23103417 (View on PubMed)

Shavit E, Beilin O, Korczyn AD, Sylantiev C, Aronovich R, Drory VE, Gurwitz D, Horresh I, Bar-Shavit R, Peles E, Chapman J. Thrombin receptor PAR-1 on myelin at the node of Ranvier: a new anatomy and physiology of conduction block. Brain. 2008 Apr;131(Pt 4):1113-22. doi: 10.1093/brain/awn005. Epub 2008 Feb 25.

Reference Type BACKGROUND
PMID: 18299297 (View on PubMed)

Shavit E, Michaelson DM, Chapman J. Anatomical localization of protease-activated receptor-1 and protease-mediated neuroglial crosstalk on peri-synaptic astrocytic endfeet. J Neurochem. 2011 Nov;119(3):460-73. doi: 10.1111/j.1471-4159.2011.07436.x. Epub 2011 Sep 23.

Reference Type BACKGROUND
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Beilin O, Gurwitz D, Korczyn AD, Chapman J. Quantitative measurements of mouse brain thrombin-like and thrombin inhibition activities. Neuroreport. 2001 Aug 8;12(11):2347-51. doi: 10.1097/00001756-200108080-00013.

Reference Type BACKGROUND
PMID: 11496108 (View on PubMed)

Beilin O, Karussis DM, Korczyn AD, Gurwitz D, Aronovich R, Hantai D, Grigoriadis N, Mizrachi-Kol R, Chapman J. Increased thrombin inhibition in experimental autoimmune encephalomyelitis. J Neurosci Res. 2005 Feb 1;79(3):351-9. doi: 10.1002/jnr.20270.

Reference Type BACKGROUND
PMID: 15605378 (View on PubMed)

Beilin O, Karussis DM, Korczyn AD, Gurwitz D, Aronovich R, Mizrachi-Kol R, Chapman J. Increased KPI containing amyloid precursor protein in experimental autoimmune encephalomyelitis brains. Neuroreport. 2007 Apr 16;18(6):581-4. doi: 10.1097/WNR.0b013e328091c1e6.

Reference Type BACKGROUND
PMID: 17413661 (View on PubMed)

Itzekson Z, Maggio N, Milman A, Shavit E, Pick CG, Chapman J. Reversal of trauma-induced amnesia in mice by a thrombin receptor antagonist. J Mol Neurosci. 2014 May;53(1):87-95. doi: 10.1007/s12031-013-0200-8. Epub 2013 Dec 19.

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Bushi D, Chapman J, Katzav A, Shavit-Stein E, Molshatzki N, Maggio N, Tanne D. Quantitative detection of thrombin activity in an ischemic stroke model. J Mol Neurosci. 2013 Nov;51(3):844-50. doi: 10.1007/s12031-013-0072-y. Epub 2013 Jul 31.

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Llorens F, Zafar S, Ansoleaga B, Shafiq M, Blanco R, Carmona M, Grau-Rivera O, Nos C, Gelpi E, Del Rio JA, Zerr I, Ferrer I. Subtype and regional regulation of prion biomarkers in sporadic Creutzfeldt-Jakob disease. Neuropathol Appl Neurobiol. 2015 Aug;41(5):631-45. doi: 10.1111/nan.12175. Epub 2015 Apr 30.

Reference Type BACKGROUND
PMID: 25134744 (View on PubMed)

Other Identifiers

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1702-14 SMC

Identifier Type: -

Identifier Source: org_study_id

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