Examining the Immune Response in Patients With Gaucher Disease and Hepatitis C
NCT ID: NCT01274208
Last Updated: 2014-11-19
Study Results
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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UNKNOWN
80 participants
OBSERVATIONAL
2011-01-31
2016-04-30
Brief Summary
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* Investigate the anti-HCV response in patients with Gaucher disease(GD)
* Define the potential role of high levels of Glucocerebroside in the immune system
Study hypothesis:
High levels of Glucocerebroside can be used as a tool in the antiviral treatment of hepatitis C by potentiating the immune response of natural killer T cells and dendritic cells
Detailed Description
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One of the hallmarks of GD is its great phenotypic heterogeneity with variable presentations and symptoms, beginning with a lethal variant of infants dying at or near birth with hydrops fetalis and ichthyoids at one extreme and totally asymptomatic individuals without any physical or laboratory abnormalities at the other extreme.
This autosomal recessive disease is pan-ethnic, but it is especially prevalent among Ashkenazi Jews. From over 300 different mutations reported in the glucocerebrosidase gene, five account for 98% of the disease-producing alleles. Of these mutations, N370S (or 1226G) occurs in 1 out of 17 Ashkenazi individuals, leading to a disease frequency of 1:850 in this ethnic group.
The high prevalence of more than a single mutation among Ashkenazi Jews and the existence of two additional rare inherited lysosomal glycolipid storage diseases, Tay Sachs and Nieman Pick, at a higher prevalence within the same ethnic group is believed to be caused by selective advantage.
Available genetic data are consistent with a founder effect(4) whereas the nature of such an advantage has not been identified.
The aim of this study was to investigate the anti-HCV immune response in patients with GD in an attempt to define the potential role of high levels of GC in the immune system and antiviral immunity.
Study importance:
The host metabolic background exerts a profound effect on antiviral immunity, which may influence the clinical course of chronic HCV infection.
The accumulation of GC in patients with GD may provide a selective evolutionary advantage to these patients.
Glucocerebroside was recently tested in human trials and shown to be effective in altering NKT- dependent metabolic pathways, insulin resistance, and associated liver injury.
The present study examine the capability of Glucocerebroside to be be used as a tool in the antiviral treatment of hepatitis C by potentiating the immune response of natural killer T cells and dendritic cells.
Statistical Analysis:
Data are presented as the mean ± standard deviation (SD). The Kruskal Wallis non-parametric ANOVA test was used to identify differences between the study groups.
The student t-test and non-parametric Mann-Whitney test were used to compare quantitative variables between the study groups as appropriate; P \<0.05 was considered to be significant.
Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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Gaucher Disease with Hepatitis C
No interventions assigned to this group
Eligibility Criteria
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Inclusion Criteria
* Patients with hepatitis C without Gaucher disease
* Individuals or patients without Gaucher disease and hepatitis C
* Individuals or patients who signed an approval for the research
* Men and women 18\< years of age , pregnant women
Exclusion Criteria
* Acute liver disease that can alter the lab results , such as: Rt. congestive heart failure
severe infection ,inflammation, medication such as : Statins , Isoniazid ,
Amiodarone
\- Patients who received treatment for hepatitis C such as: Interferon ,
Pegylated interferon , Ribavirin
\-
18 Years
90 Years
ALL
Yes
Sponsors
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Shaare Zedek Medical Center
OTHER
Responsible Party
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Ari Zimran
Prof. Ari Zimran
Principal Investigators
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Bernardo Melamud, Dr.
Role: PRINCIPAL_INVESTIGATOR
Gaucher Clinic , Shaare zedek Hospital
Locations
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Shaare Zedek , Medical Center
Jerusalem, , Israel
Hadassah Medical Center
Jerusalem, , Israel
Countries
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Central Contacts
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Facility Contacts
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References
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Elstein D, Abrahamov A, Hadas-Halpern I, Zimran A. Gaucher's disease. Lancet. 2001 Jul 28;358(9278):324-7. doi: 10.1016/S0140-6736(01)05490-3. No abstract available.
Butters TD. Gaucher disease. Curr Opin Chem Biol. 2007 Aug;11(4):412-8. doi: 10.1016/j.cbpa.2007.05.035. Epub 2007 Jul 23.
Beutler E. Gaucher disease: multiple lessons from a single gene disorder. Acta Paediatr Suppl. 2006 Apr;95(451):103-9. doi: 10.1111/j.1651-2227.2006.tb02398.x.
Slatkin M. A population-genetic test of founder effects and implications for Ashkenazi Jewish diseases. Am J Hum Genet. 2004 Aug;75(2):282-93. doi: 10.1086/423146. Epub 2004 Jun 18.
Beutler E. Gaucher disease as a paradigm of current issues regarding single gene mutations of humans. Proc Natl Acad Sci U S A. 1993 Jun 15;90(12):5384-90. doi: 10.1073/pnas.90.12.5384.
Zigmond E LG, Pappo O, Zangen S, Levy Sklair M, Hemed N, Rabbani E, Itamar R, Ilan Y, Margalit M. Treatment of non-alcoholic steatohepatitis by B-glucosylceramide: A phase I/II clinical study. Hepatology 2006;44 .180A, .
Other Identifiers
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SZMC- 89/10
Identifier Type: -
Identifier Source: org_study_id