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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT06211478
Other study ID # FMASU MD72/2022
Secondary ID
Status Completed
Phase Phase 3
First received
Last updated
Start date March 3, 2023
Est. completion date October 5, 2023

Study information

Verified date December 2023
Source Ain Shams University
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

gaucher disease (GD) can be classified into three clinical types .type 1,the most common type ,is the chronic non neuronpathic form of the disease,which shows gighly variable signs and symptoms and variable course,with visceral,skeletal and hematologic involvement among others.the neurologocal involvement can be observed in types 2 and 3


Description:

enzymatic defeciency in gaucher disease patients may induce a cascade of events that result in side effects, such as the production of reactive oxygen species(ROS) and reactive nitrogen species(RNS) that can then generate the oxidative stress whereas in body of healthy individuals,the production and degradation of ROS and RNS are generally balanced. neurochemical abnormalities in patients with gaucher disease might be related to oxidative stress and inflammation in the brain. oxidative stress induces peroxiredoxin-2 as cytoprotective response against RNS .it is able to detoxify a vast range of organic peroxides. vitamin E well accepted nature most effective lipid soluble chain breaking antioxidant .


Recruitment information / eligibility

Status Completed
Enrollment 40
Est. completion date October 5, 2023
Est. primary completion date September 5, 2023
Accepts healthy volunteers No
Gender All
Age group 2 Years to 18 Years
Eligibility Inclusion Criteria: children and adolescents aged fron 2 to 18 years old with gaucher disease both type 1 and type 3 diagnosed clinically and confirmed by laboratory analysis patients who are on stable enzyme replacement therapy for at least 6 months - Exclusion Criteria: patients with infection or another inflammatory condition patients on multivitamin therapy known chronic liver disease (viral hepatitis,HIV) patients with renal impairement defined as creatinine level greater 1.2 for female and greater than 1.4 for male. -

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Vitamin E
Vitamin E will be taken for 6 months for gaucher disease patients who have oxidative stress and vitamin E defecient

Locations

Country Name City State
Egypt Ain Shams University Cairo Abbasia

Sponsors (1)

Lead Sponsor Collaborator
Ain Shams University

Country where clinical trial is conducted

Egypt, 

References & Publications (2)

Kartha RV, Terluk MR, Brown R, Travis A, Mishra UR, Rudser K, Lau H, Jarnes JR, Cloyd JC, Weinreb NJ. Patients with Gaucher disease display systemic oxidative stress dependent on therapy status. Mol Genet Metab Rep. 2020 Dec 9;25:100667. doi: 10.1016/j.ymgmr.2020.100667. eCollection 2020 Dec. — View Citation

Pacheco N, Uribe A. Enzymatic analysis of biomarkers for the monitoring of Gaucher patients in Colombia. Gene. 2013 May 25;521(1):129-35. doi: 10.1016/j.gene.2013.03.044. Epub 2013 Mar 21. — View Citation

Outcome

Type Measure Description Time frame Safety issue
Primary effect of vitamin E on oxidative stress and antioxidant markers in gaucher disease patients Measuring the oxidative stress markers in gaucher disease MDA and anti oxidant markers which are peroxiredoxin 2 ,Glutathione peroxidase,superoxide dismutase and reduced glutathione 6 months
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