Role of Vitamin E in Gaucher Disease Patients

Gaucher Disease Clinical Trial

Official title:

Role of Vitamin E as an Adjuvant Therapy for Patients With Gaucher Disease Under Enzyme Replacement Therapy in Relation to Oxidative Stress

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT06211478
• Other study ID #: FMASU MD72/2022
• Status: Completed
• Phase: Phase 3
• 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
• 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.

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Related Conditions & MeSH terms

• Gaucher Disease

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