Role of Vitamin E in Gaucher Disease Patients

Phase 3

Completed

• Conditions: Gaucher Disease
• Interventions: Drug: Vitamin E

• Registration Number: NCT06211478
• Lead Sponsor: Ain Shams University

Brief 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

Detailed 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 .

Study Timeline

• Study Start: 2023-03-03
• Primary Completion: 2023-09-05
• Study Completion: 2023-10-05
• Status Verified: 2023-12
• Study First Submitted: 2024-01-09
• Study First Submitted QC: 2024-01-09
• Last Update Submitted: 2024-01-09
• Study First Posted: 2024-01-18
• Last Update Posted: 2024-01-18

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 40

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

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
cases who are taking vitamin E	Vitamin E	gaucher disease patients with oxidative stress and vitamin E defeciency taking vitamin E treatment (weigh below 20 kg dose will be 200mg daily and whose weight above 20 kg dose will be 400mg daily for duration 6 months )

Primary Outcome Measures

Name	Time	Method
effect of vitamin E on oxidative stress and antioxidant markers in gaucher disease patients	6 months	Measuring the oxidative stress markers in gaucher disease MDA and anti oxidant markers which are peroxiredoxin 2 ,Glutathione peroxidase,superoxide dismutase and reduced glutathione

Trial Locations

Locations (1)

Ain Shams University; 🇪🇬 Cairo, Abbasia, Egypt