Role of Vitamin E in Gaucher Disease Patients

Ain Shams University

Status and phase: Completed; Phase 3

Conditions: Gaucher Disease

Treatments: Drug: Vitamin E

Study type: Interventional

Funder types: Other

Identifiers

NCT06211478

FMASU MD72/2022

Details and patient eligibility

About

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

Full 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 .

Enrollment: 40 patients

Sex: All

Ages: 2 to 18 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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40 participants in 2 patient groups

cases who are taking vitamin E

Experimental group

Description:

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 )

Treatment:

Drug: Vitamin E

controls who are not taking vitamin E treatment

No Intervention group

Description:

gaucher disease patients with oxidative stress and vitamin E defeciency not taking vitamin E treatment but take the standard of care which is enzyme replacement therapy