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
Verified date | December 2023 |
Source | Ain Shams University |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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
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. - |
Country | Name | City | State |
---|---|---|---|
Egypt | Ain Shams University | Cairo | Abbasia |
Lead Sponsor | Collaborator |
---|---|
Ain Shams University |
Egypt,
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
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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