NCT06211478

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

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
40

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Mar 2023

Shorter than P25 for phase_3

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

March 3, 2023

Completed
6 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 5, 2023

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

October 5, 2023

Completed
3 months until next milestone

First Submitted

Initial submission to the registry

January 9, 2024

Completed
9 days until next milestone

First Posted

Study publicly available on registry

January 18, 2024

Completed
Last Updated

January 18, 2024

Status Verified

December 1, 2023

Enrollment Period

6 months

First QC Date

January 9, 2024

Last Update Submit

January 9, 2024

Conditions

Gaucher Disease

Outcome Measures

Primary Outcomes (1)

  • 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

Study Arms (2)

cases who are taking vitamin E

EXPERIMENTAL

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 )

Drug: Vitamin E

controls who are not taking vitamin E treatment

NO INTERVENTION

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

Interventions

Vitamin EDRUG

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

Also known as: No other alternatives
cases who are taking vitamin E

Eligibility Criteria

Age2 Years - 18 Years
Sexall(Gender-based eligibility)
Gender Eligibility Detailsself representation of gender identity
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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 -

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Ain Shams University

Cairo, Abbasia, 11517, Egypt

Location

Related Publications (3)

  • 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.

    PMID: 23523857BACKGROUND

  • 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.

    PMID: 33335836BACKGROUND

  • Adly AAM, Ismail EAR, Ibrahim FA, Atef M, El Sayed KA, Aly NH. A 6-month randomized controlled trial for vitamin E supplementation in pediatric patients with Gaucher disease: Effect on oxidative stress, disease severity and hepatic complications. J Inherit Metab Dis. 2025 Jan;48(1):e12792. doi: 10.1002/jimd.12792. Epub 2024 Aug 25.

    PMID: 39183538DERIVED

MeSH Terms

Conditions

Gaucher Disease

Interventions

Vitamin E

Condition Hierarchy (Ancestors)

SphingolipidosesLysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLipidosesLipid Metabolism, Inborn ErrorsLysosomal Storage DiseasesMetabolic DiseasesNutritional and Metabolic DiseasesLipid Metabolism Disorders

Intervention Hierarchy (Ancestors)

BenzopyransPyransHeterocyclic Compounds, 1-RingHeterocyclic CompoundsHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-Ring

Study Officials

  • Amira A Adly, Phd

    Ain Shams University

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
professor of pediatrics

Study Record Dates

First Submitted

January 9, 2024

First Posted

January 18, 2024

Study Start

March 3, 2023

Primary Completion

September 5, 2023

Study Completion

October 5, 2023

Last Updated

January 18, 2024

Record last verified: 2023-12

Data Sharing

IPD Sharing
Will not share

Locations

EG(1)