NCT05816993

Brief Summary

The current work aims to detect the frequency and types of neurological disorders in patient diagnosed as Gaucher disease in Assiut University Children's Hospital

Trial Health

35
At Risk

Trial Health Score

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

Trial has exceeded expected completion date
Enrollment
45

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started Apr 2023

Status
unknown

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

First Submitted

Initial submission to the registry

March 30, 2023

Completed
19 days until next milestone

First Posted

Study publicly available on registry

April 18, 2023

Completed
2 days until next milestone

Study Start

First participant enrolled

April 20, 2023

Completed
12 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2024

Completed
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

April 11, 2025

Completed
Last Updated

April 18, 2023

Status Verified

April 1, 2023

Enrollment Period

12 months

First QC Date

March 30, 2023

Last Update Submit

April 14, 2023

Conditions

Gaucher Disease

Outcome Measures

Primary Outcomes (1)

  • Number of participants with neurological disorders in pediatric patients diagnosed as Gaucher disease

    Types of neurological disorders in patients with Gaucher disease

    Baseline

Eligibility Criteria

Age1 Month - 18 Years
Sexall
Age GroupsChild (0-17), Adult (18-64)
Sampling MethodNon-Probability Sample
Study Population

Gaucher disease patients from age 1m to age 18y with neurological manifestations

You may qualify if:

  • Diagnosis of Gaucher Disease

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

Gaucher Disease

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

Study Officials

  • Mohamed Ghazaly, Prof

    Assiut University

    PRINCIPAL INVESTIGATOR

  • Mostafa Embaby, Prof

    Assiut University

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Gehan Galal

CONTACT

01011087742gehangalal2030@gmail.com

Study Design

Study Type
observational
Observational Model
OTHER
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Principle investigatir

Study Record Dates

First Submitted

March 30, 2023

First Posted

April 18, 2023

Study Start

April 20, 2023

Primary Completion

April 1, 2024

Study Completion

April 11, 2025

Last Updated

April 18, 2023

Record last verified: 2023-04

Data Sharing

IPD Sharing
Will not share