NCT06627543

Brief Summary

all cases confirmed diagnosis of Gaucher disease type 3 recieving enzyme replacement therapy at Assiut university hospital

Trial Health

55
Monitor

Trial Health Score

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

Trial has exceeded expected completion date
Enrollment
44

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started Apr 2024

Shorter than P25 for all trials

Geographic Reach
1 country

1 active site

Status
enrolling by invitation

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

April 1, 2024

Completed
6 months until next milestone

First Submitted

Initial submission to the registry

September 29, 2024

Completed
5 days until next milestone

First Posted

Study publicly available on registry

October 4, 2024

Completed
3 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2025

Completed
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2025

Completed
Last Updated

October 4, 2024

Status Verified

October 1, 2024

Enrollment Period

9 months

First QC Date

September 29, 2024

Last Update Submit

October 3, 2024

Conditions

Gaucher Disease Type 3

Outcome Measures

Primary Outcomes (2)

  • Evaluate Pulmonary Hypertension (PH) in Egyptian GD Type 3 Patients

    To assess the presence and severity of pulmonary hypertension using echocardiography. Unit of Measure: mmHg (for systolic pulmonary artery pressure)

    6 months

  • Evaluate Valvular Involvement in Egyptian GD Type 3 Patients

    To assess the degree of valvular involvement, including stenosis or regurgitation, using echocardiography. Unit of Measure: Severity grading (mild, moderate, severe)

    6 months

Study Arms (1)

Gaucher type patients

Diagnostic Test: echo

Interventions

echoDIAGNOSTIC_TEST

echocardiographic

Gaucher type patients

Eligibility Criteria

Sexall
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

gaucher diseease type 3

You may qualify if:

  • Any sex
  • No age limit
  • Gaucher disease type 3
  • Receiving enzyme replacement therapy

You may not qualify if:

  • Age18 years old
  • Cases of gaucher type 1 and 2
  • Other lipid storage disease

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Ahmed Mohammed Mohammed

Asyut, Egypt

Location

MeSH Terms

Conditions

Gaucher Disease

Interventions

Caves

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)

Geological PhenomenaPhysical PhenomenaEnvironmentEcological and Environmental PhenomenaBiological PhenomenaEnvironment and Public Health

Study Design

Study Type
observational
Observational Model
CASE CONTROL
Time Perspective
PROSPECTIVE
Target Duration
6 Months
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Lecturer

Study Record Dates

First Submitted

September 29, 2024

First Posted

October 4, 2024

Study Start

April 1, 2024

Primary Completion

January 1, 2025

Study Completion

March 1, 2025

Last Updated

October 4, 2024

Record last verified: 2024-10

Locations

EG(1)