NCT06627543

Effect of Enzyme Replacement Therapy on Cardiac Function in Children With Gaucher Disease Type 3

• Status: Enrolling By Invitation
• Phase: —
• Study type: Observational
• Enrollment: 44 (estimated)
• Sponsor: Noha Osman · Academic / Other
• Sex: All
• Age: —
• Healthy volunteers: —

Summary

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

Detailed description

evaluate cardiac involvement in a group of GD type 3 patients, evaluate pulmonary hypertension and valvular involvement, compare the findings of basic echo before the start of replacement therapy as well as with healthy controls, to assess the effects of enzyme replacement therpay on the cardiac function.

Conditions

• Gaucher Disease Type 3

Interventions

Type	Name	Description
DIAGNOSTIC_TEST	echo	echocardiographic

Timeline

• Start date: 2024-04-01
• Primary completion: 2025-01-01
• Completion: 2025-03-01
• First posted: 2024-10-04
• Last updated: 2024-10-04

Locations

1 site across 1 country: Egypt