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

NCT ID: NCT06627543

Last Updated: 2024-10-04

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ENROLLING_BY_INVITATION

Total Enrollment

44 participants

Study Classification

OBSERVATIONAL

Study Start Date

2024-04-01

Study Completion Date

2025-03-01

Brief Summary

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

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Detailed Description

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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

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Gaucher Disease Type 3

Study Design

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Observational Model Type

CASE_CONTROL

Study Time Perspective

PROSPECTIVE

Study Groups

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Gaucher type patients

echo

Intervention Type DIAGNOSTIC_TEST

echocardiographic

Interventions

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echo

echocardiographic

Intervention Type DIAGNOSTIC_TEST

Eligibility Criteria

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Inclusion Criteria

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

Exclusion Criteria

* Age18 years old
* Cases of gaucher type 1 and 2
* Other lipid storage disease
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Noha Osman

OTHER

Sponsor Role lead

Responsible Party

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Noha Osman

Lecturer

Responsibility Role SPONSOR_INVESTIGATOR

Locations

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Ahmed Mohammed Mohammed

Asyut, , Egypt

Site Status

Countries

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Egypt

Other Identifiers

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04-2023-100034

Identifier Type: -

Identifier Source: org_study_id

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