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NCT03590548 Clinical Trial

Clinical Audit of Managment of Gausher Disease in Children

Gaucher Disease Clinical Trial

Official title:
Assiut University Children 's Hospital

Clinical Trial Details — Status: Not yet recruiting

Administrative data
NCT number NCT03590548
Other study ID # egypt AssiutU
Secondary ID
Status Not yet recruiting
Phase
First received
Last updated
Start date July 2018
Est. completion date July 2019

Study information
Verified date July 2018
Source Assiut University
Contact yasmeen hamzaa, graduted
Phone 01121912793
Email Yasmeenhamzaa88@gmail.com
Is FDA regulated No
Health authority
Study type Observational [Patient Registry]

Clinical Trial Summary

clinical audit on managment of gausher disease in children

Description:

The main aim of study is to evaluate the management of Gausher disease patient under enzyme replacement therapy in Assuit University Children's Hospital and compare our policy for management with International guideline of management of Gausher disease in children (8)

- Type of study : clinical audit

- Inclusion criteria: All cases with confirmed diagnosis of Gaucher disease type 1 and type 3 receiving enzyme replacement therapy at Assuit University Children's Hospital.

- Site of study :Assuit University children's Hospital Methodiological

- Exclusion criteria 3-New diagnosed cases not on ERT. 2-other lipid storage disease.

- Patient& method The study will include 42 diagnosed Gaucher disease patient under enzyme replacement therapy attend the hematology unit in Assuit University Children's Hospital in one year Reviewing the proposal will be carried out before starting via the ethical committee of Assuit Faculty of Medicine.

- Ethical consideration The aim of the study will be explained to each patient before beginning of the process.

We will compare our policy in management of Gausher disease with International guideline of management of gausher disease

1-cases of Gaucher type 2.

Recruitment information / eligibility
Status Not yet recruiting
Enrollment 1
Est. completion date July 2019
Est. primary completion date December 2018
Accepts healthy volunteers No
Gender All
Age group 1 Month to 18 Years
Eligibility Inclusion Criteria:

- All cases with confirmed diagnosis of Gaucher disease type 1 and type 3 receiving enzyme replacement therapy at Assuit University Children's Hospital.

Exclusion Criteria:

- 1-cases of Gaucher type 2. 2-other lipid storage disease. 3-New diagnosed cases not on ERT.

Study Design

Related Conditions & MeSH terms

Intervention

Diagnostic Test:
• gausher disease
lab investigation and radio logical

Locations
Country Name City State
n/a

Sponsors (1)
Lead Sponsor Collaborator
Yasmeen Abd Elbaset Hamzaa

Outcome
Type Measure Description Time frame Safety issue
Primary evaluate the management of Gausher disease patient under enzyme replacement therapy in Assuit University Children's Hospital and compare our policy for management with International guideline of management of Gausher disease in children (8) • Type of study : clinical audit one year
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