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Clinical Audit of Managment of Gausher Disease in Children

Assiut University Children 's Hospital

Status
UNKNOWN
Phases
Unknown
Study type
Observational
Source
ClinicalTrials.gov
Registry ID
NCT03590548
Enrollment
1
Registered
2018-07-18
Start date
2018-07-31
Completion date
2019-07-31
Last updated
2018-07-18

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Gaucher Disease

Brief summary

clinical audit on managment of gausher disease in children

Detailed 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.

Interventions

DIAGNOSTIC_TESTgausher disease

lab investigation and radio logical

Sponsors

Yasmeen Abd Elbaset Hamzaa
Lead SponsorOTHER

Study design

Observational model
CASE_ONLY
Time perspective
OTHER

Eligibility

Sex/Gender
ALL
Age
1 Months to 18 Years
Healthy volunteers
No

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.

Design outcomes

Primary

MeasureTime frameDescription
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)one year• Type of study : clinical audit

Contacts

Primary Contactyasmeen hamzaa, graduted
Yasmeenhamzaa88@gmail.com01121912793

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026