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Trial record 15 of 20 for:    Lysosomal | Recruiting, Not yet recruiting, Available Studies | "Gaucher Disease"

Clinical Audit of Managment of Gausher Disease in Children

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ClinicalTrials.gov Identifier: NCT03590548
Recruitment Status : Not yet recruiting
First Posted : July 18, 2018
Last Update Posted : July 18, 2018
Sponsor:
Information provided by (Responsible Party):
Yasmeen Abd Elbaset Hamzaa, Assiut University

Brief Summary:
clinical audit on managment of gausher disease in children

Condition or disease Intervention/treatment
Gaucher Disease Diagnostic Test: gausher disease

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.


Study Type : Observational [Patient Registry]
Estimated Enrollment : 1 participants
Observational Model: Case-Only
Time Perspective: Other
Target Follow-Up Duration: 6 Months
Official Title: Assiut University Children 's Hospital
Estimated Study Start Date : July 2018
Estimated Primary Completion Date : December 2018
Estimated Study Completion Date : July 2019


Group/Cohort Intervention/treatment
gausher disease in children
• 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.
Diagnostic Test: gausher disease
lab investigation and radio logical




Primary Outcome Measures :
  1. 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) [ Time Frame: one year ]
    • Type of study : clinical audit



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Ages Eligible for Study:   1 Month to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
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
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03590548


Contacts
Contact: yasmeen hamzaa, graduted 01121912793 Yasmeenhamzaa88@gmail.com

Sponsors and Collaborators
Yasmeen Abd Elbaset Hamzaa
Investigators
Principal Investigator: Mervit Amin, doctora Assiut unversity

Responsible Party: Yasmeen Abd Elbaset Hamzaa, Assiut University
ClinicalTrials.gov Identifier: NCT03590548     History of Changes
Other Study ID Numbers: egypt AssiutU
First Posted: July 18, 2018    Key Record Dates
Last Update Posted: July 18, 2018
Last Verified: July 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Gaucher Disease
Lysosomal Storage Diseases, Nervous System
Lysosomal Storage Diseases
Sphingolipidoses
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipidoses
Lipid Metabolism, Inborn Errors
Metabolic Diseases
Lipid Metabolism Disorders