Clinical Audit of Managment of Gausher Disease in Children | NCT03590548

Studies
Clinical Audit of Managment of Gausher Disease in Children

NCT03590548

Sponsor

Yasmeen Abd Elbaset Hamzaa

Status

Unknown status

Last Update Posted

Jul 18, 2018Actual

Enrollment

1Estimated

Phase

Not provided

Conditions

Gaucher Disease

Interventions

gausher disease

Countries

Not provided

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Jul 2018

Overall Recruitment Status or Expanded Access Status

Unknown status

Last Known Status

Not yet recruiting

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jul 2018Estimated

Primary Completion Date

Dec 2018Estimated

Completion Date

Jul 2019Estimated

First Submitted Date

Jul 7, 2018

First Submission Date that Met QC Criteria

Jul 7, 2018

First Posted Date

Jul 18, 2018Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jul 7, 2018

Last Update Posted Date

Jul 18, 2018Actual

Sponsor/Collaborators Module

Responsible Party, by Official Title

Yasmeen Abd Elbaset Hamzaa, Assiut University, Assiut UniversitySponsor-Investigator

Lead Sponsor

Yasmeen Abd Elbaset HamzaaOTHER

Collaborators

Not provided

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Yes

Target Follow-Up Duration

6 Months

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

1Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
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

Interventions

Name	Type	Description	Arm Group Labels	Other Names
gausher disease	Diagnostic Test	lab investigation and radio logical	gausher disease in children

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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

Secondary Outcomes

Not provided

Other Outcomes

Not provided

Eligibility Module

Eligibility 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.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

1 Month

Maximum Age

18 Years

Standard Ages

ChildAdult

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

Sampling Method

Non-Probability Sample

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
yasmeen hamzaa, graduted	Contact	01121912793		Yasmeenhamzaa88@gmail.com

Overall Officials

Name	Affiliation	Role
Mervit Amin, doctora	Assiut unversity	Principal Investigator

Locations

Not provided

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D005776	Gaucher Disease

Ancestor Terms

ID	Term
D013106	Sphingolipidoses
D020140	Lysosomal Storage Diseases, Nervous System
D020739	Brain Diseases, Metabolic, Inborn
D001928	Brain Diseases, Metabolic

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