Gaucher DiseaseNCT03590548
Clinical Audit of Managment of Gausher Disease in Children
An observational study of Gaucher Disease, run by Yasmeen Abd Elbaset Hamzaa.
Trial summary
clinical audit on managment of gausher disease in children Read more about this study
- Who is it for?
- Ages 1 month to 18 years
- Conditions the study is for
- Gaucher Disease
- What is being tested?
- gausher disease
- How many people
- 1 participants
- Where
- No sites listed yet
- Study dates
- Jul 2018to Jul 2019
- How long will I take part?
- 6 Months
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 month to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
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
Who may be able to join
- All cases with confirmed diagnosis of Gaucher disease type 1 and type 3 receiving enzyme replacement therapy at Assuit University Children's Hospital.
Who cannot join
- 1-cases of Gaucher type 2. 2-other lipid storage disease. 3-New diagnosed cases not on ERT.
Trial locations and contacts
No sites are listed yet.
About this study
Official title: Assiut University Children 's Hospital
clinical audit on managment of gausher disease in children
Read the 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.
- Conditions
- Gaucher Disease
- Being tested
- gausher disease
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
gausher diseaseDiagnostic test
lab investigation and radio logical
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Diagnostic Test: gausher disease |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: one year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jul 2018
- Study startJul 2018 (estimated)
- Primary completionDec 2018 (estimated)
- Study completionJul 2019 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gaucher DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Other
- Enrolment
- 1 participants (estimated)
- Target duration
- 6 Months
Sponsor and investigators
- Lead sponsor
- Yasmeen Abd Elbaset Hamzaa
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Yasmeen Abd Elbaset Hamzaa, Assiut University
- Investigators
- Mervit Amin, doctora, Principal investigator, Assiut unversity
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03590548
- Sponsor's protocol ID
- egypt AssiutU
- Status last verified
- Jul 2018
- First submitted
- 7 Jul 2018
- Last updated
- 18 Jul 2018
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Jul 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Assiut University Children 's Hospital
- ClinicalTrials.gov ID
- NCT03590548
- Acronym / sponsor ID
- egypt AssiutU
- Conditions
- Gaucher Disease
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 1 participants (estimated)
- Lead sponsor
- Yasmeen Abd Elbaset HamzaaUniversity, hospital or other
- Study start
- Jul 2018 (estimated)
- Primary completion
- Dec 2018 (estimated)
- Study completion
- Jul 2019 (estimated)
- First posted
- 18 Jul 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Case only
- Time perspective
- Other
- Target follow-up
- 6 Months
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| gausher disease in children | – | Diagnostic Test: gausher disease• 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. |
Who may qualify
- Ages
- 1 month to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- 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
Key inclusion criteria (1 listed)
- All cases with confirmed diagnosis of Gaucher disease type 1 and type 3 receiving enzyme replacement therapy at Assuit University Children's Hospital.
Key exclusion criteria (1 listed)
- 1-cases of Gaucher type 2. 2-other lipid storage disease. 3-New diagnosed cases not on ERT.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| gausher diseaselab investigation and radio logical | Diagnostic test | gausher disease in children |
Primary outcome
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
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jul 2018.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mervit Amin, doctora principal investigator
Assiut unversity
Questions about this trial
What is this clinical trial studying?
An observational study of Gaucher Disease, run by Yasmeen Abd Elbaset Hamzaa.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 18 Jul 2018. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact yasmeen hamzaa, graduted (01121912793, Yasmeenhamzaa88@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Jul 2018.