Gaucher Disease Type 3NCT06627543
Effect of Enzyme Replacement Therapy on Cardiac Function in Children With Gaucher Disease Type 3
An observational study of Gaucher Disease Type 3, run by Noha Osman at 1 site in Egypt.
Trial summary
all cases confirmed diagnosis of Gaucher disease type 3 recieving enzyme replacement therapy at Assiut university hospital Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Gaucher Disease Type 3
- What is being tested?
- echo
- How many people
- 44 participants
- Where
- 1 siteEgypt
- Study dates
- 1 Apr 2024to 1 Mar 2025
- How long will I take part?
- 6 Months
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 Any age
- All sexes
- Child, Adult, Older adult
Study population: gaucher diseease type 3
Who may be able to join
- Any sex
- No age limit
- Gaucher disease type 3
- Receiving enzyme replacement therapy
Who cannot join
- Age18 years old
- Cases of gaucher type 1 and 2
- Other lipid storage disease
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
About this study
all cases confirmed diagnosis of Gaucher disease type 3 recieving enzyme replacement therapy at Assiut university hospital
Read the detailed description
evaluate cardiac involvement in a group of GD type 3 patients, evaluate pulmonary hypertension and valvular involvement, compare the findings of basic echo before the start of replacement therapy as well as with healthy controls, to assess the effects of enzyme replacement therpay on the cardiac function.
- Conditions
- Gaucher Disease Type 3
- Being tested
- echo
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
echoDiagnostic test
echocardiographic
| Study group | Role | What they receive |
|---|---|---|
| Gaucher type patients | Not described Receives: Diagnostic Test: echo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluate Pulmonary Hypertension (PH) in Egyptian GD Type 3 Patients
To assess the presence and severity of pulmonary hypertension using echocardiography. Unit of Measure: mmHg (for systolic pulmonary artery pressure)
Measured: 6 months
Evaluate Valvular Involvement in Egyptian GD Type 3 Patients
To assess the degree of valvular involvement, including stenosis or regurgitation, using echocardiography. Unit of Measure: Severity grading (mild, moderate, severe)
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Oct 2024
- Study start1 Apr 2024
- Primary completion1 Jan 2025 (estimated)
- Study completion1 Mar 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gaucher Disease Type 3Studies 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-control
- Time perspective
- Prospective
- Enrolment
- 44 participants (estimated)
- Target duration
- 6 Months
Sponsor and investigators
- Lead sponsor
- Noha Osman
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Noha Osman, Assiut University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06627543
- Sponsor's protocol ID
- 04-2023-100034
- Status last verified
- Oct 2024
- First submitted
- 29 Sep 2024
- Last updated
- 4 Oct 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of Enzyme Replacement Therapy on Cardiac Function in Children With Gaucher Disease Type 3
- ClinicalTrials.gov ID
- NCT06627543
- Acronym / sponsor ID
- 04-2023-100034
- Conditions
- Gaucher Disease Type 3
- Study type
- Observational
- Recruitment status
- Enrolling by invitation
- Enrolment
- 44 participants (estimated)
- Lead sponsor
- Noha OsmanUniversity, hospital or other
- Study start
- 1 Apr 2024 (actual)
- Primary completion
- 1 Jan 2025 (estimated)
- Study completion
- 1 Mar 2025 (estimated)
- First posted
- 4 Oct 2024 (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.
all cases confirmed diagnosis of Gaucher disease type 3 recieving enzyme replacement therapy at Assiut university hospital
Detailed description
evaluate cardiac involvement in a group of GD type 3 patients, evaluate pulmonary hypertension and valvular involvement, compare the findings of basic echo before the start of replacement therapy as well as with healthy controls, to assess the effects of enzyme replacement therpay on the cardiac function.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Target follow-up
- 6 Months
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Gaucher type patients | – | Diagnostic Test: echo |
Who may qualify
- Sex
- All
- Age groups
- Child, Adult, Older adult
- Study population
- gaucher diseease type 3
Key inclusion criteria (4 listed)
- Any sex
- No age limit
- Gaucher disease type 3
- Receiving enzyme replacement therapy
Key exclusion criteria (3 listed)
- Age18 years old
- Cases of gaucher type 1 and 2
- Other lipid storage disease
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 |
|---|---|---|
| echoechocardiographic | Diagnostic test | Gaucher type patients |
Primary outcome
Evaluate Pulmonary Hypertension (PH) in Egyptian GD Type 3 Patients
Time frame: 6 months
To assess the presence and severity of pulmonary hypertension using echocardiography. Unit of Measure: mmHg (for systolic pulmonary artery pressure)
Evaluate Valvular Involvement in Egyptian GD Type 3 Patients
Time frame: 6 months
To assess the degree of valvular involvement, including stenosis or regurgitation, using echocardiography. Unit of Measure: Severity grading (mild, moderate, severe)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Oct 2024.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Gaucher Disease Type 3, run by Noha Osman at 1 site in Egypt.
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 4 inclusion and 3 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: enrolling by invitation, as of the sponsor's last update on 4 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Egypt. Whether a site is taking part right now should be confirmed with that site. See every site.
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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluate Pulmonary Hypertension (PH) in Egyptian GD Type 3 Patients (time frame: 6 months). The study lists 2 primary outcomes in all. 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 4 Oct 2024; AzTrials last refreshed its copy on 6 Nov 2026.