ThalassaemiaNCT06720480
Thalassaemia Severity
An observational study of Thalassaemia, run by Assiut University.
Trial summary
This study aims to develop a nuanced understanding of thalassemia severity in Upper Egypt by integrating various clinical parameters beyond transfusion frequency. By doing so, it seeks to enhance patient management and ultimately improve quality of life for those affected by this condition. Read more about this study
- Who is it for?
- Ages 6 months to 18 years
- Conditions the study is for
- Thalassaemia
- What is being tested?
- Homglobin Electrophoresis
- How many people
- 85 participants
- Where
- No sites listed yet
- Study dates
- 31 Dec 2024to 31 Dec 2025
- 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 6 months to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Children from 6month till 18years who have thalassaemia
Who may be able to join
- Children and adolescents aged from 18 month to 18 year old. Those with thalassemia
Who cannot join
- Non Thalassaemia children
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Thalassaemia Severity Score in Upper Egypt Patients Attending Assuit University Children Hospital
This study aims to develop a nuanced understanding of thalassemia severity in Upper Egypt by integrating various clinical parameters beyond transfusion frequency. By doing so, it seeks to enhance patient management and ultimately improve quality of life for those affected by this condition.
Read the detailed description
Thalassemia syndrome classified as hemoglobinopathies, represent one of the most prevalent classes of single-gene disorders globally. The mutations in the HBB gene lead to variable impacts on the production of globin proteins, which in turn affects the overall function of hemoglobin. The genetic variants contribute significantly to the diverse clinical presentations and severity of thalassemia. Traditionally, thalassemia has been categorized into Major, intermediate, and Minor based on the frequency of blood transfusions required for survival It has been observed that anemia may not be the sole determinant of thalassemic disease severity, other factors are also responsible for overall clinical status.
Accordingly, clinical conditions of the thalassemia patient, cannot classified based on the transfusion status.
Phadke et al. (2006), proposed a classification that considers multiple parameters beyond transfusion status . Similarly, Sripichai et al , (2008) classified HbE/β-thalassemia into the 3 categories of mild, moderate, and severe. Another classification was also introduced by Thalassemia International Federation (TIF) , which 'was bit modification Sripichai et al 2008 . Despite these advancements, existing classifications still tend to simplify thalassemia into three categories Globally, An estimated 1-5% of the global population are carriers of a genetic thalassemia mutation.Although the epidemiology of the various clinical forms remains poorly recognized, the disease is known to be highly prevalent in the area extending from sub-Saharan Africa, through the Mediterranean region and Middle East, to the Indian subcontinent and East and Southeast Asia.Thus, \>90% of patients with these disorders live in low- and middle-income countries
- Conditions
- Thalassaemia
- Being tested
- Homglobin Electrophoresis
- Keywords
- Thalassaemia severity score
- Medical subject headings
- Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Homglobin ElectrophoresisDiagnostic test
To diagnose thalassaemia \&best method to control disease progress
What is being measured
The primary outcome is the main question the study is designed to answer.
Scoring thalassaemia severity
Measured: 1year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Dec 2024
- Study start31 Dec 2024 (estimated)
- Primary completion1 Dec 2025 (estimated)
- Study completion31 Dec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for ThalassaemiaStudies of the same condition, open and under way.
- More trials run by Assiut UniversityThe sponsor's other registered studies.
- 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
- Cross-sectional
- Enrolment
- 85 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assiut University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Alzhraa gaber mohamed, Assiut University
- Investigators
- Shereen Mansour Galal, Associate professor, Study director, Assiut University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06720480
- Sponsor's protocol ID
- Thalassaemia severity
- Status last verified
- Dec 2024
- First submitted
- 3 Dec 2024
- Last updated
- 6 Dec 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Danjou F, Francavilla M, Anni F, Satta S, Demartis FR, Perseu L, Manca M, Sollaino MC, Manunza L, Mereu E, Marceddu G, Pissard S, Joly P, Thuret I, Origa R, Borg J, Forni GL, Piga A, Lai ME, Badens C, Moi P, Galanello R. A genetic score for the prediction of beta-thalassemia severity. Haematologica. 2015 Apr;100(4):452-7. doi: 10.3324/haematol.2014.113886. Epub 2014 Dec 5.
PubMed 25480500
Study at a glance
From the registry record, last updated by the sponsor on 6 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Thalassaemia Severity Score in Upper Egypt Patients Attending Assuit University Children Hospital
- ClinicalTrials.gov ID
- NCT06720480
- Acronym / sponsor ID
- Thalassaemia severity
- Conditions
- Thalassaemia
- Keywords
- Thalassaemia severity score
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 85 participants (estimated)
- Lead sponsor
- Assiut UniversityUniversity, hospital or other
- Study start
- 31 Dec 2024 (estimated)
- Primary completion
- 1 Dec 2025 (estimated)
- Study completion
- 31 Dec 2025 (estimated)
- First posted
- 6 Dec 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.
This study aims to develop a nuanced understanding of thalassemia severity in Upper Egypt by integrating various clinical parameters beyond transfusion frequency. By doing so, it seeks to enhance patient management and ultimately improve quality of life for those affected by this condition.
Detailed description
Thalassemia syndrome classified as hemoglobinopathies, represent one of the most prevalent classes of single-gene disorders globally. The mutations in the HBB gene lead to variable impacts on the production of globin proteins, which in turn affects the overall function of hemoglobin. The genetic variants contribute significantly to the diverse clinical presentations and severity of thalassemia. Traditionally, thalassemia has been categorized into Major, intermediate, and Minor based on the frequency of blood transfusions required for survival It has been observed that anemia may not be the sole determinant of thalassemic disease severity, other factors are also responsible for overall clinical status.
Accordingly, clinical conditions of the thalassemia patient, cannot classified based on the transfusion status.
Phadke et al. (2006), proposed a classification that considers multiple parameters beyond transfusion status . Similarly, Sripichai et al , (2008) classified HbE/β-thalassemia into the 3 categories of mild, moderate, and severe. Another classification was also introduced by Thalassemia International Federation (TIF) , which 'was bit modification Sripichai et al 2008 . Despite these advancements, existing classifications still tend to simplify thalassemia into three categories Globally, An estimated 1-5% of the global population are carriers of a genetic thalassemia mutation.Although the epidemiology of the various clinical forms remains poorly recognized, the disease is known to be highly prevalent in the area extending from sub-Saharan Africa, through the Mediterranean region and Middle East, to the Indian subcontinent and East and Southeast Asia.Thus, \>90% of patients with these disorders live in low- and middle-income countries
Study design
- Observational model
- Case only
- Time perspective
- Cross-sectional
Who may qualify
- Ages
- 6 months to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Children from 6month till 18years who have thalassaemia
Key inclusion criteria (1 listed)
- Children and adolescents aged from 18 month to 18 year old. Those with thalassemia
Key exclusion criteria (1 listed)
- Non Thalassaemia children
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 |
|---|---|---|
| Homglobin ElectrophoresisTo diagnose thalassaemia \&best method to control disease progress | Diagnostic test | – |
Primary outcome
Scoring thalassaemia severity
Time frame: 1year
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 6 Dec 2024.
- Results publication: PubMed 25480500Danjou F, Francavilla M, Anni F, Satta S, Demartis FR, Perseu L, Manca M, Sollaino MC, Manunza L, Mereu E, Marceddu G, Pissard S, Joly P, Thuret I, Origa R, Borg J, Forni GL, Piga A, Lai ME, Badens C, Moi P, Galanello R. A genetic score for the prediction of beta-thalassemia severity. Haematologica. 2015 Apr;100(4):452-7. doi: 10.3324/haematol.2014.113886. Epub 2014 Dec 5.
- 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.
Shereen Mansour Galal, Associate professor study director
Assiut University
Questions about this trial
What is this clinical trial studying?
An observational study of Thalassaemia, run by Assiut University.
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: opening soon, as of the sponsor's last update on 6 Dec 2024. 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?
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?
Contact AlZhraa Gaber Mohamed, Master degree (800-555-5555 ext. 01091888986, alzhraagaber@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Scoring thalassaemia severity (time frame: 1year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06720480, which also lists 1 linked publication.
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 6 Dec 2024.