ThalassemiaNCT06058260
Assessment of Cognitive Function and Quality of Life in Thalassemic Children at Sohag University Hospital
An observational study of Thalassemia, run by Sohag University at 1 site in Egypt.
Trial summary
Thalassemia syndromes are a heterogeneous group of single gene disorders, inherited in an autosomal recessive manner ,prevalent among all ethnic groups and in almost every country around the world . Read more about this study
- Who is it for?
- Ages 4 to 18 years
- Conditions the study is for
- Thalassemia
- What is being tested?
- Pediatric Quality of Life Inventory (questionnaire) and The Stanford-Binet Intelligence Scale fifth edition
- How many people
- 100 participants
- Where
- 1 siteEgypt
- Study dates
- 20 Oct 2023to 20 Oct 2024
- 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 4 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: All the diagnosed thalassemic children age between 4 and 18 years.
Who may be able to join
- All the diagnosed thalassemic children age between 4 and 18 years.
Who cannot join
- Any other hematological disease.
- Age less than 4 years and more than 18 years.
Trial locations and contacts
1 site in 1 country.
Alzahraa A Ahmed, professor contact
About this study
Thalassemia syndromes are a heterogeneous group of single gene disorders, inherited in an autosomal recessive manner ,prevalent among all ethnic groups and in almost every country around the world .
Once a child has been diagnosed as thalassemia, he has to take lifelong treatment , where cure is not attainable and treatment may be prolonged . It is a life-threatening and life-limiting condition that affects the patient clinically and psychologically, so Health-related Quality of Life (HRQOL) is likely to be an essential outcome for these patients.
Read more
Quality of life in thalassemic children such as : Repeated visits to hospitals for regular blood transfusion, cost of chelation therapy, repeated laboratory tests for monitoring therapy and for early detection of any complications. also life-long costly therapy along with poor quality of life will have adverse impact on the family.
A better understanding of the factors associated with HRQOL among children with thalassemia could have a direct effect on the development of more suitable clinical, counselling and social support programs to enhance treatment outcomes.
Cognitive dysfunction was Reported either due to the disease or its treatment ,frequent school absences, frequent hospitalizations, and physical and social restrictions lead to cognitive dysfunction . This neurological involvement in thalassemic children is primarily silent, with subclinical manifestations that can only be detected by cognitive assessment tests.
Read the detailed description
Thalassemia syndromes are a heterogeneous group of single gene disorders, inherited in an autosomal recessive manner ,prevalent among all ethnic groups and in almost every country around the world .
Thalassemias are genetic diseases characterized by a deficit in the production of hemoglobin, the protein contained in red blood cells that transports oxygen in the bloodstream. The disease is qualified as α-thalassemia or β-thalassemia depending on whether the anomaly results from a defect in the synthesis of the alpha (α) chains or beta (β) chains making up the protein.
It is the most prevalent chronic hemolytic anemia in Egypt (85.1%), the carrier rate of thalassemia in 1000 normal subjects was found to be 9-10.2% .
Once a child has been diagnosed as thalassemia, he has to take lifelong treatment , where cure is not attainable and treatment may be prolonged . It is a life-threatening and life-limiting condition that affects the patient clinically and psychologically, so Health-related Quality of Life (HRQOL) is likely to be an essential outcome for these patients.
The principles of treatment have been well defined which include (a) maintaining hemoglobin between 9 and 10 g/dl by repeated packed cell transfusion (b) regular chelation therapy to maintain serum ferritin around 1000 ng/dl (c) preventing the development of complications of the disease or secondary to therapy (d) ensuring normal growth and development . This conventional treatment is required for life-long. The main objective of thalassemia life-long treatment is to maintain good quality of life as close to near normal life. there are many Factor that can affect Quality of life in thalassemic children such as : Repeated visits to hospitals for regular blood transfusion, cost of chelation therapy, repeated laboratory tests for monitoring therapy and for early detection of any complications. also life-long costly therapy along with poor quality of life will have adverse impact on the family.
The assessment of HRQOL in children is essential for the provision of proper care, since it helps in identifying the impact of the disease and treatment on children . A better understanding of the factors associated with HRQOL among children with thalassemia could have a direct effect on the development of more suitable clinical, counselling and social support programs to enhance treatment outcomes.
Cognitive dysfunction was Reported either due to the disease or its treatment ,frequent school absences, frequent hospitalizations, and physical and social restrictions lead to cognitive dysfunction . This neurological involvement in thalassemic children is primarily silent, with subclinical manifestations that can only be detected by cognitive assessment tests.
Previous studies showed significantly different results between patients and controls regarding the intelligence quotient (IQ).
- Conditions
- Thalassemia
- Being tested
- Pediatric Quality of Life Inventory (questionnaire) and The Stanford-Binet Intelligence Scale fifth edition
- Medical subject headings
- Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Pediatric Quality of Life Inventory (questionnaire) and The Stanford-Binet Intelligence Scale fifth editionOther
Quality of Life Assessment By Pediatric Quality of life Inventory. Cognitive Function Assessment By The Stanford-Binet Intelligence Scale fifth edition.
| Study group | Role | What they receive |
|---|---|---|
| Children With Thalassemia (Cases) | Hematological Disease which are a heterogeneous group of single gene disorders, inherited in an autosomal recessive manner ( Thalassemia Syndrome ) Receives: Other: Pediatric Quality of Life Inventory (questionnaire) and The Stanford-Binet Intelligence Scale fifth edition | |
| Healthy Children ( Controls ) | Healthy Children free from any chronic illness Receives: Other: Pediatric Quality of Life Inventory (questionnaire) and The Stanford-Binet Intelligence Scale fifth edition |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Stanford-Binet Intelligence Scale fifth edition
Assessment of Cognitive Function Minimum Value is 40 Maximum value is 160 Higher scores mean a better outcome
Measured: 12 months
Pediatric Quality of Life Inventory
Assessment of Quality of Life Minimum value is 0 Maximum value is 100 Higher scores mean a better outcome
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Sep 2023
- Study start20 Oct 2023 (estimated)
- Primary completion20 Oct 2024 (estimated)
- Study completion20 Oct 2024 (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 ThalassemiaStudies of the same condition, open and under way.
- More trials run by Sohag 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-control
- Time perspective
- Prospective
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sohag University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Mena Gerges Shawky, Sohag University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06058260
- Sponsor's protocol ID
- Soh-Med-23-09-07MS
- Status last verified
- Oct 2023
- First submitted
- 13 Sep 2023
- Last updated
- 16 Oct 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Choudhry VP. Thalassemia Minor and Major: Current Management. Indian J Pediatr. 2017 Aug;84(8):607-611. doi: 10.1007/s12098-017-2325-1. Epub 2017 Apr 24.
PubMed 28435994
Study at a glance
From the registry record, last updated by the sponsor on 16 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Assessment of Cognitive Function and Quality of Life in Thalassemic Children at Sohag University Hospital
- ClinicalTrials.gov ID
- NCT06058260
- Acronym / sponsor ID
- Soh-Med-23-09-07MS
- Conditions
- Thalassemia
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Sohag UniversityUniversity, hospital or other
- Study start
- 20 Oct 2023 (estimated)
- Primary completion
- 20 Oct 2024 (estimated)
- Study completion
- 20 Oct 2024 (estimated)
- First posted
- 28 Sep 2023 (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.
Thalassemia syndromes are a heterogeneous group of single gene disorders, inherited in an autosomal recessive manner ,prevalent among all ethnic groups and in almost every country around the world .
Once a child has been diagnosed as thalassemia, he has to take lifelong treatment , where cure is not attainable and treatment may be prolonged . It is a life-threatening and life-limiting condition that affects the patient clinically and psychologically, so Health-related Quality of Life (HRQOL) is likely to be an essential outcome for these patients.
Quality of life in thalassemic children such as : Repeated visits to hospitals for regular blood transfusion, cost of chelation therapy, repeated laboratory tests for monitoring therapy and for early detection of any complications. also life-long costly therapy along with poor quality of life will have adverse impact on the family.
A better understanding of the factors associated with HRQOL among children with thalassemia could have a direct effect on the development of more suitable clinical, counselling and social support programs to enhance treatment outcomes.
Cognitive dysfunction was Reported either due to the disease or its treatment ,frequent school absences, frequent hospitalizations, and physical and social restrictions lead to cognitive dysfunction . This neurological involvement in thalassemic children is primarily silent, with subclinical manifestations that can only be detected by cognitive assessment tests.
Detailed description
Thalassemia syndromes are a heterogeneous group of single gene disorders, inherited in an autosomal recessive manner ,prevalent among all ethnic groups and in almost every country around the world .
Thalassemias are genetic diseases characterized by a deficit in the production of hemoglobin, the protein contained in red blood cells that transports oxygen in the bloodstream. The disease is qualified as α-thalassemia or β-thalassemia depending on whether the anomaly results from a defect in the synthesis of the alpha (α) chains or beta (β) chains making up the protein.
It is the most prevalent chronic hemolytic anemia in Egypt (85.1%), the carrier rate of thalassemia in 1000 normal subjects was found to be 9-10.2% .
Once a child has been diagnosed as thalassemia, he has to take lifelong treatment , where cure is not attainable and treatment may be prolonged . It is a life-threatening and life-limiting condition that affects the patient clinically and psychologically, so Health-related Quality of Life (HRQOL) is likely to be an essential outcome for these patients.
The principles of treatment have been well defined which include (a) maintaining hemoglobin between 9 and 10 g/dl by repeated packed cell transfusion (b) regular chelation therapy to maintain serum ferritin around 1000 ng/dl (c) preventing the development of complications of the disease or secondary to therapy (d) ensuring normal growth and development . This conventional treatment is required for life-long. The main objective of thalassemia life-long treatment is to maintain good quality of life as close to near normal life. there are many Factor that can affect Quality of life in thalassemic children such as : Repeated visits to hospitals for regular blood transfusion, cost of chelation therapy, repeated laboratory tests for monitoring therapy and for early detection of any complications. also life-long costly therapy along with poor quality of life will have adverse impact on the family.
The assessment of HRQOL in children is essential for the provision of proper care, since it helps in identifying the impact of the disease and treatment on children . A better understanding of the factors associated with HRQOL among children with thalassemia could have a direct effect on the development of more suitable clinical, counselling and social support programs to enhance treatment outcomes.
Cognitive dysfunction was Reported either due to the disease or its treatment ,frequent school absences, frequent hospitalizations, and physical and social restrictions lead to cognitive dysfunction . This neurological involvement in thalassemic children is primarily silent, with subclinical manifestations that can only be detected by cognitive assessment tests.
Previous studies showed significantly different results between patients and controls regarding the intelligence quotient (IQ).
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Children With Thalassemia (Cases) | – | Other: Pediatric Quality of Life Inventory (questionnaire) and The Stanford-Binet Intelligence Scale fifth editionHematological Disease which are a heterogeneous group of single gene disorders, inherited in an autosomal recessive manner ( Thalassemia Syndrome ) |
| Healthy Children ( Controls ) | – | Other: Pediatric Quality of Life Inventory (questionnaire) and The Stanford-Binet Intelligence Scale fifth editionHealthy Children free from any chronic illness |
Who may qualify
- Ages
- 4 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- All the diagnosed thalassemic children age between 4 and 18 years.
Key inclusion criteria (1 listed)
- All the diagnosed thalassemic children age between 4 and 18 years.
Key exclusion criteria (2 listed)
- Any other hematological disease.
- Age less than 4 years and more than 18 years.
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 |
|---|---|---|
| Pediatric Quality of Life Inventory (questionnaire) and The Stanford-Binet Intelligence Scale fifth editionQuality of Life Assessment By Pediatric Quality of life Inventory. Cognitive Function Assessment By The Stanford-Binet Intelligence Scale fifth edition. | Other | Children With Thalassemia (Cases), Healthy Children ( Controls ) |
Primary outcome
The Stanford-Binet Intelligence Scale fifth edition
Time frame: 12 months
Assessment of Cognitive Function Minimum Value is 40 Maximum value is 160 Higher scores mean a better outcome
Pediatric Quality of Life Inventory
Time frame: 12 months
Assessment of Quality of Life Minimum value is 0 Maximum value is 100 Higher scores mean a better outcome
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Oct 2023.
- Background: PubMed 28435994Choudhry VP. Thalassemia Minor and Major: Current Management. Indian J Pediatr. 2017 Aug;84(8):607-611. doi: 10.1007/s12098-017-2325-1. Epub 2017 Apr 24.
- Individual participant dataSharing planned: Undecided
Related trials for Thalassemia
Active, not recruiting
Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.
Transfusion-dependent Beta-Thalassemia
RecruitingObservational study
Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
Transfusion-dependent Beta-Thalassemia, Gene Therapy
Not yet recruiting
Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector
Beta-Thalassemia
RecruitingPhase 1/2
β-Thalassemia Treatment With KL003 Cell Injection
Transfusion-dependent Beta-Thalassemia
Active, not recruitingPhase 3
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
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.
Alzahraa A Ahmed, professor contact
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 Thalassemia, run by Sohag University 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 1 inclusion and 2 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 16 Oct 2023. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Mena G Shawky, resident (01289496538, Menagergeos@med.sohag.edu.eg), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The Stanford-Binet Intelligence Scale fifth edition (time frame: 12 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?
On the ClinicalTrials.gov record for NCT06058260, 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 16 Oct 2023.