Thalassemia MajorNCT05303506
Study on the Effect of Different Risk Factors on the Growth Parameters of Thalassemic Patients in Assiut (AUCH)
An observational study of Thalassemia Major, run by Assiut University.
Trial summary
To assess the effect of different risk factors on the growth parameters of thalassemic patients in Assiut University children Hospital (AUCH) In order to help in decreasing the morbidity and mortality resulting from iron overload and improving the quality of life for thalassemic patient Read more about this study
- Who is it for?
- Ages 1 year to 16 years
- Conditions the study is for
- Thalassemia Major
- What is being tested?
- serum ferritin and AST
- How many people
- 50 participants
- Where
- No sites listed yet
- Study dates
- 2 Jul 2022to 2 Mar 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 1 year to 16 years
- All sexes
- Child
Study population: All children attending haematology unit in Assiut University Children Hospital with thalassemia treated by iron chelating agents and aged from one year to 16 years old.
Who may be able to join
- All children attending haematology unit in Assiut University Children Hospital with thalassemia treated by iron chelating agents and aged from one year to 16 years old.
Who cannot join
- Children not treated by chelating agents. Children with stem cell transplantation . Children with thalassemia minor or intermedia.
Trial locations and contacts
No sites are listed yet.
About this study
Official title: Prospective Study on the Effect of Different Risk Factors on the Growth Parameters of Thalassemic Patients in Assiut University Children Hospital (AUCH)
To assess the effect of different risk factors on the growth parameters of thalassemic patients in Assiut University children Hospital (AUCH) In order to help in decreasing the morbidity and mortality resulting from iron overload and improving the quality of life for thalassemic patient
Read the detailed description
Thalassemia major is a heterogeneous disease presenting during infancy or early childhood. Although thalassemia is preventable by premarital counseling and prenatal testing, a large number of children are born with thalassemia, and curative treatment in the form of bone marrow or stem cell transplantation is not possible for the majority of these patients. Such patients need regular transfusions of packed red blood cells (PRBCs) Excess iron can potentially penetrate cells in the liver, heart, endocrine glands and other organs, Iron overload in the heart and liver is known to be a leading cause of morbidity and mortality among patients with transfusion-dependent b-thalassemia major Physical growth is affected in a large number of the patients with transfusion-dependent thalassemia. A study of patients aged 10-27 years with thalassemia major found short stature in 70% of the males and in 73% of the females, thalassemic patients are short, have low rate of growth and BMI and have either delayed or absent pubertal spurt, which is related to low hemoglobin and high ferritin levels and sub-optimal iron chelation therapy. (Najafipour, et al 2008 ).
Therefore, iron-chelating agents, such as deferoxamine, have been used since 1970 and has shown to be effective in chelating iron from the heart and liver, with preservation of heart function and reversal of hepatic fibrosis, respectively Recent advances in the medical management of regular blood transfusion and chelation therapy have allowed most of these patients to have improved survival well into adult life and improved quality of life in patients with severe b-thalassemia promote normal growth, allow normal physical activities, minimize transfusional iron accumulation, adequately suppress bone marrow activity and reduce cardiac overload due to chronic anemia .
Hemoglobin levels maintained at 9-10 g/dl are thought of as optimal , A higher target pre-transfusion hemoglobin level of 11-12 g/dl may be appropriate for patients with heart disease or other medical conditions There is a clear link between the overall iron burden and global toxicity; in patients with thalassemia the liver iron concentration correlates with the total body iron load Serum ferritin and liver iron concentration are widely used to detect iron overload, and the latter can be measured by magnetic resonance imaging (MRI), or liver biopsy, Evidence indicates that serum ferritin is not an accurate measure iron overload and that direct measurement of liver iron concentration via liver biopsy or MRI is more precise
- Conditions
- Thalassemia Major
- Being tested
- serum ferritin and AST
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
serum ferritin and ASTDiagnostic test
observiational study
What is being measured
The primary outcome is the main question the study is designed to answer.
Measuring the Effect of different risk factors on the Growth Parameters of Thalassemic Patients in Assiut University Children Hospital (AUCH) and how these risk factors affect the quality of life by measuring anthropocentric measures
Data of the patients will be collected from thalassemic patients in form of
- Personal history (Name, Age ,Sex)
- Therapeutic history (Age of diagnosis , Age of 1st chelator ,Chelator type , Chelator dose , Compliance to treatment )
- Growth parameters (Weight , Height,and mid arm circumference))
Measured: 2 years
Decreasing the morbidity and mortality and improving the quality of life for thalassemic patient
At the end of the study there will be recommendations to help in decreasing the morbidity and mortality and improving the quality of life for thalassemic patient
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 posted31 Mar 2022
- Study start2 Jul 2022 (estimated)
- Primary completion2 Dec 2023 (estimated)
- Study completion2 Mar 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (9)Papers the sponsor has linked to this study.
- Other trials for Thalassemia MajorStudies 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
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assiut University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Mohamed Elmostafa Mohamed Hemida, Assiut University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05303506
- Sponsor's protocol ID
- thalassemia in AUCH
- Status last verified
- Mar 2022
- First submitted
- 21 Nov 2021
- Last updated
- 31 Mar 2022
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
. Pemde et al, publisher and licensee Dove Medical Press Ltd. This is an Open Access article which permits unrestricted noncommercial use, provided the original work is properly cited. Pediatric Health, Medicine and Therapeutics 2011:2 13-19
Hoffbrand AV, Taher A, Cappellini MD. How I treat transfusional iron overload. Blood. 2012 Nov 1;120(18):3657-69. doi: 10.1182/blood-2012-05-370098. Epub 2012 Aug 23.
PubMed 22919029Rachmilewitz EA, Giardina PJ. How I treat thalassemia. Blood. 2011 Sep 29;118(13):3479-88. doi: 10.1182/blood-2010-08-300335. Epub 2011 Aug 2.
PubMed 21813448Najafipour F, Aliasgarzadeh A, Aghamohamadzadeh N, Bahrami A, Mobasri M, Niafar M, Khoshbaten M. A cross-sectional study of metabolic and endocrine complications in beta-thalassemia major. Ann Saudi Med. 2008 Sep-Oct;28(5):361-6. doi: 10.5144/0256-4947.2008.361.
PubMed 18779644Wood JC, Kang BP, Thompson A, Giardina P, Harmatz P, Glynos T, Paley C, Coates TD. The effect of deferasirox on cardiac iron in thalassemia major: impact of total body iron stores. Blood. 2010 Jul 29;116(4):537-43. doi: 10.1182/blood-2009-11-250308. Epub 2010 Apr 26.
PubMed 20421452Deugnier Y, Turlin B, Ropert M, Cappellini MD, Porter JB, Giannone V, Zhang Y, Griffel L, Brissot P. Improvement in liver pathology of patients with beta-thalassemia treated with deferasirox for at least 3 years. Gastroenterology. 2011 Oct;141(4):1202-11, 1211.e1-3. doi: 10.1053/j.gastro.2011.06.065. Epub 2011 Jul 7.
PubMed 21741344Cabantchik ZI, Breuer W, Zanninelli G, Cianciulli P. LPI-labile plasma iron in iron overload. Best Pract Res Clin Haematol. 2005 Jun;18(2):277-87. doi: 10.1016/j.beha.2004.10.003.
PubMed 15737890Borgna-Pignatti C. Surviving with thalassemia major: the Italian experience. Pediatr Hematol Oncol. 2007 Jan-Feb;24(1):75-8. doi: 10.1080/08880010601001461. No abstract available.
PubMed 17130117Wood JC, Enriquez C, Ghugre N, Tyzka JM, Carson S, Nelson MD, Coates TD. MRI R2 and R2* mapping accurately estimates hepatic iron concentration in transfusion-dependent thalassemia and sickle cell disease patients. Blood. 2005 Aug 15;106(4):1460-5. doi: 10.1182/blood-2004-10-3982. Epub 2005 Apr 28.
PubMed 15860670
Study at a glance
From the registry record, last updated by the sponsor on 31 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Study on the Effect of Different Risk Factors on the Growth Parameters of Thalassemic Patients in Assiut University Children Hospital (AUCH)
- ClinicalTrials.gov ID
- NCT05303506
- Acronym / sponsor ID
- thalassemia in AUCH
- Conditions
- Thalassemia Major
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Assiut UniversityUniversity, hospital or other
- Study start
- 2 Jul 2022 (estimated)
- Primary completion
- 2 Dec 2023 (estimated)
- Study completion
- 2 Mar 2024 (estimated)
- First posted
- 31 Mar 2022 (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.
To assess the effect of different risk factors on the growth parameters of thalassemic patients in Assiut University children Hospital (AUCH) In order to help in decreasing the morbidity and mortality resulting from iron overload and improving the quality of life for thalassemic patient
Detailed description
Thalassemia major is a heterogeneous disease presenting during infancy or early childhood. Although thalassemia is preventable by premarital counseling and prenatal testing, a large number of children are born with thalassemia, and curative treatment in the form of bone marrow or stem cell transplantation is not possible for the majority of these patients. Such patients need regular transfusions of packed red blood cells (PRBCs) Excess iron can potentially penetrate cells in the liver, heart, endocrine glands and other organs, Iron overload in the heart and liver is known to be a leading cause of morbidity and mortality among patients with transfusion-dependent b-thalassemia major Physical growth is affected in a large number of the patients with transfusion-dependent thalassemia. A study of patients aged 10-27 years with thalassemia major found short stature in 70% of the males and in 73% of the females, thalassemic patients are short, have low rate of growth and BMI and have either delayed or absent pubertal spurt, which is related to low hemoglobin and high ferritin levels and sub-optimal iron chelation therapy. (Najafipour, et al 2008 ).
Therefore, iron-chelating agents, such as deferoxamine, have been used since 1970 and has shown to be effective in chelating iron from the heart and liver, with preservation of heart function and reversal of hepatic fibrosis, respectively Recent advances in the medical management of regular blood transfusion and chelation therapy have allowed most of these patients to have improved survival well into adult life and improved quality of life in patients with severe b-thalassemia promote normal growth, allow normal physical activities, minimize transfusional iron accumulation, adequately suppress bone marrow activity and reduce cardiac overload due to chronic anemia .
Hemoglobin levels maintained at 9-10 g/dl are thought of as optimal , A higher target pre-transfusion hemoglobin level of 11-12 g/dl may be appropriate for patients with heart disease or other medical conditions There is a clear link between the overall iron burden and global toxicity; in patients with thalassemia the liver iron concentration correlates with the total body iron load Serum ferritin and liver iron concentration are widely used to detect iron overload, and the latter can be measured by magnetic resonance imaging (MRI), or liver biopsy, Evidence indicates that serum ferritin is not an accurate measure iron overload and that direct measurement of liver iron concentration via liver biopsy or MRI is more precise
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
Who may qualify
- Ages
- 1 year to 16 years
- Sex
- All
- Age groups
- Child
- Study population
- All children attending haematology unit in Assiut University Children Hospital with thalassemia treated by iron chelating agents and aged from one year to 16 years old.
Key inclusion criteria (1 listed)
- All children attending haematology unit in Assiut University Children Hospital with thalassemia treated by iron chelating agents and aged from one year to 16 years old.
Key exclusion criteria (1 listed)
- Children not treated by chelating agents. Children with stem cell transplantation . Children with thalassemia minor or intermedia.
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 |
|---|---|---|
| serum ferritin and ASTobserviational study | Diagnostic test | – |
Primary outcome
Measuring the Effect of different risk factors on the Growth Parameters of Thalassemic Patients in Assiut University Children Hospital (AUCH) and how these risk factors affect the quality of life by measuring anthropocentric measures
Time frame: 2 years
Data of the patients will be collected from thalassemic patients in form of 1. Personal history (Name, Age ,Sex) 2. Therapeutic history (Age of diagnosis , Age of 1st chelator ,Chelator type , Chelator dose , Compliance to treatment ) 3. Growth parameters (Weight , Height,and mid arm circumference))
Decreasing the morbidity and mortality and improving the quality of life for thalassemic patient
Time frame: 6 months
At the end of the study there will be recommendations to help in decreasing the morbidity and mortality and improving the quality of life for thalassemic patient
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Mar 2022.
- Background. Pemde et al, publisher and licensee Dove Medical Press Ltd. This is an Open Access article which permits unrestricted noncommercial use, provided the original work is properly cited. Pediatric Health, Medicine and Therapeutics 2011:2 13-19
- Background: PubMed 22919029Hoffbrand AV, Taher A, Cappellini MD. How I treat transfusional iron overload. Blood. 2012 Nov 1;120(18):3657-69. doi: 10.1182/blood-2012-05-370098. Epub 2012 Aug 23.
- Background: PubMed 21813448Rachmilewitz EA, Giardina PJ. How I treat thalassemia. Blood. 2011 Sep 29;118(13):3479-88. doi: 10.1182/blood-2010-08-300335. Epub 2011 Aug 2.
- Background: PubMed 18779644Najafipour F, Aliasgarzadeh A, Aghamohamadzadeh N, Bahrami A, Mobasri M, Niafar M, Khoshbaten M. A cross-sectional study of metabolic and endocrine complications in beta-thalassemia major. Ann Saudi Med. 2008 Sep-Oct;28(5):361-6. doi: 10.5144/0256-4947.2008.361.
- Background: PubMed 20421452Wood JC, Kang BP, Thompson A, Giardina P, Harmatz P, Glynos T, Paley C, Coates TD. The effect of deferasirox on cardiac iron in thalassemia major: impact of total body iron stores. Blood. 2010 Jul 29;116(4):537-43. doi: 10.1182/blood-2009-11-250308. Epub 2010 Apr 26.
- Background: PubMed 21741344Deugnier Y, Turlin B, Ropert M, Cappellini MD, Porter JB, Giannone V, Zhang Y, Griffel L, Brissot P. Improvement in liver pathology of patients with beta-thalassemia treated with deferasirox for at least 3 years. Gastroenterology. 2011 Oct;141(4):1202-11, 1211.e1-3. doi: 10.1053/j.gastro.2011.06.065. Epub 2011 Jul 7.
- Background: PubMed 15737890Cabantchik ZI, Breuer W, Zanninelli G, Cianciulli P. LPI-labile plasma iron in iron overload. Best Pract Res Clin Haematol. 2005 Jun;18(2):277-87. doi: 10.1016/j.beha.2004.10.003.
- Background: PubMed 17130117Borgna-Pignatti C. Surviving with thalassemia major: the Italian experience. Pediatr Hematol Oncol. 2007 Jan-Feb;24(1):75-8. doi: 10.1080/08880010601001461. No abstract available.
- Background: PubMed 15860670Wood JC, Enriquez C, Ghugre N, Tyzka JM, Carson S, Nelson MD, Coates TD. MRI R2 and R2* mapping accurately estimates hepatic iron concentration in transfusion-dependent thalassemia and sickle cell disease patients. Blood. 2005 Aug 15;106(4):1460-5. doi: 10.1182/blood-2004-10-3982. Epub 2005 Apr 28.
- 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.
Questions about this trial
What is this clinical trial studying?
An observational study of Thalassemia Major, 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: status not confirmed, as of the sponsor's last update on 31 Mar 2022. 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 Mohamed Elmostafa Mohamed Hemida, master degree (+201097602949, mhemida92@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Measuring the Effect of different risk factors on the Growth Parameters of Thalassemic Patients in Assiut University Children Hospital (AUCH) and how these risk factors affect the quality of life by measuring anthropocentric measures (time frame: 2 years). 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 NCT05303506, which also lists 9 linked publications.
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 31 Mar 2022.