Thalassemia MajorNCT03161899
Evaluation of Nutritional Status in Thalassemia Major Patients in Assiut Children Hospital
An observational study of Thalassemia Major, run by Assiut University at 1 site in Egypt.
Trial summary
Thalassemia is a blood disorder passed down through families in which the body makes an abnormal form of hemoglobin. There are 2 main types of thalassemia; Alpha \& Beta thalassemia. Alpha thalassemia: occurs when a gene or genes related to the alpha globin protein are missing or mutated. Read more about this study
- Who is it for?
- Ages 6 to 14 years
- Conditions the study is for
- Thalassemia Major
- How many people
- 246 participants
- Where
- 1 siteEgypt
- Study dates
- 15 Dec 2018to 20 Jul 2020
- 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 6 to 14 years
- All sexes
- No healthy volunteers
- Child
Study population: all beta thalassemic patients attending the children Hematology Clinic, Children Hospital, Assiut University, during the period of 1st of June 2017 till 31 th of may 2018.
Who may be able to join
- patients diagnosed to have thalassemia
Who cannot join
- patient with any underlaying systemic diseases other than B thalassemia major.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Evaluation of Nutritional Status in Patients With Thalassemia Major in Assiut University Children Hospital
Thalassemia is a blood disorder passed down through families in which the body makes an abnormal form of hemoglobin. There are 2 main types of thalassemia; Alpha \& Beta thalassemia. Alpha thalassemia: occurs when a gene or genes related to the alpha globin protein are missing or mutated.
Beta-thalassemia syndromes are a group of hereditary blood disorders characterized by reduced or absent beta globin chain synthesis. Beta-thalassemias can be classified into:
Read more
Silent carrier: completely asymptomatic with normal hematological parameters. Beta-thalassaemia minor (beta-thalassaemia trait): usually asymptomatic; diagnosis is made during a work-up for mild anemia.
Beta-thalassaemia intermedia: usually a similar presentation to beta-thalassaemia major; symptoms are usually less pronounced and the course is usually more insidious.
Beta-thalassaemia major : In which there is complete absence of hemoglobin A
Read the detailed description
In Egypt beta thalassemia-major is the most common type with carrier rate of 5.3 to ≥9%and 1000 new cases born with beta-thalassemia major per 1.5 million live births per year.
Children born with thalassemia major are normal at birth, but develop severe hemolytic anemia during the first year of life. Symptoms are those of anemia (lethargy, poor feeding, pallor…etc.) failure to thrive and organomegaly. Later on they develop signs of extra medullary hematopoiesis .
Optimal nutritional status is important for growth, immune function, bone health and pubertal development . Various reports suggest the incidence of poor growth ranges from 25% to 75% depending on thalassemia syndrome and severity of disease. This marked growth deficits raise a red flag for any pediatrician to evaluate nutritional status of thalassemic patients and detect possible nutritional deficiencies and associated factors.
Etiology of delayed growth and malnutrition reported in thalassemic patient is multifactorial including :
- Nutritional deprivation with or without feeding difficulties arising from fatigue and breathlessness
- Increased energy expenditure secondary to hyper metabolism with or without heart failure
- Gastrointestinal hypoxia which consequently produces anorexia and malabsorption
- Reduction of biosynthetic activity of liver.
- Disturbance of the endocrine function
- Impaired synthetic hepatic function secondary to hemosiderosis and hepatitis.
The main aim of this study is to assess the nutritional status of thalassemic patient attending Assiut university Children hospital and to determine the factors involved.
- Conditions
- Thalassemia Major
- Keywords
- thalassemia - nutrition -growth
- Medical subject headings
- beta-Thalassemia
What is being measured
The primary outcome is the main question the study is designed to answer.
percentage of malnutrition
prevalence of malnutrition among thalassemic children attending assiut university children hospital
Measured: 12 month
Secondary outcomes (2)
aetiology poor growth
determination of the most common causes responsible for poor growth seen in thalassemic patients
Measured: 12 month
decrease morbidity
decrease morbidity by providing possible strategies for early prevention of the problem of poor growth occurring in patients with thalassemia major
Measured: 18 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 May 2017
- Study start15 Dec 2018
- Primary completion24 May 2020
- Study completion20 Jul 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)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
- Case only
- Time perspective
- Cross-sectional
- Enrolment
- 246 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assiut University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, ARAbdelmonem, Assiut University
- Investigators
- asmaa refaat, MBBCH, Principal investigator, Assuit , faculty of medicine, egypt
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03161899
- Sponsor's protocol ID
- ENST
- Status last verified
- Jul 2020
- First submitted
- 19 May 2017
- Last updated
- 30 Jul 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Soliman AT, El-Matary W, Fattah MM, Nasr IS, El Alaily RK, Thabet MA. The effect of high-calorie diet on nutritional parameters of children with beta-thalassaemia major. Clin Nutr. 2004 Oct;23(5):1153-8. doi: 10.1016/j.clnu.2004.03.001.
PubMed 15380908Fung EB. Nutritional deficiencies in patients with thalassemia. Ann N Y Acad Sci. 2010 Aug;1202:188-96. doi: 10.1111/j.1749-6632.2010.05578.x.
PubMed 20712792Galanello R, Origa R. Beta-thalassemia. Orphanet J Rare Dis. 2010 May 21;5:11. doi: 10.1186/1750-1172-5-11.
PubMed 20492708
Study at a glance
From the registry record, last updated by the sponsor on 30 Jul 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of Nutritional Status in Patients With Thalassemia Major in Assiut University Children Hospital
- ClinicalTrials.gov ID
- NCT03161899
- Acronym / sponsor ID
- ENST
- Conditions
- Thalassemia Major
- Keywords
- thalassemia - nutrition -growth
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 246 participants (actual)
- Lead sponsor
- Assiut UniversityUniversity, hospital or other
- Study start
- 15 Dec 2018 (actual)
- Primary completion
- 24 May 2020 (actual)
- Study completion
- 20 Jul 2020 (actual)
- First posted
- 22 May 2017 (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 is a blood disorder passed down through families in which the body makes an abnormal form of hemoglobin. There are 2 main types of thalassemia; Alpha \& Beta thalassemia. Alpha thalassemia: occurs when a gene or genes related to the alpha globin protein are missing or mutated.
Beta-thalassemia syndromes are a group of hereditary blood disorders characterized by reduced or absent beta globin chain synthesis. Beta-thalassemias can be classified into:
Silent carrier: completely asymptomatic with normal hematological parameters. Beta-thalassaemia minor (beta-thalassaemia trait): usually asymptomatic; diagnosis is made during a work-up for mild anemia.
Beta-thalassaemia intermedia: usually a similar presentation to beta-thalassaemia major; symptoms are usually less pronounced and the course is usually more insidious.
Beta-thalassaemia major : In which there is complete absence of hemoglobin A
Detailed description
In Egypt beta thalassemia-major is the most common type with carrier rate of 5.3 to ≥9%and 1000 new cases born with beta-thalassemia major per 1.5 million live births per year.
Children born with thalassemia major are normal at birth, but develop severe hemolytic anemia during the first year of life. Symptoms are those of anemia (lethargy, poor feeding, pallor…etc.) failure to thrive and organomegaly. Later on they develop signs of extra medullary hematopoiesis .
Optimal nutritional status is important for growth, immune function, bone health and pubertal development . Various reports suggest the incidence of poor growth ranges from 25% to 75% depending on thalassemia syndrome and severity of disease. This marked growth deficits raise a red flag for any pediatrician to evaluate nutritional status of thalassemic patients and detect possible nutritional deficiencies and associated factors.
Etiology of delayed growth and malnutrition reported in thalassemic patient is multifactorial including :
- Nutritional deprivation with or without feeding difficulties arising from fatigue and breathlessness
- Increased energy expenditure secondary to hyper metabolism with or without heart failure
- Gastrointestinal hypoxia which consequently produces anorexia and malabsorption
- Reduction of biosynthetic activity of liver.
- Disturbance of the endocrine function
- Impaired synthetic hepatic function secondary to hemosiderosis and hepatitis.
The main aim of this study is to assess the nutritional status of thalassemic patient attending Assiut university Children hospital and to determine the factors involved.
Study design
- Observational model
- Case only
- Time perspective
- Cross-sectional
Who may qualify
- Ages
- 6 to 14 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- all beta thalassemic patients attending the children Hematology Clinic, Children Hospital, Assiut University, during the period of 1st of June 2017 till 31 th of may 2018.
Key inclusion criteria (1 listed)
- patients diagnosed to have thalassemia
Key exclusion criteria (1 listed)
- patient with any underlaying systemic diseases other than B thalassemia major.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
percentage of malnutrition
Time frame: 12 month
prevalence of malnutrition among thalassemic children attending assiut university children hospital
2 secondary outcomes
aetiology poor growth
Time frame: 12 month
decrease morbidity
Time frame: 18 month
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Jul 2020.
- Background: PubMed 15380908Soliman AT, El-Matary W, Fattah MM, Nasr IS, El Alaily RK, Thabet MA. The effect of high-calorie diet on nutritional parameters of children with beta-thalassaemia major. Clin Nutr. 2004 Oct;23(5):1153-8. doi: 10.1016/j.clnu.2004.03.001.
- Background: PubMed 20712792Fung EB. Nutritional deficiencies in patients with thalassemia. Ann N Y Acad Sci. 2010 Aug;1202:188-96. doi: 10.1111/j.1749-6632.2010.05578.x.
- Background: PubMed 20492708Galanello R, Origa R. Beta-thalassemia. Orphanet J Rare Dis. 2010 May 21;5:11. doi: 10.1186/1750-1172-5-11.
- 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.
asmaa refaat, MBBCH principal investigator
Assuit , faculty of medicine, egypt
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 Major, run by Assiut 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 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: finished, as of the sponsor's last update on 30 Jul 2020. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
percentage of malnutrition (time frame: 12 month). 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 NCT03161899, which also lists 3 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 30 Jul 2020.