Evaluate Bone Denisty Changes in Children With Beta Thalassemia MajorNCT06069089
Bone Denisty Change in Children With Beta Thalassemia Major
An observational study of Evaluate Bone Denisty Changes in Children With Beta Thalassemia Major, run by Marwa Hassan Abdelhamed Hassan.
Trial summary
Bone denisty changes in children with beta thalassemia major Read more about this study
- Who is it for?
- Ages 10 to 18 years
- Conditions the study is for
- Evaluate Bone Denisty Changes in Children With Beta Thalassemia Major
- What is being tested?
- Dexa scan
- Type of treatment
- Surgery or procedure
- How many people
- 42 participants
- Where
- No sites listed yet
- Study dates
- 15 Dec 2023to 31 Dec 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 10 to 18 years
- All sexes
- Child, Adult
Study population: Children with beta thalassemia major with transfusion dependent
Who may be able to join
- Patient diagnosed as B thalassemia major of both sexes, age range from 10-18 year, who are poorly controlled on frequant blood transfusion. The patient who doesn't have Hb level from 9-10 g/dl, in almost always less than 9 g/dl.
Who cannot join
- Known metabolic bone disease. Less than 10 year or more than 18 year. Bone disease Other than hemolytic anemia.
About this study
Official title: Assiut University, Pediatrician Department
Bone denisty changes in children with beta thalassemia major
Read the detailed description
Beta Thalassemia major (TM) is a hereditary disease caused by defective Beta globin chain synthesis, resulting in abnormal as well as a decreased quantity of globin chains, ineffective erythropoiesis, haemolysis and increased red blood cell turnover (Cooley, etal, 1927). described the first patient with anemia, splenomegaly, cranial \& facial bone enlargement. Pathophysiology of bone denisity changes in beta thalassemia major Several studies had been previously evaluated; shown that multiple factors may act in concert to produce bone disease in beta thalassemia major (TM) including bone marrow expansion (Shamshirsaz, etal, 2003). hypogonadism (Anapliotou,Saka\&Jensen,1998), defective growth hormone-insulin-like growth factor-1 (GH-IGF-1) axis (Soliman,etal,1998), altered pattern of cytokines (Morabito,etal,2007), iron deposit in bone ((Bordat,etal,1993),deferoxamine bone toxicity (Chan ,etal, 2002),and vitamin D deficiency (Dandona, etal, 1987). Some of these pathogenic factors, directly and/or indirectly, affect osteoblastic population, leading to depressed bone formation, while others often increase osteoclastic bone resorption.
Complications of transfusion dependent poorly controlled beta thalassemia major are;(1)-Osteoprosis; Iron overload impairs osteoid maturation and inhibits local mineralization to form focal osteomalacia. In addition, integration of iron in calcium hydroxyapatite affects the growth of crystals, which causes mineralization failure (Chan, etal, 2002), defective growth hormone-insulin-like growth factor-1 (GH-IGF-1) axis (Soliman, etal, 1998),altered pattern of cytokines (Morabito,etal, 2007), iron deposit in bone (Bordat, etal, 1993), deferoxamine bone toxicity (Chan, etal,2002). and vitamin D deficiency (Dandona, etal, 1987). (2)-Fractures; The introduction of red blood cell transfusion and concomitant iron chelation therapy has led to improved bone health through various mechanisms. It leads to a reduction in medullary expansion and cortical bone thinning, the reduced incidence of hypogonadism, and a reduction in other endocrine complications such as hypoparathyroidism and metabolic disorders that predispose to low bone density and fractures( Multicentre study, italian working group 1995). Z-score of bone density will be calculated. Z score is the preferred parameter in children. which is calculated as the number of standard deviations above or below the mean for the patient's age, sex,
- Being tested
- Dexa scan
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Dexa scanRadiation
No intervention
| Study group | Role | What they receive |
|---|---|---|
| Controlled beta thalassemia major | Controlled beta thalassemia major Hb>9g/dl , serum ferritin <500ng/ml Receives: Radiation: Dexa scan | |
| Uncontrolled beta thalassemia major | Uncontrolled beta thalassemia major Hb<9g/dl , serum ferritin >500ng/ml Receives: Radiation: Dexa scan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluate bone denisty in children with beta thalassemia major
To evaluate bone denisty in transfusion dependent beta thalassemia major \& its relation to serum minerals \&vit D To evaluate bone denisity in transfusion dependent beta thalassemia major \& its relation to serum minerals \&vit D To evaluate bone denisity in transfusion dependent beta thalassemia major \&its relation to serrum minerals \&vitD
Measured: One year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Oct 2023
- Study start15 Dec 2023 (estimated)
- Primary completion15 Dec 2024 (estimated)
- Study completion31 Dec 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 Evaluate Bone Denisty Changes in Children With Beta Thalassemia MajorStudies 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
- Other
- Time perspective
- Cross-sectional
- Enrolment
- 42 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Marwa Hassan Abdelhamed Hassan
- Sponsor type
- University, hospital or other
- Collaborators
- Assiut University
- Responsible party
- Sponsor-investigator, Marwa Hassan Abdelhamed Hassan, Assiut University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06069089
- Sponsor's protocol ID
- Thalassemia major in children
- Status last verified
- Sep 2023
- First submitted
- 29 Sep 2023
- Last updated
- 5 Oct 2023
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Gaudio A, Morabito N, Catalano A, Rapisarda R, Xourafa A, Lasco A. Pathogenesis of Thalassemia Major-associated Osteoporosis: A Review with Insights from Clinical Experience. J Clin Res Pediatr Endocrinol. 2019 May 28;11(2):110-117. doi: 10.4274/jcrpe.galenos.2018.2018.0074. Epub 2018 Jul 11.
PubMed 29991466
Study at a glance
From the registry record, last updated by the sponsor on 5 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Assiut University, Pediatrician Department
- ClinicalTrials.gov ID
- NCT06069089
- Acronym / sponsor ID
- Thalassemia major in children
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 42 participants (estimated)
- Lead sponsor
- Marwa Hassan Abdelhamed HassanUniversity, hospital or other
- Collaborators
- Assiut University
- Study start
- 15 Dec 2023 (estimated)
- Primary completion
- 15 Dec 2024 (estimated)
- Study completion
- 31 Dec 2024 (estimated)
- First posted
- 5 Oct 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.
Bone denisty changes in children with beta thalassemia major
Detailed description
Beta Thalassemia major (TM) is a hereditary disease caused by defective Beta globin chain synthesis, resulting in abnormal as well as a decreased quantity of globin chains, ineffective erythropoiesis, haemolysis and increased red blood cell turnover (Cooley, etal, 1927). described the first patient with anemia, splenomegaly, cranial \& facial bone enlargement. Pathophysiology of bone denisity changes in beta thalassemia major Several studies had been previously evaluated; shown that multiple factors may act in concert to produce bone disease in beta thalassemia major (TM) including bone marrow expansion (Shamshirsaz, etal, 2003). hypogonadism (Anapliotou,Saka\&Jensen,1998), defective growth hormone-insulin-like growth factor-1 (GH-IGF-1) axis (Soliman,etal,1998), altered pattern of cytokines (Morabito,etal,2007), iron deposit in bone ((Bordat,etal,1993),deferoxamine bone toxicity (Chan ,etal, 2002),and vitamin D deficiency (Dandona, etal, 1987). Some of these pathogenic factors, directly and/or indirectly, affect osteoblastic population, leading to depressed bone formation, while others often increase osteoclastic bone resorption.
Complications of transfusion dependent poorly controlled beta thalassemia major are;(1)-Osteoprosis; Iron overload impairs osteoid maturation and inhibits local mineralization to form focal osteomalacia. In addition, integration of iron in calcium hydroxyapatite affects the growth of crystals, which causes mineralization failure (Chan, etal, 2002), defective growth hormone-insulin-like growth factor-1 (GH-IGF-1) axis (Soliman, etal, 1998),altered pattern of cytokines (Morabito,etal, 2007), iron deposit in bone (Bordat, etal, 1993), deferoxamine bone toxicity (Chan, etal,2002). and vitamin D deficiency (Dandona, etal, 1987). (2)-Fractures; The introduction of red blood cell transfusion and concomitant iron chelation therapy has led to improved bone health through various mechanisms. It leads to a reduction in medullary expansion and cortical bone thinning, the reduced incidence of hypogonadism, and a reduction in other endocrine complications such as hypoparathyroidism and metabolic disorders that predispose to low bone density and fractures( Multicentre study, italian working group 1995). Z-score of bone density will be calculated. Z score is the preferred parameter in children. which is calculated as the number of standard deviations above or below the mean for the patient's age, sex,
Study design
- Observational model
- Other
- Time perspective
- Cross-sectional
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Controlled beta thalassemia major | – | Radiation: Dexa scanControlled beta thalassemia major Hb>9g/dl , serum ferritin <500ng/ml |
| Uncontrolled beta thalassemia major | – | Radiation: Dexa scanUncontrolled beta thalassemia major Hb<9g/dl , serum ferritin >500ng/ml |
Who may qualify
- Ages
- 10 to 18 years
- Sex
- All
- Age groups
- Child, Adult
- Study population
- Children with beta thalassemia major with transfusion dependent
Key inclusion criteria (1 listed)
- Patient diagnosed as B thalassemia major of both sexes, age range from 10-18 year, who are poorly controlled on frequant blood transfusion. The patient who doesn't have Hb level from 9-10 g/dl, in almost always less than 9 g/dl.
Key exclusion criteria (1 listed)
- Known metabolic bone disease. Less than 10 year or more than 18 year. Bone disease Other than hemolytic anemia.
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 |
|---|---|---|
| Dexa scanNo intervention | Radiation | Controlled beta thalassemia major, Uncontrolled beta thalassemia major |
Primary outcome
Evaluate bone denisty in children with beta thalassemia major
Time frame: One year
To evaluate bone denisty in transfusion dependent beta thalassemia major \& its relation to serum minerals \&vit D To evaluate bone denisity in transfusion dependent beta thalassemia major \& its relation to serum minerals \&vit D To evaluate bone denisity in transfusion dependent beta thalassemia major \&its relation to serrum minerals \&vitD
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Oct 2023.
- Background: PubMed 29991466Gaudio A, Morabito N, Catalano A, Rapisarda R, Xourafa A, Lasco A. Pathogenesis of Thalassemia Major-associated Osteoporosis: A Review with Insights from Clinical Experience. J Clin Res Pediatr Endocrinol. 2019 May 28;11(2):110-117. doi: 10.4274/jcrpe.galenos.2018.2018.0074. Epub 2018 Jul 11.
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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
An observational study of Evaluate Bone Denisty Changes in Children With Beta Thalassemia Major, run by Marwa Hassan Abdelhamed Hassan.
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 5 Oct 2023. 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?
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 bone denisty in children with beta thalassemia major (time frame: One year). 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 NCT06069089, 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 5 Oct 2023.