Beta-ThalassemiaNCT06568926
Adherence of Beta Thalssemia Patients to Oral Chelation Therapy
A study testing Serum Ferritin level in people with Beta-Thalassemia, run by Sohag University at 1 site in Egypt.
Trial summary
The β-thalassemias are a group of inherited disorders of hemoglobin (Hb) synthesis characterized by chronic anemia of varying severity. The degree of anemia relies on several genetic and environmental factors and determines the need for regular transfusion therapy. Read more about this study
- Who is it for?
- Ages 2 to 18 years
- Conditions the study is for
- Beta-Thalassemia
- What is being tested?
- Serum Ferritin level
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 200 participants
- Where
- 1 siteEgypt
- Study dates
- 14 Jul 2024to 14 Jul 2025
- Phase
- Interventional
- 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 2 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- confirmed diagnosis of beta thalassemia major or intermedia,
- Age between 2-18 years,
- Receiving regular blood transfusions every 2-5 weeks
- Prescribed oral iron chelation therapy with deferasirox for at least 1 year prior to enrollment.
Who cannot join
- Age less than 2 years and more than 18 years
- Any cause of blood transfusion other than beta Thalassemia
- Patients on deferoxamine
Trial locations and contacts
1 site in 1 country.
ALzahraa Elsayad Ahmed, professor contact
Egypt1 site, 1 recruiting
- Recruiting
Sohag university Hospital
Sohag
Magdy M Amin, professor
About this study
The β-thalassemias are a group of inherited disorders of hemoglobin (Hb) synthesis characterized by chronic anemia of varying severity. The degree of anemia relies on several genetic and environmental factors and determines the need for regular transfusion therapy. It is now common practice to classify patients as having transfusion dependent β-thalassemia (TDT) or non-transfusion-dependent β-thalassemia (NTDT). Regarding geographical distribution of β-thalassemia, it prevails more in countries on the Mediterranean, South east of Asia and in the east of Europe. In Egypt, it is the most common cause of chronic blood loss: One thousand cases are recorded annually for every 1.5 million live births the disease prevalence is equal to1000 cases per 1.5 million live births (Ghazala et al., 2021).
The only curative treatment for thalassemia currently is a bone marrow transplant. However, it is restricted to only a few patients due to the non-availability of an HLA-matched donor and high cost. Thus, most patients receive regular blood transfusions accompanied by iron chelation therapy (ICT) as the standard of care. The ideal management of a patient with transfusion-dependent thalassemia (TDT) requires a multidisciplinary therapeutic approach. The main iron chelating agents include deferoxamine (DFO), deferiprone (DFP), and deferasirox (DFX). Due to poor oral bioavailibility, DFO is the only chelator that must be administered subcutaneously or intravenously up to once a day; DFP and DFX may be administered orally up to three times a day. The known side effects associated with each chelator include infusion reactions in DFO, gastrointestinal distress, agranulocytosis in DFP, and transaminitis in DFP and DFX.
- Conditions
- Beta-Thalassemia
- Being tested
- Serum Ferritin level
- 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 levelDiagnostic test
follow up to the ferritin level in beta thalaseemia patients on oral chelation therapy
| Study group | Role | What they receive |
|---|---|---|
| patient group | Experimental | Beta thalassemia patients on oral chelation therapy Receives: Diagnostic Test: Serum Ferritin level |
What is being measured
The primary outcome is the main question the study is designed to answer.
serum ferritin level in beta thalassemia patients adherent to oral chelation therapy
Adherence of Beta Thalssemia Patients to Oral Chelation Therapy
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Aug 2024
- Study start14 Jul 2024
- Primary completion14 Jul 2025 (estimated)
- Study completion14 Jul 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Beta-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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Health services research
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 200 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sohag University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Marwa Aly Mousa, Sohag University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06568926
- Sponsor's protocol ID
- Soh-Med-24-07-17MS
- Status last verified
- Aug 2024
- First submitted
- 21 Aug 2024
- Last updated
- 23 Aug 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Belhoul KM, Bakir ML, Saned MS, Kadhim AM, Musallam KM, Taher AT. Serum ferritin levels and endocrinopathy in medically treated patients with beta thalassemia major. Ann Hematol. 2012 Jul;91(7):1107-14. doi: 10.1007/s00277-012-1412-7. Epub 2012 Jan 28.
PubMed 22281991Delea TE, Edelsberg J, Sofrygin O, Thomas SK, Baladi JF, Phatak PD, Coates TD. Consequences and costs of noncompliance with iron chelation therapy in patients with transfusion-dependent thalassemia: a literature review. Transfusion. 2007 Oct;47(10):1919-29. doi: 10.1111/j.1537-2995.2007.01416.x.
PubMed 17880620Kannan S, Singh A. Compliance score as a monitoring tool to promote treatment adherence in children with thalassemia major for improved physical growth. Asian J Transfus Sci. 2017 Jul-Dec;11(2):108-114. doi: 10.4103/ajts.AJTS_61_16.
PubMed 28970676Nazir HF, Alshizawi M. Neutropenia and Life-threatening Agranulocytosis Among Children With beta-Thalassemia Treated With Oral Iron Chelators in a Community With Background of Ethnic Neutropenia. J Pediatr Hematol Oncol. 2020 Nov;42(8):e750-e755. doi: 10.1097/MPH.0000000000001699.
PubMed 31876777
Study at a glance
From the registry record, last updated by the sponsor on 23 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Adherence of Beta Thalssemia Patients to Oral Chelation Therapy
- ClinicalTrials.gov ID
- NCT06568926
- Acronym / sponsor ID
- Soh-Med-24-07-17MS
- Conditions
- Beta-Thalassemia
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- Sohag UniversityUniversity, hospital or other
- Study start
- 14 Jul 2024 (actual)
- Primary completion
- 14 Jul 2025 (estimated)
- Study completion
- 14 Jul 2025 (estimated)
- First posted
- 23 Aug 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.
The β-thalassemias are a group of inherited disorders of hemoglobin (Hb) synthesis characterized by chronic anemia of varying severity. The degree of anemia relies on several genetic and environmental factors and determines the need for regular transfusion therapy. It is now common practice to classify patients as having transfusion dependent β-thalassemia (TDT) or non-transfusion-dependent β-thalassemia (NTDT). Regarding geographical distribution of β-thalassemia, it prevails more in countries on the Mediterranean, South east of Asia and in the east of Europe. In Egypt, it is the most common cause of chronic blood loss: One thousand cases are recorded annually for every 1.5 million live births the disease prevalence is equal to1000 cases per 1.5 million live births (Ghazala et al., 2021).
The only curative treatment for thalassemia currently is a bone marrow transplant. However, it is restricted to only a few patients due to the non-availability of an HLA-matched donor and high cost. Thus, most patients receive regular blood transfusions accompanied by iron chelation therapy (ICT) as the standard of care. The ideal management of a patient with transfusion-dependent thalassemia (TDT) requires a multidisciplinary therapeutic approach. The main iron chelating agents include deferoxamine (DFO), deferiprone (DFP), and deferasirox (DFX). Due to poor oral bioavailibility, DFO is the only chelator that must be administered subcutaneously or intravenously up to once a day; DFP and DFX may be administered orally up to three times a day. The known side effects associated with each chelator include infusion reactions in DFO, gastrointestinal distress, agranulocytosis in DFP, and transaminitis in DFP and DFX.
Study design
- Primary purpose
- Health services research
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| patient group | Experimental | Diagnostic Test: Serum Ferritin levelBeta thalassemia patients on oral chelation therapy |
Who may qualify
- Ages
- 2 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- confirmed diagnosis of beta thalassemia major or intermedia,
- Age between 2-18 years,
- Receiving regular blood transfusions every 2-5 weeks
- Prescribed oral iron chelation therapy with deferasirox for at least 1 year prior to enrollment.
Key exclusion criteria (3 listed)
- Age less than 2 years and more than 18 years
- Any cause of blood transfusion other than beta Thalassemia
- Patients on deferoxamine
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 levelfollow up to the ferritin level in beta thalaseemia patients on oral chelation therapy | Diagnostic test | patient group |
Primary outcome
serum ferritin level in beta thalassemia patients adherent to oral chelation therapy
Time frame: 1 year
Adherence of Beta Thalssemia Patients to Oral Chelation Therapy
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Aug 2024.
- Background: PubMed 22281991Belhoul KM, Bakir ML, Saned MS, Kadhim AM, Musallam KM, Taher AT. Serum ferritin levels and endocrinopathy in medically treated patients with beta thalassemia major. Ann Hematol. 2012 Jul;91(7):1107-14. doi: 10.1007/s00277-012-1412-7. Epub 2012 Jan 28.
- Background: PubMed 17880620Delea TE, Edelsberg J, Sofrygin O, Thomas SK, Baladi JF, Phatak PD, Coates TD. Consequences and costs of noncompliance with iron chelation therapy in patients with transfusion-dependent thalassemia: a literature review. Transfusion. 2007 Oct;47(10):1919-29. doi: 10.1111/j.1537-2995.2007.01416.x.
- Background: PubMed 28970676Kannan S, Singh A. Compliance score as a monitoring tool to promote treatment adherence in children with thalassemia major for improved physical growth. Asian J Transfus Sci. 2017 Jul-Dec;11(2):108-114. doi: 10.4103/ajts.AJTS_61_16.
- Background: PubMed 31876777Nazir HF, Alshizawi M. Neutropenia and Life-threatening Agranulocytosis Among Children With beta-Thalassemia Treated With Oral Iron Chelators in a Community With Background of Ethnic Neutropenia. J Pediatr Hematol Oncol. 2020 Nov;42(8):e750-e755. doi: 10.1097/MPH.0000000000001699.
- Individual participant dataSharing planned: Undecided
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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.
ALzahraa Elsayad Ahmed, professor contact
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Serum Ferritin level in people with Beta-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 4 inclusion and 3 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 23 Aug 2024. 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 Marwa Ali Mousa, resident (01111815617, marwa.mousaa@med.sohag.edu.eg), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
serum ferritin level in beta thalassemia patients adherent to oral chelation therapy (time frame: 1 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 NCT06568926, which also lists 4 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 23 Aug 2024.