Transfusion Dependent Beta-thalassemiaPhase 4NCT06606886
Effects of Hydroxyurea and Metformin in Transfusion Dependent Beta-Thalassemia
A Phase 4 study testing Tablet Metformin (Group A) and Tablet Hydroxyurea (Group B) in people with Transfusion Dependent Beta-thalassemia, run by Bahria University at 1 site in Pakistan.
Trial summary
This clinical trial aims to evaluate and compare the effects and safety of hydroxyurea and metformin in transfusion-dependent beta-thalassemia major and correlate responders/non-responders with the single nucleotide polymorphism mutations (SNPs) by DNA analysis. Read more about this study
- Who is it for?
- Ages 10 to 25 years
- Conditions the study is for
- Transfusion Dependent Beta-thalassemia
- What is being tested?
- Tablet Metformin (Group A) and Tablet Hydroxyurea (Group B)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 96 participants
- Where
- 1 sitePakistan
- Study dates
- 16 Sep 2024to 9 Feb 2026
- Phase
- Phase 4
- 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 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosed cases of beta-thalassemia major
- Transfusion dependent patients and on iron chelating agents
- Males and females 10 to 25 years of age
- Creatinine ≤1.4 mg/dL
- Written informed consent from the patients and up to 18 years consent from the Parents/Guardians.
Who cannot join
- Failure to meet inclusion criteria
- Refusal to participate
- Creatinine >1.4mg/dL
- Liver function tests (ALT and conjugated bilirubin) > 4 times upper limits of normal
- Massive splenomegaly \& Liver fibrosis
- Patient on growth hormone
Show all 10Show fewer
- Sickle beta thalassemia
- Severe osteoporosis
- Hepatitis B \& C
- HIV
Trial locations and contacts
1 site in 1 country.
About this study
This clinical trial aims to evaluate and compare the effects and safety of hydroxyurea and metformin in transfusion-dependent beta-thalassemia major and correlate responders/non-responders with the single nucleotide polymorphism mutations (SNPs) by DNA analysis.
The main question to answer is:
Read more
- Is there a significant difference in the effects and safety of hydroxyurea and metformin in transfusion-dependent beta-thalassemia major?
Participants will be divided into two treatment groups. Participants of group A will receive Tab. Metformin 500mg/dose twice daily orally for one year. Participants of group B will receive Tab. Hydroxyurea 10-20mg/kg once daily orally for one year.
At the end of the study, Investigators will compare the effects and safety of hydroxyurea and metformin in transfusion-dependent beta-thalassemia major and correlate responders/non-responders with the single nucleotide polymorphism mutations (SNPs) by DNA analysis.
Read the detailed description
The study will be conducted after obtaining approval from the Institutional Review Board (IRB) at Bahria University Health Sciences Campus Karachi (BUHSCK) and the Faculty Research Committee (FRC). After obtaining written informed consent, 48 participants in each group with beta-thalassemia major will be selected from the National Institute of Blood Diseases and Bone Marrow Transplantation. Participants will be randomized into two treatment groups using computer-generated randomization.
Group A participants will receive Metformin 500 mg/dose twice daily orally for one year. Group B participants will receive Hydroxyurea 10-20 mg/kg once daily orally for one year.
Dose escalation therapy will be implemented to improve participant compliance and tolerance. The first month will not be included in the study period for each participant. A general physical examination will be conducted monthly. Blood pressure (mm Hg) will be assessed using a sphygmomanometer. The pulse rate (beats per minute) will be determined by palpating the radial artery, and the Body Mass Index (kg/m^2) will be calculated for each participant.
Dietary Counseling
People with thalassemia are advised to consume vitamin C-rich foods like vegetables and fruits to prevent depletion from iron overload. They should also include dairy products in their diet to counter calcium depletion, while avoiding high-iron foods such as beef, liver, spinach, and dates.
SF (Short-form) Questionnaire
All participants will complete the SF-36, a 36-item Short-Form Health Survey, at baseline, 6 months, and 12 months. The SF-36 is a widely used tool for evaluating overall health status, consisting of 36 items across 8 domains. The scores contribute to two main components: the physical component summary (PCS) and the mental component summary (MCS). The SF-36 is employed to assess Health-Related Quality of Life (HRQoL) in both the general population and individuals with various medical conditions, including beta-thalassemia major (β-TM), allowing for comparisons of HRQoL across different illnesses and health statuses.
- Baseline Investigations/Parameters (at day zero)
Baseline investigations will include Complete Blood Count (CBC), Random Blood Sugar (RBS), Erythrocyte Sedimentation Rate (ESR), Serum Creatinine, Serum Ferritin, Liver Function Tests (LFTs), HCV RNA test, Fetal Hemoglobin (HbF) level, Abdominal Ultrasound, Dual-Energy X-ray Absorptiometry (DEXA) Scan, and DNA mutations. Health-Related Quality of Life (HRQoL) will be assessed by the SF-36 Questionnaire at baseline, 6 months, and 12 months.
II) Monthly Follow-up for Clinical Features
Only CBC will be performed monthly, and any adverse effects related to the drugs will be inquired about.
III) At 3 Months
CBC, RBS, Serum Creatinine, and LFTs will be assessed for any adverse effects on blood, kidneys, and liver.
IV) At 6 \& 12 Months
All baseline investigations, except DNA mutations, will be repeated.
- At 9 Months
Random Blood Sugar, Serum Ferritin, and Serum Creatinine will be assessed.
Adverse Effects of Drugs
Adverse effects will be explained to each participant as follows: A) Metformin: nausea, vomiting, abdominal bloating, diarrhea, etc. B) Hydroxyurea: cytopenia, nausea, vomiting, abdominal pain, rashes, etc.
DNA mutations will be analyzed using Amplification Refractory Mutation System-Polymerase Chain Reaction (ARMS-PCR) in responders and non-responders.
Data will be collected using the Consent Form and SF-36 Questionnaire. Data analysis will be performed using IBM Statistical Package for the Social Sciences (SPSS), version 27. A p-value ≤ 0.05 will be considered statistically significant. Descriptive statistics (mean ± SD) will be used for continuous variables, while categorical variables will be presented as frequencies and percentages. To determine the mean difference, a repeated-measures two-way ANOVA within each group will be used. For correlation analysis, an independent T-test will be employed.
- Conditions
- Transfusion Dependent Beta-thalassemia
- Being tested
- Tablet Metformin (Group A), Tablet Hydroxyurea (Group B)
- Keywords
- Beta-Thalassemia Major, Fetal hemoglobin, Hydroxyurea, Metformin
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Tablet Metformin (Group A)Drug
Also called Tab. Glucophage 500mg
Tablet Metformin 500 mg/dose orally twice a day with meal for 1 year
Tablet Hydroxyurea (Group B)Drug
Also called Cap. Hydrea 500mg
Tablet Hydroxyurea 10-20mg/kg once daily orally with breakfast for 1 year
| Study group | Role | What they receive |
|---|---|---|
| (Group A) Tablet Metformin | Active comparator | Tablet Metformin 500 mg/dose orally twice a day with meal for 1 year Receives: Drug: Tablet Metformin (Group A) |
| (Group B) Tablet Hydroxyurea | Active comparator | Tablet Hydroxyurea 10-20mg/kg once daily orally with breakfast for 1 year Receives: Drug: Tablet Hydroxyurea (Group B) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Fetal Haemoglobin (HbF) Percentage in responders and non-responders
Assessment will be done at 6 months and 1 year after the baseline
Measured: 6 months to 1 year after the baseline
Change in frequency of blood transfusions
Assessment will be done at 6 months and 1 year after the baseline
Measured: 6 months to 1 year after the baseline
Secondary outcomes (2)
Change in quality of life assessed by the 36-Item Short Form Health Survey (SF-36) Questionnaire
Assessment will be conducted on a scoring scale of 0 to 100, with 0 being the lowest and 100 the highest possible scores, at 6 months and 1 year after the baseline.
Measured: 6 months to 1 year from the baseline
Correlations of responders and non-responders with DNA mutations
Assessment will be done after 1 year from the baseline
Measured: After 1 year at the end of study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Sep 2024
- Study start16 Sep 2024
- Primary completion9 Feb 2026
- Study completion9 Feb 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (7)Papers the sponsor has linked to this study.
- Other trials for Transfusion Dependent Beta-thalassemiaStudies 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
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 96 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bahria University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Ayesha Khan, Bahria University
- Investigators
- Ayesha Khan, MBBS MPhil, Principal investigator, Bahria University Islamabad
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06606886
- Sponsor's protocol ID
- BUHS-IRB # R-063/24
- Status last verified
- Apr 2026
- First submitted
- 11 Sep 2024
- Last updated
- 28 Apr 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: All data will be shared
Publications
ElTagui M, El-Ghamrawy M, AlDeeb SK, Nassim MS. Metformin as a Fetal Hemoglobin Inducer in Non-transfusion Dependent Thalassemia Patients. Indian J Hematol Blood Transfus. 2024 Jan;40(1):68-73. doi: 10.1007/s12288-023-01662-1. Epub 2023 May 5.
PubMed 38312174Yasara N, Wickramarathne N, Mettananda C, Silva I, Hameed N, Attanayaka K, Rodrigo R, Wickramasinghe N, Perera L, Manamperi A, Premawardhena A, Mettananda S. A randomised double-blind placebo-controlled clinical trial of oral hydroxyurea for transfusion-dependent beta-thalassaemia. Sci Rep. 2022 Feb 17;12(1):2752. doi: 10.1038/s41598-022-06774-8.
PubMed 35177777Arian M, Mirmohammadkhani M, Ghorbani R, Soleimani M. Health-related quality of life (HRQoL) in beta-thalassemia major (beta-TM) patients assessed by 36-item short form health survey (SF-36): a meta-analysis. Qual Life Res. 2019 Feb;28(2):321-334. doi: 10.1007/s11136-018-1986-1. Epub 2018 Sep 7.
PubMed 30194626Yasara N, Premawardhena A, Mettananda S. A comprehensive review of hydroxyurea for beta-haemoglobinopathies: the role revisited during COVID-19 pandemic. Orphanet J Rare Dis. 2021 Mar 1;16(1):114. doi: 10.1186/s13023-021-01757-w.
PubMed 33648529Mu W, Liang G, Feng Y, Jiang Y, Qu F. The Potential Therapeutic Role of Metformin in Diabetic and Non-Diabetic Bone Impairment. Pharmaceuticals (Basel). 2022 Oct 17;15(10):1274. doi: 10.3390/ph15101274.
PubMed 36297386Zhang Y, Wang H, Xiao H. Metformin Actions on the Liver: Protection Mechanisms Emerging in Hepatocytes and Immune Cells against NASH-Related HCC. Int J Mol Sci. 2021 May 9;22(9):5016. doi: 10.3390/ijms22095016.
PubMed 34065108Gluba-Brzozka A, Franczyk B, Rysz-Gorzynska M, Rokicki R, Koziarska-Rosciszewska M, Rysz J. Pathomechanisms of Immunological Disturbances in beta-Thalassemia. Int J Mol Sci. 2021 Sep 7;22(18):9677. doi: 10.3390/ijms22189677.
PubMed 34575839
Study at a glance
From the registry record, last updated by the sponsor on 28 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effects of Hydroxyurea and Metformin in Transfusion Dependent Beta-Thalassemia
- ClinicalTrials.gov ID
- NCT06606886
- Acronym / sponsor ID
- BUHS-IRB # R-063/24
- Conditions
- Transfusion Dependent Beta-thalassemia
- Keywords
- Beta-Thalassemia Major, Fetal hemoglobin, Hydroxyurea, Metformin
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 96 participants (actual)
- Lead sponsor
- Bahria UniversityUniversity, hospital or other
- Study start
- 16 Sep 2024 (actual)
- Primary completion
- 9 Feb 2026 (actual)
- Study completion
- 9 Feb 2026 (actual)
- First posted
- 23 Sep 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.
This clinical trial aims to evaluate and compare the effects and safety of hydroxyurea and metformin in transfusion-dependent beta-thalassemia major and correlate responders/non-responders with the single nucleotide polymorphism mutations (SNPs) by DNA analysis.
The main question to answer is:
- Is there a significant difference in the effects and safety of hydroxyurea and metformin in transfusion-dependent beta-thalassemia major?
Participants will be divided into two treatment groups. Participants of group A will receive Tab. Metformin 500mg/dose twice daily orally for one year. Participants of group B will receive Tab. Hydroxyurea 10-20mg/kg once daily orally for one year.
At the end of the study, Investigators will compare the effects and safety of hydroxyurea and metformin in transfusion-dependent beta-thalassemia major and correlate responders/non-responders with the single nucleotide polymorphism mutations (SNPs) by DNA analysis.
Detailed description
The study will be conducted after obtaining approval from the Institutional Review Board (IRB) at Bahria University Health Sciences Campus Karachi (BUHSCK) and the Faculty Research Committee (FRC). After obtaining written informed consent, 48 participants in each group with beta-thalassemia major will be selected from the National Institute of Blood Diseases and Bone Marrow Transplantation. Participants will be randomized into two treatment groups using computer-generated randomization.
Group A participants will receive Metformin 500 mg/dose twice daily orally for one year. Group B participants will receive Hydroxyurea 10-20 mg/kg once daily orally for one year.
Dose escalation therapy will be implemented to improve participant compliance and tolerance. The first month will not be included in the study period for each participant. A general physical examination will be conducted monthly. Blood pressure (mm Hg) will be assessed using a sphygmomanometer. The pulse rate (beats per minute) will be determined by palpating the radial artery, and the Body Mass Index (kg/m^2) will be calculated for each participant.
Dietary Counseling
People with thalassemia are advised to consume vitamin C-rich foods like vegetables and fruits to prevent depletion from iron overload. They should also include dairy products in their diet to counter calcium depletion, while avoiding high-iron foods such as beef, liver, spinach, and dates.
SF (Short-form) Questionnaire
All participants will complete the SF-36, a 36-item Short-Form Health Survey, at baseline, 6 months, and 12 months. The SF-36 is a widely used tool for evaluating overall health status, consisting of 36 items across 8 domains. The scores contribute to two main components: the physical component summary (PCS) and the mental component summary (MCS). The SF-36 is employed to assess Health-Related Quality of Life (HRQoL) in both the general population and individuals with various medical conditions, including beta-thalassemia major (β-TM), allowing for comparisons of HRQoL across different illnesses and health statuses.
- Baseline Investigations/Parameters (at day zero)
Baseline investigations will include Complete Blood Count (CBC), Random Blood Sugar (RBS), Erythrocyte Sedimentation Rate (ESR), Serum Creatinine, Serum Ferritin, Liver Function Tests (LFTs), HCV RNA test, Fetal Hemoglobin (HbF) level, Abdominal Ultrasound, Dual-Energy X-ray Absorptiometry (DEXA) Scan, and DNA mutations. Health-Related Quality of Life (HRQoL) will be assessed by the SF-36 Questionnaire at baseline, 6 months, and 12 months.
II) Monthly Follow-up for Clinical Features
Only CBC will be performed monthly, and any adverse effects related to the drugs will be inquired about.
III) At 3 Months
CBC, RBS, Serum Creatinine, and LFTs will be assessed for any adverse effects on blood, kidneys, and liver.
IV) At 6 \& 12 Months
All baseline investigations, except DNA mutations, will be repeated.
- At 9 Months
Random Blood Sugar, Serum Ferritin, and Serum Creatinine will be assessed.
Adverse Effects of Drugs
Adverse effects will be explained to each participant as follows: A) Metformin: nausea, vomiting, abdominal bloating, diarrhea, etc. B) Hydroxyurea: cytopenia, nausea, vomiting, abdominal pain, rashes, etc.
DNA mutations will be analyzed using Amplification Refractory Mutation System-Polymerase Chain Reaction (ARMS-PCR) in responders and non-responders.
Data will be collected using the Consent Form and SF-36 Questionnaire. Data analysis will be performed using IBM Statistical Package for the Social Sciences (SPSS), version 27. A p-value ≤ 0.05 will be considered statistically significant. Descriptive statistics (mean ± SD) will be used for continuous variables, while categorical variables will be presented as frequencies and percentages. To determine the mean difference, a repeated-measures two-way ANOVA within each group will be used. For correlation analysis, an independent T-test will be employed.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| (Group A) Tablet Metformin | Active comparator | Drug: Tablet Metformin (Group A)Tablet Metformin 500 mg/dose orally twice a day with meal for 1 year |
| (Group B) Tablet Hydroxyurea | Active comparator | Drug: Tablet Hydroxyurea (Group B)Tablet Hydroxyurea 10-20mg/kg once daily orally with breakfast for 1 year |
Who may qualify
- Ages
- 10 to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Diagnosed cases of beta-thalassemia major
- Transfusion dependent patients and on iron chelating agents
- Males and females 10 to 25 years of age
- Creatinine ≤1.4 mg/dL
- Written informed consent from the patients and up to 18 years consent from the Parents/Guardians.
Key exclusion criteria (10 listed)
- Failure to meet inclusion criteria
- Refusal to participate
- Creatinine >1.4mg/dL
- Liver function tests (ALT and conjugated bilirubin) > 4 times upper limits of normal
- Massive splenomegaly \& Liver fibrosis
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 |
|---|---|---|
| Tablet Metformin (Group A)Also: Tab. Glucophage 500mgTablet Metformin 500 mg/dose orally twice a day with meal for 1 year | Drug | (Group A) Tablet Metformin |
| Tablet Hydroxyurea (Group B)Also: Cap. Hydrea 500mgTablet Hydroxyurea 10-20mg/kg once daily orally with breakfast for 1 year | Drug | (Group B) Tablet Hydroxyurea |
Primary outcome
Change in Fetal Haemoglobin (HbF) Percentage in responders and non-responders
Time frame: 6 months to 1 year after the baseline
Assessment will be done at 6 months and 1 year after the baseline
Change in frequency of blood transfusions
Time frame: 6 months to 1 year after the baseline
Assessment will be done at 6 months and 1 year after the baseline
2 secondary outcomes
Change in quality of life assessed by the 36-Item Short Form Health Survey (SF-36) Questionnaire
Time frame: 6 months to 1 year from the baseline
Correlations of responders and non-responders with DNA mutations
Time frame: After 1 year at the end of study
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 28 Apr 2026.
- Background: PubMed 38312174ElTagui M, El-Ghamrawy M, AlDeeb SK, Nassim MS. Metformin as a Fetal Hemoglobin Inducer in Non-transfusion Dependent Thalassemia Patients. Indian J Hematol Blood Transfus. 2024 Jan;40(1):68-73. doi: 10.1007/s12288-023-01662-1. Epub 2023 May 5.
- Background: PubMed 35177777Yasara N, Wickramarathne N, Mettananda C, Silva I, Hameed N, Attanayaka K, Rodrigo R, Wickramasinghe N, Perera L, Manamperi A, Premawardhena A, Mettananda S. A randomised double-blind placebo-controlled clinical trial of oral hydroxyurea for transfusion-dependent beta-thalassaemia. Sci Rep. 2022 Feb 17;12(1):2752. doi: 10.1038/s41598-022-06774-8.
- Background: PubMed 30194626Arian M, Mirmohammadkhani M, Ghorbani R, Soleimani M. Health-related quality of life (HRQoL) in beta-thalassemia major (beta-TM) patients assessed by 36-item short form health survey (SF-36): a meta-analysis. Qual Life Res. 2019 Feb;28(2):321-334. doi: 10.1007/s11136-018-1986-1. Epub 2018 Sep 7.
- Background: PubMed 33648529Yasara N, Premawardhena A, Mettananda S. A comprehensive review of hydroxyurea for beta-haemoglobinopathies: the role revisited during COVID-19 pandemic. Orphanet J Rare Dis. 2021 Mar 1;16(1):114. doi: 10.1186/s13023-021-01757-w.
- Background: PubMed 36297386Mu W, Liang G, Feng Y, Jiang Y, Qu F. The Potential Therapeutic Role of Metformin in Diabetic and Non-Diabetic Bone Impairment. Pharmaceuticals (Basel). 2022 Oct 17;15(10):1274. doi: 10.3390/ph15101274.
- Background: PubMed 34065108Zhang Y, Wang H, Xiao H. Metformin Actions on the Liver: Protection Mechanisms Emerging in Hepatocytes and Immune Cells against NASH-Related HCC. Int J Mol Sci. 2021 May 9;22(9):5016. doi: 10.3390/ijms22095016.
- Background: PubMed 34575839Gluba-Brzozka A, Franczyk B, Rysz-Gorzynska M, Rokicki R, Koziarska-Rosciszewska M, Rysz J. Pathomechanisms of Immunological Disturbances in beta-Thalassemia. Int J Mol Sci. 2021 Sep 7;22(18):9677. doi: 10.3390/ijms22189677.
- Metformin as a Fetal Hemoglobin Inducer in Non-transfusion Dependent Thalassemia PatientsLink provided by the sponsor.
- A randomised double-blind placebo-controlled clinical trial of oral hydroxyurea for transfusion-dependent β-thalassaemiaLink provided by the sponsor.
- Health-related quality of life (HRQoL) in beta-thalassemia major (β TM) patients assessed by 36-item short form health survey (SF-36): a meta-analysisLink provided by the sponsor.
- A comprehensive review of hydroxyurea for β-haemoglobinopathies: the role revisited during COVID-19 pandemicLink provided by the sponsor.
- The Potential Therapeutic Role of Metformin in Diabetic and Non-Diabetic Bone Impairment. Pharmaceuticals (Basel)Link provided by the sponsor.
- Metformin Actions on the Liver: Protection Mechanisms Emerging in Hepatocytes and Immune Cells against NASH-Related HCCLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. All data will be shared
Related trials for Transfusion Dependent Beta-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
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
RecruitingPhase 3
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
Transfusion-dependent Alpha-Thalassemia, 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.
Ayesha Khan, MBBS MPhil principal investigator
Bahria University Islamabad
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Tablet Metformin (Group A) and Tablet Hydroxyurea (Group B) in people with Transfusion Dependent Beta-thalassemia, run by Bahria University at 1 site in Pakistan.
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 5 inclusion and 10 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 28 Apr 2026. 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 Pakistan. 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.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Change in Fetal Haemoglobin (HbF) Percentage in responders and non-responders (time frame: 6 months to 1 year after the baseline). 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 NCT06606886, which also lists 7 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 28 Apr 2026.