Transfusion Dependent Beta ThalassemiaPhase 2Thal-HNCT07292259
Combination of Thalidomide and Hydroxyuria in Transfusion Dependent Thalasemmia
A Phase 2 study testing ADDITION OF THALIDOMIDE AND HYDROXYUREA in people with Transfusion Dependent Beta Thalassemia, run by Pakistan Blood and Marrow Transplant (PBMT) Group at 9 sites in Pakistan.
Trial summary
Beta thalassemia Major (BTM) is the most common hemoglobinopathy caused by mutations in the beta-globin gene . Worldwide, approximately 80 million people carry thalassemia gene mutation. Around 23,000 babies are affected by BTM each year, of which around 90% belong to low- or middle-income nations. Read more about this study
- Who is it for?
- Ages 2 to 12 years
- Conditions the study is for
- Transfusion Dependent Beta Thalassemia
- What is being tested?
- ADDITION OF THALIDOMIDE AND HYDROXYUREA
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 9 sitesPakistan
- Study dates
- 1 Jan 2024to 30 Dec 2025
- Phase
- Phase 2
- 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 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Patients suffering from Transfusion Dependent β-thalassemia (TDBT) more than two years of age of either sex will be included in the study.
Who cannot join
- Age less than two years.
- Patients having cardiac, hepatic, pulmonary, renal or neurological dysfunction or history of thrombosis.
- Both male and female participants of childbearing potential will be excluded due to the teratogenicity of thalidomide.
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
Pakistan9 sites
Armed Forces Bone Marrow Transplant Center
Islamabad, Punjab Province, 46000
Armed Forces Bone Marrow Transplant Center
Rawalpindi, Punjab Province, 44000
AFBMTC (Clinical Trial and Research Cell)
Rawalpindi, Punjab Province, 46000
AFBMTC (CT&RC), CMH Medical Complex
Rawalpindi, Punjab Province, 46000
AFBMTC (CT&RC), Medical Complex
Rawalpindi, Punjab Province, 46000
AFBMTC, CMH Medical Complex
Rawalpindi, Punjab Province, 46000
Armed Forces Bone Marrow Transplant Center Rawalpindi Pakistan
Rawalpindi, Punjab Province, 46000
Armed Forces Bone Marrow Transplant Center
Rawalpindi, Punjab Province, 46000
Armed Forces Bone Marrow Transplant Center Rawalpindi Pakistan
Rawalpindi, Punjab Province, 60000
About this study
Official title: The Efficacy of Combined Thalidomide and Hydroxyurea Therapy in Transfusion Dependent β-thalassemia (TDBT), Phase II Trial
Beta thalassemia Major (BTM) is the most common hemoglobinopathy caused by mutations in the beta-globin gene . Worldwide, approximately 80 million people carry thalassemia gene mutation. Around 23,000 babies are affected by BTM each year, of which around 90% belong to low- or middle-income nations.
In Pakistan, the carrier prevalence of thalassemia is 5-7% resulting in a significant population of approximately 10 million carriers in the general population. There are 50,000 thalassemia patients registered in treatment facilities around the country, one of the highest global prevalence rates for transfusion dependent BTM. The average life expectancy of BTM patients in Pakistan is around 10 years of age, while life expectancy in developed countries is around 50 to 60 years. This difference is due to poor transfusion support, transfusion-transmitted infections (TTIs) and inadequate iron chelation leading to hepatotoxicity and cardiac failure.
The standard of care for BTM remains bone marrow transplantation or lifelong blood transfusions followed by iron chelation therapies. While standard care involves, challenges such as limited resources, lack of access to transplant services, and transfusion-related complications persist, particularly in low-and-middle-income countries.
Read the detailed description
Beta thalassemia Major (BTM) is the most common hemoglobinopathy caused by mutations in the beta-globin gene . Worldwide, approximately 80 million people carry thalassemia gene mutation. Around 23,000 babies are affected by BTM each year, of which around 90% belong to low- or middle-income nations.
In Pakistan, the carrier prevalence of thalassemia is 5-7% resulting in a significant population of approximately 10 million carriers in the general population. There are 50,000 thalassemia patients registered in treatment facilities around the country, one of the highest global prevalence rates for transfusion dependent BTM. The average life expectancy of BTM patients in Pakistan is around 10 years of age, while life expectancy in developed countries is around 50 to 60 years. This difference is due to poor transfusion support, transfusion-transmitted infections (TTIs) and inadequate iron chelation leading to hepatotoxicity and cardiac failure.
The standard of care for BTM remains bone marrow transplantation or lifelong blood transfusions followed by iron chelation therapies. While standard care involves, challenges such as limited resources, lack of access to transplant services, and transfusion-related complications persist, particularly in low-and-middle-income countries.
Hydroxyurea (HU), an FDA-approved inducer of fetal hemoglobin (HbF), has shown promise in reducing or eliminating the need for frequent blood transfusions in β-thalassemia patients. However, a subset of patients exhibits limited responsiveness to HU, necessitating exploration of adjunct or alternative therapies. Thalidomide, an immune modulator, has demonstrated transfusion reduction by suppressing nuclear factor-κB induction, potentially increasing HbF levels.
The primary aim of this prospective study is to determine the efficacy of the combination of thalidomide and hydroxyurea in reducing transfusion frequency in patients with β-thalassemia Major. The secondary objectives are to document the spectrum of significant adverse drug reactions as well to document any alteration in the spleen size and serum ferritin levels.
- Conditions
- Transfusion Dependent Beta Thalassemia
- Being tested
- ADDITION OF THALIDOMIDE AND HYDROXYUREA
- Keywords
- Thalidomide, Hydoxyurea, Transfusion dependent thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ADDITION OF THALIDOMIDE AND HYDROXYUREADrug
Also called Hydrea and Thalidomide
The intervention includes Hydroxyurea (HU) and Thalidomide in combination. The starting dose of Hydroxyurea will be 20 mg/kg once daily and of thalidomide will be 2.5-3 mg/kg once a day adjusted to nearest multiple of 10, at bedtime. Among those with partial response (PR) or no response (NR) after two months, the dose of thalidomide will be escalated in increments of 1 mg/kg/day at four weeks interval to a maximum of 5 mg/kg/day (maximum dose 100 mg/day).
- To prevent thrombosis, aspirin (2-4 mg/kg per day) will be used. All patients will receive Folic acid 2 to 5 mg once daily.
- Patients will also continue the iron chelation therapy (Deferasirox, Deferiprone or Deferoxamine) in case of iron overload.
| Study group | Role | What they receive |
|---|---|---|
| A combination of thalidomide and hydroxyurea is added to patients diagnosed with TDT | Experimental | * The trial include Hydroxyurea (HU) and Thalidomide in combination. The starting dose of Hydroxyurea will be 20 mg/kg once daily and of thalidomide will be 2.5-3 mg/kg once a day adjusted to nearest multiple of 10, at bedtime. Among those with partial response (PR) or no response (NR) after two months, the dose of thalidomide will be escalated in increments of 1 mg/kg/day at four weeks interval to a maximum of 5 mg/kg/day (maximum dose 100 mg/day).
* To prevent thrombosis, aspirin (2-4 mg/kg per day) will be used. All patients will receive Folic acid 2 to 5 mg once daily. Receives: Drug: ADDITION OF THALIDOMIDE AND HYDROXYUREA |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Red Blood Cell Transfusions after starting a combination of Thalidomide and hydroxyurea
The frequency of Red Blood cell transfusions before the start of intervention will be noted. At 03 months, 06 months, and 12 months of intervention (Thalidomide and hydroxyurea) number of Red Blood cell transfusions will be documented.
Measured: 01 Year
Secondary outcomes (3)
To document the spectrum and frequency of significant adverse drug reactions.
Monitoring and recording any adverse effects associated with thalidomide and hydroxyurea therapy at Day-90, Day-180, and Day-365.
Patients will be contacted on the telephone weekly by the drug safety monitoring board and will be screened for any side effects of the intervention.
Measured: 01 Year
To assess changes in spleen size during the intervention.
Assessment of spleen size in centimeters at Day-01, Day-90, Day-180, and Day-365 of intervention.
Measured: 01 Year
Monitoring of Serum Ferritin levels in ng/ml during the time of intervention.
Measurement of Serum Ferritin Levels in ng/ml at Day-01, Day-90, Day-180, and Day-365.
Measured: 01 Year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Dec 2025
- Study start1 Jan 2024
- Primary completion30 Dec 2025
- Study completion30 Dec 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 100 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pakistan Blood and Marrow Transplant (PBMT) Group
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Professor Tariq Ghafoor, Pakistan Blood and Marrow Transplant (PBMT) Group
- Investigators
- Tariq Ghafoor, FCPS,FRCP, Study director, National Institute of Blood and Marrow Transplant (NIBMT), Pakistan
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07292259
- Sponsor's protocol ID
- PBMT-1
- Other IDs
- 6871
- Acronym
- Thal-H
- Status last verified
- Dec 2025
- First submitted
- 13 Nov 2025
- Last updated
- 5 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: The investigators may share de-identified IPD upon reasonable request, beginning 9 months after publication and available for up to 24 months, following review and data-sharing agreement.
Publications
Li X, Hu S, Liu Y, Huang J, Hong W, Xu L, Xu H, Fang J. Efficacy of Thalidomide Treatment in Children With Transfusion Dependent beta-Thalassemia: A Retrospective Clinical Study. Front Pharmacol. 2021 Aug 12;12:722502. doi: 10.3389/fphar.2021.722502. eCollection 2021.
PubMed 34456732Garg A, Patel K, Shah K, Trivedi D, Raj A, Yadav R, Shah S. Safety and Efficacy of Thalidomide and Hydroxyurea Combination in Beta Thalassemia Patients. Indian J Hematol Blood Transfus. 2023 Jan;39(1):85-89. doi: 10.1007/s12288-022-01536-y. Epub 2022 Apr 21.
PubMed 36699430Chen J, Zhu W, Cai N, Bu S, Li J, Huang L. Thalidomide induces haematologic responses in patients with beta-thalassaemia. Eur J Haematol. 2017 Nov;99(5):437-441. doi: 10.1111/ejh.12955. Epub 2017 Sep 27.
PubMed 28850716Nag A, Radhakrishnan VS, Kumar J, Bhave S, Mishra DK, Nair R, Chandy M. Thalidomide in Patients with Transfusion-Dependent E-Beta Thalassemia Refractory to Hydroxyurea: A Single-Center Experience. Indian J Hematol Blood Transfus. 2020 Apr;36(2):399-402. doi: 10.1007/s12288-020-01263-2. Epub 2020 Mar 2.
PubMed 32425398Ansari SH, Ansari I, Wasim M, Sattar A, Khawaja S, Zohaib M, Hussain Z, Adil SO, Ansari AH, Ansari UH, Farooq F, Masqati NU. Evaluation of the combination therapy of hydroxyurea and thalidomide in beta-thalassemia. Blood Adv. 2022 Dec 27;6(24):6162-6168. doi: 10.1182/bloodadvances.2022007031.
PubMed 35477175
Study at a glance
From the registry record, last updated by the sponsor on 5 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Efficacy of Combined Thalidomide and Hydroxyurea Therapy in Transfusion Dependent β-thalassemia (TDBT), Phase II Trial
- ClinicalTrials.gov ID
- NCT07292259
- Acronym / sponsor ID
- Thal-H / PBMT-1
- Conditions
- Transfusion Dependent Beta Thalassemia
- Keywords
- Thalidomide, Hydoxyurea, Transfusion dependent thalassemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 100 participants (actual)
- Lead sponsor
- Pakistan Blood and Marrow Transplant (PBMT) GroupUniversity, hospital or other
- Study start
- 1 Jan 2024 (actual)
- Primary completion
- 30 Dec 2025 (actual)
- Study completion
- 30 Dec 2025 (actual)
- First posted
- 18 Dec 2025 (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.
Beta thalassemia Major (BTM) is the most common hemoglobinopathy caused by mutations in the beta-globin gene . Worldwide, approximately 80 million people carry thalassemia gene mutation. Around 23,000 babies are affected by BTM each year, of which around 90% belong to low- or middle-income nations.
In Pakistan, the carrier prevalence of thalassemia is 5-7% resulting in a significant population of approximately 10 million carriers in the general population. There are 50,000 thalassemia patients registered in treatment facilities around the country, one of the highest global prevalence rates for transfusion dependent BTM. The average life expectancy of BTM patients in Pakistan is around 10 years of age, while life expectancy in developed countries is around 50 to 60 years. This difference is due to poor transfusion support, transfusion-transmitted infections (TTIs) and inadequate iron chelation leading to hepatotoxicity and cardiac failure.
The standard of care for BTM remains bone marrow transplantation or lifelong blood transfusions followed by iron chelation therapies. While standard care involves, challenges such as limited resources, lack of access to transplant services, and transfusion-related complications persist, particularly in low-and-middle-income countries.
Detailed description
Beta thalassemia Major (BTM) is the most common hemoglobinopathy caused by mutations in the beta-globin gene . Worldwide, approximately 80 million people carry thalassemia gene mutation. Around 23,000 babies are affected by BTM each year, of which around 90% belong to low- or middle-income nations.
In Pakistan, the carrier prevalence of thalassemia is 5-7% resulting in a significant population of approximately 10 million carriers in the general population. There are 50,000 thalassemia patients registered in treatment facilities around the country, one of the highest global prevalence rates for transfusion dependent BTM. The average life expectancy of BTM patients in Pakistan is around 10 years of age, while life expectancy in developed countries is around 50 to 60 years. This difference is due to poor transfusion support, transfusion-transmitted infections (TTIs) and inadequate iron chelation leading to hepatotoxicity and cardiac failure.
The standard of care for BTM remains bone marrow transplantation or lifelong blood transfusions followed by iron chelation therapies. While standard care involves, challenges such as limited resources, lack of access to transplant services, and transfusion-related complications persist, particularly in low-and-middle-income countries.
Hydroxyurea (HU), an FDA-approved inducer of fetal hemoglobin (HbF), has shown promise in reducing or eliminating the need for frequent blood transfusions in β-thalassemia patients. However, a subset of patients exhibits limited responsiveness to HU, necessitating exploration of adjunct or alternative therapies. Thalidomide, an immune modulator, has demonstrated transfusion reduction by suppressing nuclear factor-κB induction, potentially increasing HbF levels.
The primary aim of this prospective study is to determine the efficacy of the combination of thalidomide and hydroxyurea in reducing transfusion frequency in patients with β-thalassemia Major. The secondary objectives are to document the spectrum of significant adverse drug reactions as well to document any alteration in the spleen size and serum ferritin levels.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| A combination of thalidomide and hydroxyurea is added to patients diagnosed with TDT | Experimental | Drug: ADDITION OF THALIDOMIDE AND HYDROXYUREA* The trial include Hydroxyurea (HU) and Thalidomide in combination. The starting dose of Hydroxyurea will be 20 mg/kg once daily and of thalidomide will be 2.5-3 mg/kg once a day adjusted to nearest multiple of 10, at bedtime. Among those with partial response (PR) or no response (NR) after two months, the dose of thalidomide will be escalated in increments of 1 mg/kg/day at four weeks interval to a maximum of 5 mg/kg/day (maximum dose 100 mg/day). * To prevent thrombosis, aspirin (2-4 mg/kg per day) will be used. All patients will receive Folic acid 2 to 5 mg once daily. |
Who may qualify
- Ages
- 2 to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- Patients suffering from Transfusion Dependent β-thalassemia (TDBT) more than two years of age of either sex will be included in the study.
Key exclusion criteria (3 listed)
- Age less than two years.
- Patients having cardiac, hepatic, pulmonary, renal or neurological dysfunction or history of thrombosis.
- Both male and female participants of childbearing potential will be excluded due to the teratogenicity of thalidomide.
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 |
|---|---|---|
| ADDITION OF THALIDOMIDE AND HYDROXYUREAAlso: Hydrea and ThalidomideThe intervention includes Hydroxyurea (HU) and Thalidomide in combination. The starting dose of Hydroxyurea will be 20 mg/kg once daily and of thalidomide will be 2.5-3 mg/kg once a day adjusted to nearest multiple of 10, at bedtime. Among those with partial response (PR) or no response (NR) after two months, the dose of thalidomide will be escalated in increments of 1 mg/kg/day at four weeks interval to a maximum of 5 mg/kg/day (maximum dose 100 mg/day). * To prevent thrombosis, aspirin (2-4 mg/kg per day) will be used. All patients will receive Folic acid 2 to 5 mg once daily. * Patients will also continue the iron chelation therapy (Deferasirox, Deferiprone or Deferoxamine) in case of iron overload. | Drug | A combination of thalidomide and hydroxyurea is added to patients diagnosed with TDT |
Primary outcome
Number of Red Blood Cell Transfusions after starting a combination of Thalidomide and hydroxyurea
Time frame: 01 Year
The frequency of Red Blood cell transfusions before the start of intervention will be noted. At 03 months, 06 months, and 12 months of intervention (Thalidomide and hydroxyurea) number of Red Blood cell transfusions will be documented.
3 secondary outcomes
To document the spectrum and frequency of significant adverse drug reactions.
Time frame: 01 Year
To assess changes in spleen size during the intervention.
Time frame: 01 Year
Monitoring of Serum Ferritin levels in ng/ml during the time of intervention.
Time frame: 01 Year
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 5 May 2026.
- Results publication: PubMed 34456732Li X, Hu S, Liu Y, Huang J, Hong W, Xu L, Xu H, Fang J. Efficacy of Thalidomide Treatment in Children With Transfusion Dependent beta-Thalassemia: A Retrospective Clinical Study. Front Pharmacol. 2021 Aug 12;12:722502. doi: 10.3389/fphar.2021.722502. eCollection 2021.
- Results publication: PubMed 36699430Garg A, Patel K, Shah K, Trivedi D, Raj A, Yadav R, Shah S. Safety and Efficacy of Thalidomide and Hydroxyurea Combination in Beta Thalassemia Patients. Indian J Hematol Blood Transfus. 2023 Jan;39(1):85-89. doi: 10.1007/s12288-022-01536-y. Epub 2022 Apr 21.
- Results publication: PubMed 28850716Chen J, Zhu W, Cai N, Bu S, Li J, Huang L. Thalidomide induces haematologic responses in patients with beta-thalassaemia. Eur J Haematol. 2017 Nov;99(5):437-441. doi: 10.1111/ejh.12955. Epub 2017 Sep 27.
- Results publication: PubMed 32425398Nag A, Radhakrishnan VS, Kumar J, Bhave S, Mishra DK, Nair R, Chandy M. Thalidomide in Patients with Transfusion-Dependent E-Beta Thalassemia Refractory to Hydroxyurea: A Single-Center Experience. Indian J Hematol Blood Transfus. 2020 Apr;36(2):399-402. doi: 10.1007/s12288-020-01263-2. Epub 2020 Mar 2.
- Results publication: PubMed 35477175Ansari SH, Ansari I, Wasim M, Sattar A, Khawaja S, Zohaib M, Hussain Z, Adil SO, Ansari AH, Ansari UH, Farooq F, Masqati NU. Evaluation of the combination therapy of hydroxyurea and thalidomide in beta-thalassemia. Blood Adv. 2022 Dec 27;6(24):6162-6168. doi: 10.1182/bloodadvances.2022007031.
- Individual participant dataSharing planned: Yes. The investigators may share de-identified IPD upon reasonable request, beginning 9 months after publication and available for up to 24 months, following review and data-sharing agreement.
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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.
Tariq Ghafoor, FCPS,FRCP study director
National Institute of Blood and Marrow Transplant (NIBMT), Pakistan
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ADDITION OF THALIDOMIDE AND HYDROXYUREA in people with Transfusion Dependent Beta Thalassemia, run by Pakistan Blood and Marrow Transplant (PBMT) Group at 9 sites 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 1 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: finished, as of the sponsor's last update on 5 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Number of Red Blood Cell Transfusions after starting a combination of Thalidomide and hydroxyurea (time frame: 01 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 NCT07292259, which also lists 5 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 5 May 2026.