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Transfusion-dependent ThalassemiaPhase 4NCT06299670

Efficacy of Combination of Hdroxyurea and Thalidomide Over Hydroxyurea or Thalidomide in the Treatment of Transfusion Dependent Thalassemia in Children

A Phase 4 study testing Thalidomide, Hydroxy Urea and Combinations in people with Transfusion-dependent Thalassemia, run by Bangabandhu Sheikh Mujib Medical University, Dhaka, Bangladesh at 1 site in Bangladesh.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The goal of this clinical trial is to find out the efficacy of combined Hydroxyurea and thalidomide transfusion dependent thalassemia patients. The main objectives are to compare the level of Hb, Transfusion interval, serum ferritin level before \& after treatment between single and combination of thalidomide and HU. Read more about this study

Who is it for?
Ages 3 to 18 years
Conditions the study is for
Transfusion-dependent Thalassemia
What is being tested?
Thalidomide, Hydroxy Urea and Combinations
Could I receive a placebo?
No, everyone receives an active treatment
How many people
90 participants
Where
1 siteBangladesh
Study dates
1 Nov 2023to 30 Jan 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 3 to 18 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Patients diagnosed as transfusion dependent thalassemia
  • Age ranged from 3-18 years
  • Blood transfusion more than 1 year.
  • no bleeding disorder

Who cannot join

  • Active systemic co-morbidity,
  • Past personal or family history of thrombophilia,
  • Recent fracture or recent major surgery
  • Use of drugs that might affect Hb levels 15 days before enrollment

Trial locations and contacts

1 site in 1 country.

Bangladesh1 site

  • Bangabandhu Sheikh Mujib Medical University

    Dhaka, 1000

Other trials in Bangladesh

About this study

Official title: Efficacy of Combination of Hdroxyurea and Thalidomide Over Either Hydroxyurea or Thalidomide Alone in the Treatment of Transfusion Dependent Thalassemia in Children: A Quasi-Randomised Clinical Trial

The goal of this clinical trial is to find out the efficacy of combined Hydroxyurea and thalidomide transfusion dependent thalassemia patients. The main objectives are to compare the level of Hb, Transfusion interval, serum ferritin level before \& after treatment between single and combination of thalidomide and HU.

Researcher will compare the effectiveness of combined hydroxyurea and thalidomide and hydroxyurea and thalidomide alone. Participants will be divided in three groups:

Group I: will take combination of HU and Thalidomide. Group II: Will take HU alone and Group III: Will take Thalidomide alone and outcome will be recorded.

Read the detailed description

Thalassemia is a monogenic hematological disorder caused as a result of defect in synthesis of globin chains of hemoglobin. It causes ineffective erythropoiesis \& lysis of red blood cells due to relative excess of unaffected globin chain.

Annually about 50,000 children with a severe form of thalassemia (β-thalassemia major and HbE β-thalassemia) born globally among which 26,000 patients are regular blood transfusion dependent. About 70-75% patients are found in southeast asia \& eastern mediterranean region.

Hematopoietic stem cell transplant (HSCT) is the only curative treatment option for homozygous thalassemia patients. Unfortunately its application is limited due to scarcity of HLA-matched donor, high cost , lack of specialized dedicated centers and risk of transplant related morbidity and mortality.

Regular blood transfusion is an essential life saving supportive care to maintain growth and development in children with severe β-thalassemia. However, Long term blood transfusion causes iron overload with cardiac, hepatic and endocrine coomplications , spread transfusion transmitted infections and formation of antibody. These limitations have compelled researchers to search for novel therapeutic modalities.

In recent years, induction of Fetal Hemoglobin (HbF) production pharmacologically is an promising treatment options for hemoglobinopathies. Different HbF inducing agents like Hydroxyurea (HU), Butyrate derivatives, Azacitidine, Decitabine, Tricostatin-A are shown to be effective in decreasing clinical severity and complications of TDT. HU induces a 2-9 fold increase in γ-globin gene and being used for decades in thalassemia treatment. But its utility is limited due to its mild and ill sustained therapeutic effect in HbF synthesis. Thalidomide, an immunomodulatory drug also shown to produce significant and persistent rise in Hb in few small studies and several case reports. It induces Gamma Globin gene expression by increasing reactive oxygen species-mediated p38 mitogen-activated protein kinase (MAPK) signaling and histone H4 acetylation.

Recent studies with combination of HU and Thalidomide have shown promising results in treatment of Thalassemia patients. However, most of those studies are retrospective or single arm nonrandomized trials \& The study population includes both adult and children age group. So the effectiveness of combination therapy of Thalidomide and HU needs to be established in children through randomized trials.

So the goal of our study to evaluate the effectiveness of combination of Thalidomide and HU in comaprison to Thalisomide or HU alone in children with TDT through a three arm quasi randomized trial.

Being tested
Thalidomide, Hydroxy Urea, Combinations
Keywords
Transfusion Dependent Thalassemia, Hydroxyurea, Thalidomide

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ThalidomideDrug

    Patients will get only Thalidomide

  • Hydroxy UreaDrug

    Patients will get only Hydroxyurea

  • CombinationsDrug

    Combination Of Thalidomide \& Hydroxyurea

Study groups in NCT06299670
Study groupRoleWhat they receive
ThalidomideExperimentalPatient getting Thalidomide
Receives: Drug: Thalidomide
HydroxyureaExperimentalHydroxyurea
Receives: Drug: Hydroxy Urea
CombinationExperimentalcombination of Thalidomide \& Hydroxyurea
Receives: Drug: Combinations

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change in Hb Level

    Change in Hb gm/dl after 3 month

    Measured: 12 weeks after treatment initiation

  2. Change in Blood transfusion frequency

    Change in Blood transfusion frequency after 3 month

    Measured: 12 weeks after treatment initiation

  3. Change in HbF

    Change in Hb F after 3 month

    Measured: 12 weeks after treatment initiation

Secondary outcomes (6)

  1. Adverse effect

    Adverse effect of drugs

    Measured: 12 weeks from treatment initiation

  2. Change in Serum Ferritin level

    Change in serum ferritin level after 3 months

    Measured: 12 weeks after treatment initiation

  3. Change in SGPT level

    Change of serum ferritin after 3 months

    Measured: 12 weeks from treatment initiation

  4. Change in serum bilirubin level

    Change of Serum Bilirubin level after 3 months

    Measured: 12 weeks after initiation of treatment

  5. Change of Serum Creatinine level

    Change of Serum Creatinine after 3 months

    Measured: 12 weeks after treatment initiation

  6. Change of Serum LDH level

    Change of LDH level after 3 months

    Measured: 12 weeks from treatment initiation

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted8 Mar 2024
  2. Study start1 Nov 2023
  3. Primary completion30 Jan 2026
  4. Study completion30 Jan 2026

Resources

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
Single blind
Who is masked
Participant
Enrolment
90 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06299670
Sponsor's protocol ID
939
Status last verified
Jun 2026
First submitted
2 Mar 2024
Last updated
17 Jun 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Aerbajinai W, Zhu J, Gao Z, Chin K, Rodgers GP. Thalidomide induces gamma-globin gene expression through increased reactive oxygen species-mediated p38 MAPK signaling and histone H4 acetylation in adult erythropoiesis. Blood. 2007 Oct 15;110(8):2864-71. doi: 10.1182/blood-2007-01-065201. Epub 2007 Jul 9.

    PubMed 17620452

  • Aguilar-Lopez LB, Delgado-Lamas JL, Rubio-Jurado B, Perea FJ, Ibarra B. Thalidomide therapy in a patient with thalassemia major. Blood Cells Mol Dis. 2008 Jul-Aug;41(1):136-7. doi: 10.1016/j.bcmd.2008.03.001. Epub 2008 Apr 24. No abstract available.

    PubMed 18439858

  • Bou-Fakhredin R, De Franceschi L, Motta I, Cappellini MD, Taher AT. Pharmacological Induction of Fetal Hemoglobin in beta-Thalassemia and Sickle Cell Disease: An Updated Perspective. Pharmaceuticals (Basel). 2022 Jun 16;15(6):753. doi: 10.3390/ph15060753.

    PubMed 35745672

  • Cappellini MD, Porter JB, Viprakasit V, Taher AT. A paradigm shift on beta-thalassaemia treatment: How will we manage this old disease with new therapies? Blood Rev. 2018 Jul;32(4):300-311. doi: 10.1016/j.blre.2018.02.001. Epub 2018 Feb 12.

    PubMed 29455932

  • Chen JM, Zhu WJ, Liu J, Wang GZ, Chen XQ, Tan Y, Xu WW, Qu LW, Li JY, Yang HJ, Huang L, Cai N, Wang WD, Huang K, Xu JQ, Li GH, He S, Luo TY, Huang Y, Liu SH, Wu WQ, Lu QY, Zhou MG, Chen SY, Li RL, Hu ML, Huang Y, Wei JH, Li JM, Chen SJ, Zhou GB. Safety and efficacy of thalidomide in patients with transfusion-dependent beta-thalassemia: a randomized clinical trial. Signal Transduct Target Ther. 2021 Nov 18;6(1):405. doi: 10.1038/s41392-021-00811-0.

    PubMed 34795208

  • Costa E, Cappellini MD, Rivella S, Chilin A, Alessi E, Riccaboni M, Leufkens HGM, Luzzatto L. Emergent treatments for beta-thalassemia and orphan drug legislations. Drug Discov Today. 2022 Nov;27(11):103342. doi: 10.1016/j.drudis.2022.103342. Epub 2022 Sep 1.

    PubMed 36058507

  • Fard AD, Hosseini SA, Shahjahani M, Salari F, Jaseb K. Evaluation of Novel Fetal Hemoglobin Inducer Drugs in Treatment of beta-Hemoglobinopathy Disorders. Int J Hematol Oncol Stem Cell Res. 2013;7(3):47-54.

    PubMed 24505535

  • Farmakis D, Porter J, Taher A, Domenica Cappellini M, Angastiniotis M, Eleftheriou A. 2021 Thalassaemia International Federation Guidelines for the Management of Transfusion-dependent Thalassemia. Hemasphere. 2022 Jul 29;6(8):e732. doi: 10.1097/HS9.0000000000000732. eCollection 2022 Aug.

    PubMed 35928543

  • Fozza C, Pardini S, Giannico DB, Targhetta C, Di Tucci AA, Dessalvi P, Angelucci E, Dore F. Dramatic erythroid response to low-dose thalidomide in two patients with transfusion independent thalassemia and severe post-transfusional alloimmune hemolysis. Am J Hematol. 2015 Jul;90(7):E141. doi: 10.1002/ajh.24030. No abstract available.

    PubMed 25850682

  • Green NS, Manwani D, Qureshi M, Ireland K, Sinha A, Smaldone AM. Decreased fetal hemoglobin over time among youth with sickle cell disease on hydroxyurea is associated with higher urgent hospital use. Pediatr Blood Cancer. 2016 Dec;63(12):2146-2153. doi: 10.1002/pbc.26161. Epub 2016 Aug 30.

    PubMed 27573582

  • Hossain MS, Raheem E, Sultana TA, Ferdous S, Nahar N, Islam S, Arifuzzaman M, Razzaque MA, Alam R, Aziz S, Khatun H, Rahim A, Morshed M. Thalassemias in South Asia: clinical lessons learnt from Bangladesh. Orphanet J Rare Dis. 2017 May 18;12(1):93. doi: 10.1186/s13023-017-0643-z.

    PubMed 28521805

  • Jain M, Chakrabarti P, Dolai TK, Ghosh P, Mandal PK, Baul SN, De R. Comparison of efficacy and safety of thalidomide vs hydroxyurea in patients with Hb E-beta thalassemia - a pilot study from a tertiary care Centre of India. Blood Cells Mol Dis. 2021 May;88:102544. doi: 10.1016/j.bcmd.2021.102544. Epub 2021 Feb 3.

    PubMed 33610115

  • 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 34456732

  • Masera N, Tavecchia L, Capra M, Cazzaniga G, Vimercati C, Pozzi L, Biondi A, Masera G. Optimal response to thalidomide in a patient with thalassaemia major resistant to conventional therapy. Blood Transfus. 2010 Jan;8(1):63-5. doi: 10.2450/2009.0102-09. No abstract available.

    PubMed 20104280

  • Rachmilewitz EA, Giardina PJ. How I treat thalassemia. Blood. 2011 Sep 29;118(13):3479-88. doi: 10.1182/blood-2010-08-300335. Epub 2011 Aug 2.

    PubMed 21813448

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing Thalidomide, Hydroxy Urea and Combinations in people with Transfusion-dependent Thalassemia, run by Bangabandhu Sheikh Mujib Medical University, Dhaka, Bangladesh at 1 site in Bangladesh.

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 4 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 17 Jun 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 Bangladesh. 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 Hb Level (time frame: 12 weeks after treatment initiation). The study lists 3 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 NCT06299670, which also lists 22 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 17 Jun 2026.

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