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

June 16, 2026 updated by: MD ANWARUL KARIM, Bangabandhu Sheikh Mujib Medical University, Dhaka, Bangladesh

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

90

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Bangladesh
      • Dhaka, Bangladesh, 1000
        • Bangabandhu Sheikh Mujib Medical University

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

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

Exclusion Criteria:

  • 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Single

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Thalidomide
Patient getting Thalidomide
Drug: Thalidomide
Patients will get only Thalidomide
Experimental: Hydroxyurea
Drug: Hydroxy Urea
Patients will get only Hydroxyurea
Experimental: Combination
combination of Thalidomide & Hydroxyurea
Drug: Combinations
Combination Of Thalidomide & Hydroxyurea

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in Hb Level
Time Frame: 12 weeks after treatment initiation
Change in Hb gm/dl after 3 month
12 weeks after treatment initiation
Change in Blood transfusion frequency
Time Frame: 12 weeks after treatment initiation
Change in Blood transfusion frequency after 3 month
12 weeks after treatment initiation
Change in HbF
Time Frame: 12 weeks after treatment initiation
Change in Hb F after 3 month
12 weeks after treatment initiation

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Adverse effect
Time Frame: 12 weeks from treatment initiation
Adverse effect of drugs
12 weeks from treatment initiation
Change in Serum Ferritin level
Time Frame: 12 weeks after treatment initiation
Change in serum ferritin level after 3 months
12 weeks after treatment initiation
Change in SGPT level
Time Frame: 12 weeks from treatment initiation
Change of serum ferritin after 3 months
12 weeks from treatment initiation
Change in serum bilirubin level
Time Frame: 12 weeks after initiation of treatment
Change of Serum Bilirubin level after 3 months
12 weeks after initiation of treatment
Change of Serum Creatinine level
Time Frame: 12 weeks after treatment initiation
Change of Serum Creatinine after 3 months
12 weeks after treatment initiation
Change of Serum LDH level
Time Frame: 12 weeks from treatment initiation
Change of LDH level after 3 months
12 weeks from treatment initiation

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Bangabandhu Sheikh Mujib Medical University, Dhaka, Bangladesh

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 1, 2023

Primary Completion (Actual)

January 30, 2026

Study Completion (Actual)

January 30, 2026

Study Registration Dates

First Submitted

March 2, 2024

First Submitted That Met QC Criteria

March 2, 2024

First Posted (Actual)

March 8, 2024

Study Record Updates

Last Update Posted (Actual)

June 17, 2026

Last Update Submitted That Met QC Criteria

June 16, 2026

Last Verified

June 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 939

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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