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

NCT06299670 | Recruiting Phase 4 DMC

• 1 other identifier: 939
• Study Type: interventional
• Target: 90
• Locations: 1 country
• Sites: 1

Brief Summary

Transfusion Dependent Thalassemia (TDT) is an emerging global public health concern. Hemopoietic stem cell transplantation (HSCT) is the only curative treatment. But its adoption is limited due to lack of HLA matched donor, experienced centers and high initial cost. So, researches are going on in search of an effective, safe, easily available treatment option. Use of fetal haemoglobin inducing agents shows promising effects in treatment of TDT patients. Thalidomide an immunomodulating and anti-angiogenic drug has been shown to induce γ-globin gene expression and increase the proliferation of erythroid cells. Furthermore Hydroxyurea (HU) is known to increase haemoglobin (Hb) by HbF induction and reduction of inflammation and hypercoagulability. 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 through randomized trials. This single centered non blinded quasi randomized clinical trial will be conducted at the Department of Pediatric Hematology and Oncology in Bangabandhu Sheikh Mujib Medical University (BSMMU), Bangladesh for one year of period. Thirty transfusion dependent thalassemia children of 3-18 years old will be included in each group. The objective of this study is to assess the effectiveness of combination of Thalidomide and Hydroxyurea in TDT children. It will play an important role in planning a cost effective and affordable treatment option for TDT children.This study will involve minimum physical risk to the patient. Written informed consent will be taken from parents or study subjects after brief explanation of the purpose and procedure. They will also be informed about the freedom to participate or not to participate at any time. Privacy and confidentiality will be safe guarded. History regarding age, sex, height, weight of these patients will be taken. Thorough physical examinations and laboratory investigations including CBC, Hb electrophoresis, serum Ferritin, serum creatinine, SGPT will be done. Data will be collected in a predesigned questionnaire and will be kept confidential. Statistical analysis will be done using the statistical software SPSS.

Conditions

Transfusion-dependent Thalassemia

Keywords

Trnafusion dependent thalssemia, Hydroxyurea, Thalidomide

Outcome Measures

Primary Outcomes (3)

• Change in Hb Level

  Change in Hb gm/dl after 3 month

  12 weeks after treatment initiation

• Change in Blood transfusion frequency

  Change in Blood transfusion frequency after 3 month

  12 weeks after treatment initiation

• Change in HbF

  Change in Hb F after 3 month

  12 weeks after treatment initiation

Secondary Outcomes (2)

• Change in Biochemicals

  12 weeks after treatment initiation

• Adverse effect

  12 weeks from treatment initiation

Study Arms (3)

Thalidomide

EXPERIMENTAL

Patient getting Thalidomide

Drug: Thalidomide

Hydroxyurea

EXPERIMENTAL

Hydroxyurea

Drug: Hydroxy Urea

Combination

EXPERIMENTAL

combination of Thalidomide \& Hydroxyurea

Drug: Combinations

Interventions

Thalidomide DRUG

Patients will get only Thalidomide

Thalidomide

Hydroxy Urea DRUG

Patients will get only Hydroxyurea

Hydroxyurea

Combinations DRUG

Combination Of Thalidomide \& Hydroxyurea

Combination

Eligibility Criteria

• Age: 3 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Bangabandhu Sheikh Mujib Medical University, Dhaka, Bangladesh: lead

Study Sites (1)

Bangabandhu Sheikh Mujib Medical University

Dhaka, 1000, Bangladesh

RECRUITING

Related Publications (22)

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MeSH Terms

Interventions

Thalidomide; Hydroxyurea

Intervention Hierarchy (Ancestors)

Phthalimides; Phthalic Acids; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Piperidones; Piperidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Isoindoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Urea; Amides

Central Study Contacts

Md. Anwarul Karim, MBBS,FCPS, MD

CONTACT

+880255165814; drkarim1990@gmail.com

Mohammad Hafizurr Rahman, MBBS,MD

CONTACT

+8801521438565; registrar@bsmmu.edu.bd

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: PARTICIPANT
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: March 2, 2024
• First Posted: March 8, 2024
• Study Start: November 1, 2023
• Primary Completion: June 30, 2024
• Study Completion: December 31, 2024
• Last Updated: March 8, 2024

Record last verified: 2024-03

Data Sharing

• IPD Sharing: Will not share

Locations

BA (1)