Clinical trial

Long Term Comparison of Combination Therapy (Thalidomide and Hydroxyurea) vs Thalidomide Alone in Beta Thalassemia.

Name

NIBD/IRB-242/12-2022

Description

The project, titled "Long Term Beta Thalassemia Treatment: Findings From The Extension Period Of Phase 2 Clinical Trial," aims to compare the efficacy and safety of combination therapy (thalidomide and hydroxyurea) versus thalidomide alone. The study, lasting three years, is a Phase 2 single-center, open-label interventional study with a sample size of 30 participants aged 8-35 years. It includes specific inclusion and exclusion criteria for participant selection. Data will be collected through clinical interviews and medical records and analyzed using(Statistical Package for the Social Sciences. This project aims to enhance beta thalassemia treatment strategies, focusing on reducing transfusion dependency and improving patient quality of life.

Trial arms

Trial start

2022-04-01

Estimated PCD

2024-04-01

Trial end

2025-04-01

Status

Active (not recruiting)

Phase

Early phase I

Treatment

thalidomide and hydroxyurea

Thalidomide: Thalidomide glutarimide is derivation of glutamic acid. Potentiating of fetal hemoglobin expression occurs by up regulation of Erythroid transcription factor and Erythroid Krüppel-like factor expression Furthermore few studies also concluded that thalidomide hypomethylate 27th amino acid in Histone H3 in the gamma globin gene. Initiating cause of this process is suppression of Nuclear factor (kappa-light-chain-enhancer of activated B cells) activation by tumor necrosis factor- alpha Vascular endothelial growth factor , Prostaglandin E2 and inflammatory cytokine. Hydroxyurea: Hydroxyurea or hydroxycarbamide (HU) lies in the category of antimetabolite. Mechanism of fetal hemoglobin induction includes increase in erythropoietin and nitric oxide production, apoptosis induction and potentiating in granulocyte cycling activity.

Arms:

combination therapy (thalidomide and hydroxyurea)

Other names:

combination therapy in beta thalassemia

Thalidomide

Thalidomide glutarimide is derivation of glutamic acid. Potentiating of fetal hemoglobin expression occurs by up regulation of Erythroid transcription factor and Erythroid Krüppel-like factor expression Furthermore few studies also concluded that thalidomide hypomethylate 27th amino acid in Histone H3 in the gamma globin gene. Initiating cause of this process is suppression of Nuclear factor (kappa-light-chain-enhancer of activated B cells) activation by tumor necrosis factor -alpha , Vascular endothelial growth factor , Prostaglandin E2 and inflammatory cytokine. Investigating the impact of thalidomide on transfusion-dependent beta thalassemia patients is essential for discerning its therapeutic efficacy and safety profile.

Arms:

thalidomide alone

Other names:

fetal hemoglobin inducer

Size

Primary endpoint

hemoglobin levels

3 years

red blood cell count.

3 years

leukocyte count

3 years

reticulocyte count

3 years

Transfusion Frequency:

3 years

Eligibility criteria

Inclusion Criteria: * Known case of beta thalassemia major/intermediate (transfusion dependent) * Willing to give informed consent Exclusion Criteria * Patients with comorbidities such as liver dysfunction * Married patients * Lactating mothers * History of thrombosis and fits

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'study participants were divided into 2 groups. one was treated with combination therapy that includes thalidomide and hydroxyurea whereas another group was treated with thalidomide alone.', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 30, 'type': 'ESTIMATED'}}

Updated at

2024-07-08

1 organization

2 products

3 indications

Organization

National Institute of Blood and Marrow Transplant

Product

Thalidomide + Hydroxyurea

Indication

Indication

Indication

Product