NCT06490601

Long Term Beta Thalassemia Treatment: Findings From The Extension Period

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

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 30 (estimated)
• Sponsor: National Institute of Blood and Marrow Transplant (NIBMT), Pakistan · Other Government
• Sex: All
• Age: 8 Years – 35 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

Study titled Long Term Beta Thalassemia Treatment: Findings From The Extension Period Of Phase 2 Clinical Trial, conducted at the National Institute of Blood Disease \& Bone Marrow Transplantation (NIBD \& BMT). This study focuses on the long-term comparison of combination therapy (thalidomide and hydroxyurea) versus thalidomide alone in treating beta thalassemia. The objective is to evaluate the efficacy and safety of the combination therapy compared to thalidomide alone, with the hypothesis that the combination will be more effective. Beta thalassemia is defined as an autosomal recessive disorder affecting beta-globin production, influenced by genetic modifiers. Key variables include hemoglobin, red blood cells, leukocyte count, reticulocyte count, platelets, lactate dehydrogenase, nucleated red blood cells, ferritin, bilirubin, Serum Glutamate Pyruvate Transaminase, creatinine, transfusion frequency, spleen and liver size, hemoglobin subunit beta \[ Homo sapiens (human) \] mutation, and certain polymorphism in gamma globin gene . The study took place at NIBD hospital over three years, designed as a Phase 2 single-center, two-arm open-label interventional study with a sample size of 30 participants using simple randomized sampling. Inclusion criteria are beta thalassemia major/intermediate patients aged 8-35 years, while exclusion criteria include patients with liver dysfunction, married patients, lactating mothers, and those with a history of thrombosis and fits. Data will be collected through clinical interviews and medical record reviews and analyzed using (Statistical Package for the Social Sciences.

Conditions

• Fetal Hemoglobin
• Beta-Thalassemia
• Hemoglobinopathies

Interventions

Type	Name	Description
DRUG	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.
DRUG	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.

Timeline

• Start date: 2022-04-01
• Primary completion: 2024-04-01
• Completion: 2025-07-01
• First posted: 2024-07-08
• Last updated: 2025-05-30

Locations

1 site across 1 country: Pakistan