Deciphering Effects of Thalidomide on Red Blood Cells in Transfusion Dependents Beta Thalassemia Patients: A Pharmacodynamics and Pharmacogenetics Analysis

Dir/KMU-EB/TC/000863

The goal of this Non-Randomized Clinical Trial is to determine the effects of thalidomide on red blood cells in transfusion dependent beta thalassemia patients. The main aims of this study are: * To determine the therapeutic effect of Thalidomide on hemoglobin. * To analyze association of different β- globin mutations with response to thalidomide in β-thalassemia patients. * To analyze association of Single Nucleotide Polymorphisms (SNPS) of HBG2, BCL11A and HBS1L-MYB with response to thalidomide in β-thalassemia patients. * To correlate GATA1 and KLF1 gene expression with response to thalidomide in β-thalassemia patients. Patients will be grouped into thalidomide and non-thalidomide groups on the basis of their willingness to receive thalidomide therapy. Thalidomide will be given at an average dose of 1.5mg/kg/day (range 1-2mg/kg/day). Patients will be followed up for 12 months and data will be collected at different visits. After 12 months of thalidomide therapy patients will be divided into responders and non-responders for comparative analyses on the basis of increase in hemoglobin level.

2022-01-25

2023-07-20

2023-10-31

Completed

Early phase I

200

Inclusion Criteria: * Diagnosed transfusion dependent β-thalassemia patients * Possess a verified pre transfusion Hb electrophoresis report performed at age≥ 6 months Exclusion Criteria: * Patients with active metabolic or systemic comorbidities * Patients with autologous antibodies, AIHA or hypersplenism

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'Patients will be grouped into thalidomide and non-thalidomide groups on the basis of their willingness to receive thalidomide therapy. Thalidomide will be given at an average dose of 1.5mg/kg/day (range 1-2mg/kg/day). Patients will be followed up for 12 months and data will be collected at different visits.', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE', 'maskingDescription': 'No masking'}}, 'enrollmentInfo': {'count': 200, 'type': 'ACTUAL'}}

2023-11-24