Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia
NCT Number: NCT06490627
Acronym: BTM
Status: RECRUITING
Study Type: INTERVENTIONAL
Phases: PHASE2
Conditions: Fetal Hemoglobin|Thalassemia Major|Genetic Disease
Interventions: DRUG: Thalidomide|DRUG: Thalidomide
Sponsor: National Institute of Blood and Marrow Transplant (NIBMT), Pakistan
Sex: ALL
Age: CHILD, ADULT
Enrollment: 54
Study Design: Allocation: RANDOMIZED|Intervention Model: PARALLEL|Masking: NONE|Primary Purpose: TREATMENT
Start Date: 2024-04-22
Completion Date: 2026-04-22
Locations: National Institute of blood disease and bone marrow transplant, Karachi, Sindh, Pakistan

Summary:
The project "Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia" investigates the safety and efficacy of low-dose thalidomide in managing beta thalassemia, a genetic disorder causing anemia. Conducted over two years at NIBD hospital, the study involves 54 transfusion-dependent patients aged 8-35. The primary objective is to correlate thalidomide doses with disease severity, adverse effects, and treatment response, aiming to optimize treatment strategies and reduce side effects.

Data will be collected through clinical interviews and medical record reviews and analyzed using SPSS. Key variables include hemoglobin levels, leukocyte and reticulocyte counts, platelets, liver and spleen size, genetic modifiers, and transfusion frequency. Inclusion criteria are specific to beta thalassemia patients, while exclusion criteria rule out those with liver dysfunction, married patients, lactating mothers, and those with a history of thrombosis or fits.

Primary Outcome Measures: transfusion dependency, Transfusion frequency will be assessed by recording the number of blood transfusions a patient receives over a specified period, such as weekly, monthly, or annually. This data will be collected from patient medical records and/or transfusion logs, ensuring accurate tracking of each transfusion event, 1 year
Secondary Outcome Measures: hemoglobin levels., Hemoglobin levels will be assessed using a complete blood count (CBC) test, measured in grams per deciliter (g/dL) of blood. This test is conducted through a venous blood sample, which is then analyzed using an automated hematology analyzer to determine the hemoglobin concentration., 2 years|total leukocyte count., Leukocyte count will be assessed using a complete blood count (CBC) test, measured in thousands of cells per microliter (thousand cells/µL) of blood. This test involves analyzing a venous blood sample with an automated hematology analyzer to determine the total number of white blood cells present., 2 years|reticulocyte count, Reticulocyte count will be assessed using a complete blood count (CBC) test, measured as a percentage of the total red blood cells or as an absolute number per microliter (µL) of blood. This test involves analyzing a venous blood sample with an automated hematology analyzer, which identifies and quantifies reticulocytes using specific staining techniques., 2 years|Platelets, Platelet count will be assessed using a complete blood count (CBC) test, measured in thousands of cells per microliter (thousand cells/µL) of blood. This test involves analyzing a venous blood sample with an automated hematology analyzer to determine the number of platelets., 2 years|impact of genetic modifiers (e.g., HBB mutation, XMN polymorphism, BCL11A polymorphism, and co-inheritance of alpha chain) on the treatment outcomes with thalidomide., Genetic modifiers will be assessed through genetic testing E.g thalassemia genetic profile, focusing on specific genes or genetic variations known to influence the expression or function of proteins related to the condition under study. This analysis helps identify how genetic factors may modify disease progression, treatment response, or other relevant outcomes., 2 years|Spleen Size, Spleen size will be assessed using imaging techniques such as ultrasound, computed tomography (CT) scan, or magnetic resonance imaging (MRI). Measurements will be reported in centimeters (cm)., 2 years|Serum Glutamate Pyruvate Transaminase,, Serum Glutamate Pyruvate Transaminase, also known as alanine aminotransferase (ALT), will be assessed through a blood test, measured in units per liter (U/L). This test analyzes a venous blood sample to determine the concentration of ALT, an enzyme primarily found in the liver cells., 2 years|Serum Ferritin Levels, Serum ferritin levels will be assessed using a blood test, measured in nanograms per milliliter (ng/mL). A venous blood sample will be analyzed in a clinical laboratory using immunoassay techniques to determine the concentration of ferritin, providing an indicator of the body's iron stores., 2 years|lactate dehydrogenase, Lactate dehydrogenase (LDH) levels will be assessed through a blood test, measured in units per liter (U/L). This test analyzes a venous blood sample to determine the concentration of LDH, an enzyme involved in cellular metabolism. Elevated LDH levels may indicate tissue damage or disease,, 2 years|Nucleated red blood cells, Nucleated red blood cells (NRBCs) will be assessed using a complete blood count (CBC) test with a differential, measured as the number of NRBCs per 100 white blood cells (WBCs) or as an absolute number per microliter (µL) of blood. This test involves analyzing a venous blood sample with an automated hematology analyzer, which can differentiate and count NRBCs to provide insights into bone marrow function and potential hematologic disorders., 2 years|D-dimer, D-dimer levels will be assessed through a blood test, typically measured in nanograms per milliliter (ng/mL) or micrograms per liter (µg/L). This test analyzes a venous blood sample to determine the concentration of D-dimer, a fibrin degradation product., 2 years|Total bilirubin, Total bilirubin (TBIL) levels will be assessed through a blood test, measured in milligrams per deciliter (mg/dL). This test analyzes a venous blood sample to determine the total concentration of bilirubin, including both direct (conjugated) and indirect (unconjugated) bilirubin., 2 years|Indirect bilirubin, Indirect bilirubin (IBIL) levels will be assessed through a blood test, measured in milligrams per deciliter (mg/dL). This test analyzes a venous blood sample to determine the concentration of unconjugated bilirubin, which is a byproduct of red blood cell breakdown before it is processed by the liver., 2 years|Fibroscan, FibroScan will be used to assess liver stiffness and steatosis, which can be indicative of liver iron overload. Elevated serum ferritin levels may suggest iron accumulation in the liver, and FibroScan provides a non-invasive method to evaluate the extent of liver fibrosis and fat content., 2 years|T2 star imaging, T2\* (T2 star) magnetic resonance imaging (MRI) will be used to assess liver iron concentration in relation to serum ferritin levels. This imaging technique measures the rate of signal decay in liver tissue, which is inversely correlated with iron content. A shorter T2\* value indicates higher iron concentration. T2\* MRI provides a non-invasive, quantitative assessment of liver iron overload., 2 years|transfusion dependency, Transfusion frequency will be assessed by recording the number of blood transfusions a patient receives over a specified period, such as weekly, monthly, or annually. This data will be collected from patient medical records and/or transfusion logs, ensuring accurate tracking of each transfusion event, 2 year
Other Outcome Measures: Safety Profile:, The safety profile will be assessed by monitoring and documenting adverse events, side effects, and any other safety-related outcomes throughout the study period. This includes recording and analyzing data on patient-reported symptoms, clinical observations, laboratory test results (including those for the parameters previously discussed like liver enzymes, kidney function tests, etc.), and any other relevant safety assessments specified in the study protocol., 2 years