Long Term Beta Thalassemia Treatment: Findings From The Extension Period
NCT Number: NCT06490601
Acronym: Thalidomide
Status: ACTIVE_NOT_RECRUITING
Study Type: INTERVENTIONAL
Phases: PHASE2
Conditions: Fetal Hemoglobin|Beta-Thalassemia|Hemoglobinopathies
Interventions: DRUG: thalidomide and hydroxyurea|DRUG: Thalidomide
Sponsor: National Institute of Blood and Marrow Transplant (NIBMT), Pakistan
Sex: ALL
Age: CHILD, ADULT
Enrollment: 30
Study Design: Allocation: RANDOMIZED|Intervention Model: PARALLEL|Masking: NONE|Primary Purpose: TREATMENT
Start Date: 2022-04-01
Completion Date: 2025-04-01
Locations: National Institute of blood disease and bone marrow transplant, Karachi, Sindh, Pakistan

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.

Primary Outcome Measures: hemoglobin levels, Measure the improvement in 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., 3 years|red blood cell count., Measure the improvement in red blood cell count. Red blood cell count will be assessed using a complete blood count (CBC) test, measured in millions of cells per microliter (million cells/µL) of blood. This test is conducted through a venous blood sample, analyzed using an automated hematology analyzer to determine the number of red blood cells present., 3 years|leukocyte count, Measure the effect on in 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., 3 years|reticulocyte count, Measure the effect on 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., 3 years|Transfusion Frequency:, Document and compare the frequency of blood transfusions required by patients in both treatment arms over the study period.

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., 3 years
Secondary Outcome Measures: Spleen and Liver Size, Measure changes in spleen and liver size as a response to the treatments. Spleen and liver size will be assessed using imaging techniques such as ultrasound.. Measurements will be reported in centimeters (cm), capturing the dimensions of each organ to evaluate any enlargement or abnormalities., 3 years|Serum Ferritin Levels, Evaluate the effectiveness of the combination therapy in reducing serum ferritin levels, indicating decreased iron overload.

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., 3 years|Genetic Modifiers:, Analyze the influence of genetic modifiers. 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., 3 years|bilirubin, Monitor and compare changes in biochemical parameters such as bilirubin. Bilirubin levels will be assessed through a blood test, typically measured in milligrams per deciliter (mg/dL). This test analyzes a venous blood sample to determine the concentration of bilirubin, a pigment produced from the breakdown of red blood cells, providing insights into liver function and potential health conditions such as jaundice or liver disease., 3 years|lactate dehydrogenase., Monitor and compare changes in biochemical parameters such as 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., 3 years|Serum Glutamate Pyruvate Transaminase,, Monitor and compare changes in biochemical parameters such as 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. Elevated ALT levels may indicate liver damage or disease., 3 years|creatinine, Monitor and compare changes in biochemical parameters such as creatinine. Creatinine levels will be assessed through a blood test, typically measured in milligrams per deciliter (mg/dL) or micromoles per liter (µmol/L). This test involves analyzing a venous blood sample to determine the concentration of creatinine, a waste product from muscle metabolism filtered by the kidneys. Monitoring creatinine levels helps evaluate kidney function and detect conditions such as kidney disease or impaired renal function., 3 years
Other Outcome Measures: Safety Profile:, Assess the safety and tolerability of the combination therapy versus thalidomide alone by monitoring adverse effects and clinical complications.

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., 3 years