View clinical trials related to Beta-Thalassemia.
Filter by:This is a Phase I/II clinical trial of gene transfer for treating Beta-thalassemia using a self-inactivating lentiviral vector to functionally correct the defective gene(s). The objectives are to evaluate the safety and efficacy of the gene transfer clinical protocol.
This is a single group, open label study in 10 subjects who are 8 years of age or older with beta-thalassemia major. The objective of this study is to evaluate the safety and efficacy of autologous hematopoietic stem cell transduced with lentiviral vector for the treatment of beta-thalassemia major.
A prospective randomized study on Safety, Tolerability and Efficacy of oral Low dose DFP (50 mg/kg/day) in minimally transfused B-TM after 5 transfusions when SF reaches 500 ng/m and with either appearance of LPI > 0.2 or TSAT reaches 50% compared with non treatment arm. So the aim of this study: 1. To determine the time as well as amount of transfused iron ( calculated in mg iron/kg ) which lead to Serum ferritin reaches 500 ng /ml and LPI appearance >0.2 as well as TSAT reaches 50 % . 2. Tolerability and safety of early low dose DFP 50mg/kg and effectiveness to postpone or prevent SF from reaching 1000 ng/ml or LPI >0.6 or TSAT >70% in comparison to patients not starting chelation therapy 3. Determine adverse events whether drug or non drug related
role of Vit C to Augment iron chelation with DFP or DFX in thalassemic patients.
safety and efficacy of different iron chelation therapy in transfusion dependent beta-thalaasemia patients with low serum ferritin and continued regular transfusion regimen.
The study hypothesis that treatment with Erythropoietin (EPO) combined with Human Erythropoietin (HUO) therapy will result in hematologic improvement in thalassemia intermedia patients. Second is to determine whether any of the following correlate with improved hematologic response: A decrease in hemolysis, as assayed by a decrease in LDH, compared to baseline levels,baseline Erythropoietin levels,baseline hemoglobin levels and baseline reticulocyte counts (or % circulating nucleated erythroblasts/100 WBCs). Goal: The aim is to assess the possibility of steady increase of hemoglobin levels in thalassemia intermedia patients by at least 1g/dl above baseline levels during therapy using Hydroxyurea and Erythropoietin, growth evaluation,quality of life (QoL) and decline transfusion requirements during study period. Also to report and compare adverse events with other published data regarding.
Interventional Allocation: Randomized Endpoint Classification: Safety/Efficacy Study of combined chelation therapy Masking: Open Label Primary Purpose: Treatment of transfusional iron overload Primary Outcome Measures: • The primary outcome measure is to assess efficacy in lowering serum ferritin level(the change in serum ferritin compared to baseline) with combining DFP and deferasirox compared to combined DFP and DFO in conditions with severe chronic iron overload; showing an up-trend of SF over previous 12 months on single chelator. Secondary Outcome Measures: • The secondary outcome measure is to determine the number of patients who will develop adverse events in order to assess safety upon administering the drugs in combination (DFP and DFX) compared to the combination of DFO and DFP.
The purpose of this pilot study is to determine the effect of various doses of vitamin D supplementation on vitamin D stores and calcium excretion in the urine in subjects with Thalassemia Major (TM). Subjects with TM are routinely placed on vitamin D supplements because they frequently have osteoporosis (a condition in which bone tissue thins and loses density and strength) and low vitamin D stores. The amount of vitamin D supplementation that is required to raise vitamin D stores in optimal levels is not known in TM, and will be determined in this study. Finally, a recent study in TM has linked blood vitamin D levels to urine calcium excretion, which is a risk factor for kidney stones. Therefore, we want to determine changes in calcium excretion with various vitamin D doses and with increasing vitamin D stores. We plan to test 3 doses of vitamin D for 3 months in children and adults with TM. Changes in vitamin D blood levels and urinary calcium will be determined. The results of this pilot study will be used in future studies that will examine the effect of various doses of vitamin D supplementation in the treatment of osteoporosis in TM.
Patients with thalassemia intermedia, congenital dyserythropoietic anemia type I , and sideroblastic anemia were found to express very high levels of serum GDF15, and this contributed to the inappropriate suppression of hepcidin with subsequent secondary iron overload.The aim of our present study is to asses the levels of GDF15 and hepcidin in patients with Sickle cell disease and hereditary spherocytosis
Sickle cell nephropathy is a known complication of sickle cell anemia (SCA) manifested by increase in glomerular filtration rate (glomerular hyperfiltration) and results in proteinuria and chronic renal failure. Our goal is to examine the prevalence of proteinuria and microalbuminuria as an early predictive factor of glomerular injury, among young people who suffer from SCA as well as those who suffer from combined sickle cell/beta-thalassemia.