View clinical trials related to Thalassemia.
Filter by:Thalassemic patients often suffer from iron overload due to frequent blood transfusion. Oral iron chelators reduce iron overload in transfusion dependent patients. The aim of this study is to compare the efficacy and safety of osveral and desferal in transfusional iron overload patients with β-Thalassemia and intermediate Thalassemia in Bandarabbas.
Fludarabine is a chemotherapy drug used extensively in bone marrow transplantation. The goal of this study is to determine what causes some children to have different drug concentrations of fludarabine in their bodies and if drug levels are related to whether or not a child experiences severe side-effects during their bone marrow transplant. The hypothesis is that clinical and genetic factors cause changes in fludarabine drug levels in pediatric bone marrow transplant patients and that high levels may cause severe side-effects.
The results will allow us to evaluate the role of MP in the thrombo-embolic risk observed in thalassemic patients and to underline a possible difference between TM and TI. The in vitro and in vivo study of MP in erythrocytes concentrates is a new approach to explore the consequence of transfusion in polytransfused patients. Finally, the identification of a possible relationship between the oxidative stress and the production of MP may lead to the development of specific therapeutical approaches
This study will evaluate the efficacy and safety of deferasirox in patients with MDS, thalassemia and rare anemia patients with transfusion iron overload.
The proposed research project will continue the application and development of a new method (biomagnetic susceptometry) that measures magnetic fields to determine how much iron is in the liver. The amount of iron in the liver is the best indicator of the amount of iron in the whole body. Measuring the amount of iron in the body is important because either too much (iron overload) or too little iron (iron deficiency) can be harmful. At present, the most reliable way to measure the amount of iron in the liver is to remove a sample of the liver by biopsy, either by surgery or by using a needle which pierces the skin and liver. Iron stored in the liver can be magnetized to a small degree when placed in a magnetic field. In patients with iron overload, the investigators previous studies have shown that magnetic measurements of liver iron in patients with iron overload are quantitatively equivalent to biochemical determinations on tissue obtained by biopsy. In the past the investigators have developed a device to measure the amount of magnetization, which was called a SQUID (Superconducting QUantum Interference Device) susceptometer. This device was validated and in use for over 20 years. The safety, ease, rapidity and comfort of magnetic measurements make frequent, serial studies technically feasible and practically acceptable to patients. The investigators have now developed a new susceptometer, which uses very similar technology to the SQUID, but the investigators believe is more accurate and precise. This study aims to validate this new instrument. The investigators will do prospective, serial studies of the diagnosis and management of patients with iron overload, including thalassemia major (Cooley's anemia), sickle cell disease, aplastic anemia, myelodysplasia, hereditary hemochromatosis, and other disorders. Funding Source - FDA OOPD.
The purpose of this study is to study the efficacy of Traditional Chinese Medicine (TCM) on anaemic and cytopenic haematological disorders including myelodysplastic syndrome (MDS), aplastic anaemia (AA), myelofibrosis (MF) and thalassemia intermedia who do not have or did not respond to available treatment options.
Thalassemia is considered the most common genetic disorder worldwide, occurring with high frequency in Mediterranean areas, the Middle East, Southeast Asia, and the Pacific Islands. Currently, the only cure for thalassemia is bone marrow transplantation from a related, compatible donor. Gene transfer, achieved by transplantation of the patient's own blood stem cells that have been genetically-modified with the corrected gene, could potentially cure thalassemia. The first step in developing gene transfer for treatment of thalassemia is to develop a safe and effective method to obtain blood stem cells from thalassemia patients. Eventually, high numbers of genetically modified cells will need to be infused into the patient for clinical gene transfer to be effective. The blood stem cells are obtained by giving a "mobilization" agent to the patients. This causes the stem cells to leave the bone marrow and go into the blood. The purpose of this study is to test the safety and effectiveness of the new mobilization agent, Mozobil, in causing mobilization of blood stem cells for patients with beta-thalassemia major.
The purpose of this research study is to evaluate the safety of two doses of FBS0701, a new oral iron chelator, and its effectiveness in clearing iron from the liver. FBS0701 is a medication taken by mouth that causes the body to get rid of iron. Iron chelators are used in patients with β-thalassemia and other forms of anemia who experience iron overload - iron increases in the body as a result of regularly required blood transfusions. Patients who qualify will be randomized to receive one of two doses of FBS0701 for up to 24 weeks (6 months) with a total study duration of up to 33 weeks. These patients will be eligible to participate in a dosing extension for up to 72 weeks. The maximum duration of dosing will be up to 96 weeks. The safety of patients will be monitored frequently during the study by physical exams, ECGs, and blood tests. To assess the amount of iron in the liver and heart, each patient must undergo 6 MRI scans during the study. Patients will not need to stay in the hospital for this study but will need to visit the outpatient clinic up to 28 times over the 96 week period. Patients currently taking an iron chelator will be required to stop for a total of up to 26 weeks. The results of this study will help to determine if FBS0701 may be effective as an iron chelator.
The purpose of this research is to evaluate the effects of L-glutamine as a therapy for Sickle Cell Anemia or Sickle ß0 Thalassemia as evaluated by the number of occurrences of sickle cell crises.
Iron overload, which can be defined operationally as too much iron in the body, develops as a consequence of too many blood transfusions given, or due to genetic defects hereditary hemochromatosis). Iron accumulates in several organs in the body, such as the heart, liver, endocrine glands (pancreas, thyroid, etc.), and spleen. Excessive iron can damage organs and may even cause death. Iron overload needs to be appropriately monitored and treated to avoid unnecessary morbidity and mortality. The present study, GENIOS, proposes to test prospectively the hypothesis that genetic modifiers influence the iron overload status of patients receiving transfusions. To test this hypothesis, the study will perform genetic studies to investigate possible genetic influences for iron accumulation in the body and will study iron accumulation not only in the liver, but also in the heart, pancreas, kidneys, and spleen. In addition: the study will investigate if these same genes have any role during treatment of iron overload, in other words, if certain genetic mutations will influence how iron exits the body. This study will also investigate how substances that are known to control the trafficking of iron in and out of the body and its damaging effects to the tissues (hepcidin and non transferrin-bound iron) are linked to the accumulation of iron in the heart and liver. Iron in the body will be measured by R2*MRI and no liver biopsies will be required. Genetic studies will be done by specialized tests using peripheral blood DNA. Iron accumulates differently in different people and in different organs of the body. Some people accumulate iron faster than others, even when receiving the same number of blood transfusions