View clinical trials related to Beta-Thalassemia.
Filter by:This study compares the effects of Packed Red Blood Cells (PRBCs) prepared in two different ways on the transfusion indices in beta(ß)-Thalassemia transfusion-dependent patients. The two blood components types derive from the whole blood. In one case, the whole blood is leukoreduced with subsequent plasma removal. In the other case, plasma, buffy coat, and red blood cells (RBCs) are first separated and subsequently, the RBCs leukoreduced. Each type of blood components will be subsequently given to one-half of the patients for a 6-month period and to the other half for other 6-month at the randomization phase, for a total of 12 months of crossed-treatment per patient.
Patients with transfusion dependent Beta Thalassemia suffer from a high incidence of Hepatitis C infection especially in developed countries as Egypt. In our patients we also found a high correlation between hepatitic C infection and Liver fibrosis. in this study we offer our patients treatment with Direct antiviral drugs and assessed the degree of fibrosis before and after treatment. We tested Hyalornic acid as a predictor of the degree of fibrosis before and after treatment.
The accumulation of unpaired α-globin chains in β-thalassemia major patients may clinically create ineffective erythropoiesis, hemolysis, and chronic anemia. Multiple blood transfusions and iron overload cause cellular oxidative damage. However, α-tocopherol, an antioxidant, has been known as a potent scavenger of lipid radicals in the red cell membrane of β-thalassemia major patient. By this randomized controlled trial, the investigators would like to evaluate the effects of α-tocopherol in hemolysis and oxidative stress on the red cell membrane of β-thalassemia major.
The aim of this study was to determine the anthropometric measurements in transfusion-dependent β-thalassemia children in Pakistan. The secondary aim was to correlate serum ferritin with the physical growth
It is known that postprandial hyperglycemia increases the cardiometabolic risk in both diabetic and non-diabetic patients. Moreover, there is insufficient data on the effectiveness of exercise on preventing Type II diabetes mellitus in individuals with insulin resistance and prediabetes. This study aims to examine the effectiveness of resistance exercise in limiting postprandial hyperglycemia and the necessity of prescribing medication particularly in patients with beta-thalassemia and insulin resistance.
Beta-thalassemias are hereditary blood disorders caused by reduced or absent synthesis of hemoglobin beta chains, with variable outcomes ranging from severe anemia to clinically asymptomatic individuals. Treatment is symptomatic and thalassemia is a major unmet medical need. Survival is increased, even in patients needing transfusions, in comparison with a few years ago, but the quality of life is poor for many patients. In some patients, an anomalous expression of gamma-globin genes has been observed, with a consequent rise in Fetal Hemoglobin levels. The patients displaying a clinical phenotype known as Hereditary Persistence of Fetal Hemoglobin (HPFH) exhibit a positive clinical status. To mimick HPFH, several compounds able to induce expression of fetal hemoglobins (HbF) have been evaluated. Within this framework, sirolimus is particularly interesting as an inducer of HbF. It has been used for many years for different indications and the available preclinical evidence warrant the start of a clinical development plan in thalassemia. The investigators propose a clinical trial in beta-thalassemia patients, designed to evaluate the effect of sirolimus on several parameters related to red blood cell status and to the level of HbF in particular, as a first step for the full clinical development in this new indication.
Beta-thalassemia represents a group of recessive inherited hemoglobin disorders characterized by reduced synthesis of β-globin chain. The homozygous state (β-thalassemia major) "TM" results in severe anemia, which needs regular blood transfusion . The life expectancy in patients with TM has increased due to therapeutically management, such as frequent transfusion, desferal administration and bone marrow transplantation. Diabetes is clinically characterized by hyperglycemia due to either low circulating concentrations of, or decreased sensitivity to, insulin. Patients with TM typically exhibit β-cell or insulin insufficiency, and may develop diabetes due to toxic levels of iron in their pancreas, one of the strongest predictors of β-cell destruction. By contrast, hyperinsulinemia, secondary to insulin resistance, with normal glucose tolerance has also been observed. The pathogenic mechanisms leading from siderosis to diabetes are poorly understood.
This is a Phase 2 open-label, single-arm design study with dose escalation by subject cohort . The study is designed to monitor the PTG-300 safety profile, to obtain preliminary evidence of efficacy of PTG-300 for the treatment in β-thalassemia.
An open label, safety and pharmacokinetic study of oral hydroxyurea solution administered to children from 6 months to 17.99 years (i.e. to the day before 18th birthday), with a 12 to 15 month treatment period for each participant. The study treatment duration will be for 6 months at the maximum tolerated dose [MTD], which is usually reached by 6 months after initiation of treatment. For patients in whom time to MTD is longer than 6 months or not achieved at all, the maximum duration of study treatment will be 15 months.
This study is a multicenter, open-label, two-period crossover design that evaluates the safety, tolerability, pharmacokinetics and preliminary evidence of iron chelating activity of DST-0509 as compared to Jadenu and Exjade in transfusion-dependent thalassemia patients with transfusional iron overload, requiring iron chelation therapy and demonstrating an inadequate response to Jadenu or Exjade for greater than 3 months duration. Up to 36 patients will be evaluated (18 in each treatment arm), however, the balanced randomization may enroll fewer patients based on recruitment status.