View clinical trials related to Thalassemia.
Filter by:The purpose of this study is to examine choroidal thickness in beta-thalassemia patients and compare it to the one of healthy controls. An equal number of transfusion dependent beta-thalassemic patients and age and sex matched healthy volunteers will undergo spectral-domain optical coherence tomography utilizing the enhanced depth imaging application to visualize and measure the choroid.
In this study will investigate long term safety and use of the PTG-300 in Beta Thalassemia patients.
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
1. Primary objectives: - To study the tolerance and safety of multiple oral administration of CN128 in patients with thalassemia aged 16 years and above. - To study the pharmacokinetics of CN128 in thalassemia patients aged 16 and above by multiple oral administrations of CN128 2. Design: The study is designed as a safety, tolerability and pharmacokinetic parameters study, phase Ib trial. The study is consisted of: multiple dose tolerance and safety study; multiple administration pharmacokinetics. 3. Subject inclusion criteria: - Thalassemia patients with serum ferritin ≥ 500 µg/L - Patients aged 16 and above - HB≥80 g/L before administration - Voluntarily participate in the experiment, and the process of obtaining informed consent met the requirements of GCP. 4. Subject exclusion criteria: - Hepatitis B surface antigen positive, hepatitis B core antibody positive and HBV-DNA positive, hepatitis C anti-HCV positive, HIV positive, Treponema pallidum positive - History of active digestive tract diseases (including gastric ulcer, duodenal ulcer, gastroesophageal varices, ulcerative colitis, Crohn's disease, digestive tract tumors, familial genetic polyps), history of digestive tract perforation, history of digestive tract surgery and influence on drug absorption, and other investigators believe that patients with potential intestinal complications - Liver dysfunction (ALT or AST > 2.5×ULN); or renal dysfunction (serum creatinine > 1.5×ULN) - Uncontrolled active infections - Patients currently taking CYP3A strong inducer or inhibitor drugs or drugs that may prolong the QT interval without temporary suspension of use or temporary substitution of the said drugs - ect. 5. Usage: All subjects fasted prior to administration of study drug using 240 ml warm water. The people can not drink water within 1h before administration. 6. Pharmacokinetic assessment of CN128 administration: PK parameters of CN128 include AUC 0-t, AUC 0-∞, Cmax, Tmax, t1/2, CL/F, Vd/ F, MRT, λz, Css-av, Css-min, Css-max, Accumulation rate, Fluctuation index, etc. 7. Safety and tolerability assessments: Evaluation was based on the incidence rate of adverse events (AE) after the administration, study termination information, vital signs, physical examination, laboratory tests and ECG. 8. Statistics
Thalassemia becomes one of global health issue and so does Indonesia. In 2015, more than 7600 children were diagnosed as this hemoglobin genetic disease wherein anemia and lifetime blood transfusion contribute to their morbidity and mortality in Indonesia. Major β-Thalassemia is the most common type found. However, along with disease progression and age, iron accumulation and dysregulation becomes the most common complication exist. In cellular level, this condition results in cell and tissue damage especially immune cells and promotes favor condition for siderophilic bacteria such as Mycobaterium tuberculosis (Mtb) to growth rapidly. Severe infection becomes the second most cause of death in thalassemia-β major patients. Tuberculosis (Tb) remains the global health issue especially in developing countries. Based on World Health Organization (WHO) report on 2015, Indonesia is the second highest burden of TB in the world. Both of adaptive and innate immune system plays important role in Mtb recognition and eradication. However, immune cells mechanism and activity in response to Mtb infection during iron accumulation condition on thalassemia-β major patients may be altered therefore need for further study. Macrophage is an adaptive immune cell, has a pivotal role on circulating-iron regulation and serves as Mtb host cell. To understand macrophage activity on thalassemia-β major patients can be studied by monocyte characteristic stimulated by Mtb antigen and evaluated by its differentiation into three subsets based on CD14 and CD16. Mtb antigen presentation is identified by HLA-DR expression on monocyte membrane. Vitamin D is one of the most affected micronutrients on major β-thalassemia patients, yet it has immunomodulatory effect on immune system. Recent finding of vitamin D receptor (VDR) expressed in monocyte strongly convince that vitamin D should be maintained in major β-thalassemia patients where it is found lower in these patients. Thus, this original and true report aimed to declare that the research activity has finished and the data has been elaborated. Future plan is developing the original article based on the research finding corroborating the previous knowledge and innovative suggestion for the quality of thalassemia.
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.