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NCT06931912
There are nearly 300,000 patients with severe or intermediate thalassemia in China. Growth retardation is the most significant health issue for children and adolescents with transfusion-dependent thalassemia (TDT), placing a substantial economic burden on their families and a serious social strain on the labor force. Investigating the growth and development of these children and adolescents, and establishing targeted intervention plans, holds significant social value for public health practice. 1. To screen and identify pediatric patients with growth problems by conducting growth and development assessments in high-incidence areas of China, including physical development, endocrine function, nutritional status, brain function and lifestyle behaviors. 2. Implement the MENBS clinical interventions for pediatric patients with growth problems, concentrating on the following areas: * Monitor: Continuously monitor health-related indicators through regular follow-up. * Education: Provide health education to improve the cognition of patients and their families. * Nutrition: Assess patients' nutritional risks and develop personalized diet plans. * Behavior: Recommend appropriate exercise plans to promote physical development. * Support: Conduct home visits, offer free clinics and establish a support network. 3. Repeat growth assessment for pediatric patients with growth problems after 1-year clinical interventions. 4. Evaluate the effectiveness of MENBS interventions by comparing changes in growth and development indicators.
NCT05444894
The purpose of this study is to evaluate the safety, tolerability, and efficacy of treatment with EDIT-301 in adult participants with Transfusion Dependent beta Thalassemia
NCT06831799
Patients with red blood cell disorders (RBCDs), such as Sickle cell disease (SCD) and Thalassemia, are chronic, life-threatening conditions that can become multi-organ complications over time, and are likely at an increased risk of COVID-19-related complications. Patients at the highest risk include the elderly (\>50 in our population), those with a history of respiratory or cardiac disease and those with other comorbidities. Several patients affected by RBCDs undergo splenectomy as therapeutic option to improve their level of hemoglobin concentration. Splenectomized patients, or in the case of SCD with functional hyposplenism, are more vulnerable to bacterial infections / superinfections after viral infection. Acute pulmonary syndrome (ACS) is the main cause of morbidity in SCD in middle-high income countries, and is often triggered by infectious events. Currently, there is no literature on the subject. Thus, any recommendation available comes from the experience gained with previous Coronaviruses infections. Accordingly, the correct treatment and management of infection by Coronavirus SARS-COV-2 (COVID-19) in patients affected by RBCDs may be challenging given the rapid spread of the pandemic and limited literature so far, especially in some countries. Accordingly, there is an urgent need to pool evidence in a unique repository on patients affected by RBCDs and COVID-19 in order to reach critical numbers to facilitate the medical decision making process across Europe. The Registry on patients with rare RBCDs and COVID-19 is an initiative conceived in the core of the European Reference Network on Rare Hematological Diseases (ERN-EuroBloodNet, FPA 739541, www.eurobloodnet.eu) aiming at supporting medical practice of COVID-19 in these patients by gathering evidence on pediatric and adult COVID-19 confirmed cases in RBCDs across Europe.
NCT05883254
1. To study the expression pattern of PUM1 gene in patients with sickle cell anemia and β-thalassemia intermedia. 2. To detect PUM1 protein levels in sickle cell anemia and β-thalassemia intermedia patients. 3. To correlate PUM1 gene expression pattern and protein levels with HbF levels in sickle cell anemia and β-thalassemia intermedia patients.
NCT03993613
The aim of the trial is to study the effect of apotransferrin administration in patients suffering from β-thalassemia intermedia in order to restore the erythropoiesis as reflected by enhanced haemoglobin levels or reduced transfusion dependency.
NCT01496963
This is a multicenter observational case-control analysis lasting 12 months aimed at determining the prevalence of pulmonary hypertension (PAH) in patients with Thalassemia Major and Intermedia. The patients will be followed, treated and examined according to the best standard clinical practice dictated by the Italian Society for the study of Hemoglobinopathies (SITE), Thalassemia International Federation (TIF)and the Task Force for Diagnosis and Treatment of Pulmonary Hypertension of European Society of Cardiology (ESC); European Respiratory Society (ERS); International Society of Heart and Lung Transplantation (ISHLT) guidelines.
NCT01224496
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.
NCT01642758
Beta thalassemia intermedia syndromes are genetic anemias caused by mutations which reduce production of beta globin, a major component of adult hemoglobin A, the protein which delivers oxygen throughout the body. Patients suffer from poor growth, fatigue, heart failure, endocrine deficiencies, and eventually, many require chronic blood transfusions. There is no approved therapeutic for the deficiency of beta globin chains in beta thalassemia. This trial will study an oral therapeutic which stimulates production of fetal globin, an alternate type which is produced by all humans, but is normally switched off in infancy. This type of globin can compensate for the missing protein in beta thalassemia.
NCT01624038
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.