View clinical trials related to Thalassemia.
Filter by:The production of Hb F after birth is an important factor in modifying the clinical severity of beta thalassemia because an increased gamma-globin level will bind the additional a-globin and form Hb F. The objective of this project is to evaluate the association of Hb F level with phenotypic diversity of patients with beta thalassemia.
This is an international prospective registry of patients with Alpha thalassemia to understand the natural history of the disease and the outcomes of fetal therapies, with the overall goal of improving the prenatal management of patients with Alpha thalassemia.
This clinical trial studies the effect of pre-transplant immunosuppression (PTIS) and donor stem cell transplant in treating patients with severe blood diseases (hemoglobinopathies). PTIS helps prepare the body for the transplant and lowers the risk of developing graft versus host disease (GVHD). Hematopoietic cells are found in the bone marrow and produce blood cells. Hematopoietic cell transplantation (HCT) injects healthy hematopoietic cells into the body to support blood cell production. PTIS and HCT may help to control severe hemoglobinopathies.
The primary purpose of this study is to compare the effect of mitapivat versus placebo on transfusion burden in participants with transfusion-dependent alpha- or beta-thalassemia (TDT).
The primary purpose of this study is to compare the effect of mitapivat versus placebo on anemia in participants with alpha- or beta-non-transfusion dependent thalassemia (NTDT).
This study will investigate the safety and tolerability of SLN124 in patients with Thalassaemia or patients with Very Low- and Low-risk Myelodysplastic Syndrome (MDS) after single ascending s.c. doses and multiple doses in healthy male and female subjects. Up to 7 cohorts of 56 patients with Thalassaemia and up to 7 cohorts of 56 patients with MDS will be enrolled. Each subject will receive single or multiple doses of SLN124 or placebo given by subcutaneous (s.c) injection.
There is still an ongoing lack of awareness about thalassemia among Indonesians despite high disease prevalence, and this is associated with poor implementation of preventive and promotive measures. Thalassemia experts argue that education and raising awareness about thalassemia must be done from an early age in order to relieve the heavy disease burden of thalassemia, especially in Indonesia as it is geographically located in the thalassemia belt. In spite of this, there has been limited research on public perception towards thalassemia in Indonesia, particularly among young people. In this study, the researcher will ask young people in Indonesia between the age of 15-24 who are currently attending high school or university-level education to fill in an online survey. The survey consists of questions about the participants' demographic information as well as their knowledge, attitude, and practice (KAP) scores towards thalassemia. The study is expected to run from December 2020 to March 2021, with the online survey distributed in January 2021. Data collection will immediately follow, and it will be analyzed using univariate, bivariate, and multivariate analysis using the appropriate statistical tests. The findings gathered from this study will be vital in providing new insight into how the youth sees thalassemia in Indonesia. Such public health information will prove to be important in designing successful thalassemia intervention and education programs for Indonesian youth.
The primary objective of this study is to better understand factors contributing to variations in hydroxyurea (HU) adherence behavior in adolescents and young adults (AYA) with sickle cell disease (SCD). To meet this objective, the researchers will conduct a prospective cohort study to determine the longitudinal relationship between HU adherence and health-related quality of life (HRQOL) overtime among AYA with SCD. The long-term goal of this research is to promote medication adherence behavior and improve health outcomes in AYA with SCD.
The primary objective of this study is to evaluate a potential behavioral intervention (MED-Go app). To meet this objective, the researchers will conduct a pilot randomized controlled trial to test the feasibility and acceptability of MED-Go app in adolescents and young adults (AYA) with sickle cell disease (SCD). The long-term goal of this research is to promote medication adherence behavior and improve health outcomes in AYA with SCD.
The overarching goal of this proposal is to identify modifiable behavioral strategies based on patient-reported outcomes (PROs) and health-related quality of life (HRQOL) that will improve hydroxyurea (HU) adherence among adolescents and young adults with sickle cell disease (SCD). In this proposed study, we intend to test the functionality of a PROs-toolbox feature, which will be integrated into our existing smartphone application platform (SCD-app), over a 24-week period in a cohort of SCD patients and their caregivers.