View clinical trials related to Beta-Thalassemia.
Filter by:This is a single-dose, open-label study in pediatric participants with TDT. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).
To assess the effect of different risk factors on the growth parameters of thalassemic patients in Assiut University children Hospital (AUCH) In order to help in decreasing the morbidity and mortality resulting from iron overload and improving the quality of life for thalassemic patient
Primary Objectives: Long-term safety of BIVV003 in participants with severe sickle cell disease (SCD) and ST- 400 in participants with transfusion-dependent beta-thalassemia (TDT) Secondary Objectives: - Long-term efficacy of the biological treatment effect of BIVV003 in SCD - Long-term efficacy of the clinical treatment effect of BIVV003 on SCD-related clinical events - Long-term efficacy of the biological treatment effect of ST-400 in TDT - Long-term efficacy of the clinical treatment effect of ST-400 in TDT
- To design an amplification-refractory mutation system (ARMS) for the DNA diagnosis of the IVS I-110 (G>A) [HBB:c.93-21G˃A] mutation. - To detect the prevelence of the mutation among Assiut University Hospital patients. - Phenotype/genotype correlation of the mutation.
Objectives Primary objective: • To determine the efficacy and safety of the combination therapy of Hydroxyurea and thalidomide in beta-thalassemia patients. Secondary objective: • To determine the change in liver and spleen size of beta-thalassemia patients on the combination therapy A single-arm non-randomized trial to evaluate the efficacy and safety of combination therapy of hydroxyurea and thalidomide in beta-thalassemia patients. It was a twelve months study. Participants were monitored for six months on Hydroxyurea alone and then the combination therapy of hydroxyurea and thalidomide for another six months. Findings of physical examination, vital signs, laboratory, and ultrasound findings were recorded at baseline, during and end of the study. Sample Size and Population This study included 135 Beta-thalassemia patients.
Multiple studies showed the negative impact thalassemia disease and its therapy have on the cognition of these patients. This study aims to assess and compare the cognition, psychological impact and quality of life among patients with B thalassemia major, requiring regular blood transfusion, patients with B thalassemia intermedia, who don't, and healthy controls. Thus, estimating the role of blood transfusion and hemosiderosis in cognitive impairment, psychological symptoms, and low quality of life in these patients.
This is a Phase 1,open label,safety,and efficacy study in subjects with non-β0/β0 TDT β-thalassemia Major by transplanting BD211 drug product which is for autologous use only,via a single IV administration.
This is a non-interventional post-authorization safety study (PASS) employing a cross sectional design to evaluate the effectiveness of the additional risk minimization measures (aRMMs) for REBLOZYL. A survey will be used to measure the knowledge and comprehension of the REBLOZYL aRMMs among European Economic Area (EEA) based healthcare professionals (HCPs). The PASS will be conducted among HCPs in a representative sample of EEA countries where REBLOZYL is commercially available, potentially including Austria, Germany, Italy, Norway, Sweden, the Netherlands, Poland, and Spain. Additional EEA countries may be included, as needed, based on commercial availability and reimbursement status.
This study aims to evaluate the persistence of a hypercoagulable state in chronically-transfused patients with beta thalassemia major, by using the thrombin generation test (performed in whole blood and plasma). Patients will be compared with 2 control groups: 1/ healthy volunteers and 2/ carriers of beta-thalassemia trait
To assess the possible role of iron overload as a cause of liver dysfunction in thalassemic childrens receiving multiple blood transfusion and its correlation with serum aminotransferases.