View clinical trials related to Beta-Thalassemia.
Filter by:Observe long-term safety risk and long-term efficacy after intravenous infusion of BHC001 in TDT subjects.
This is an interventional study to evaluate the safety and efficacy of autologous Hematopoietic Stem Cells (HSCs) transduced with lentiviral vector encoding functional hemoglobin subunit beta (HBB) gene in patients with transfusion-dependent beta-thalassemia.
This is a phase II, randomized, double-blinded, placebo-controlled study to treat patients with transfusion-dependent and non-transfusion dependent β -thalassemia with AND017 and optimal supportive care, including blood transfusion and iron removal, based on the clinician's judgment and practice.
Observe long-term safety risk and long-term efficacy after intravenous infusion of BRL-101 in TDT subjects.
The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 in treating patients with β-thalassemia major anemia.
This is a non-randomized, open label, single-dose study in up to 41 participants with β-thalassemia major. The goal of this clinical trial is to evaluate the safety and efficacy of KL003 cell injection in subjects with β-thalassemia major.
The main aim of this study is to collect real-world longitudinal data on participants with β-thalassemia treated with betibeglogene autotemcel (beti-cel) in the post marketing setting. To assess the long-term safety, including the risk of newly diagnosed malignancies, after treatment with beti-cel and evaluate the long-term effectiveness of treatment with beti-cel.
The goal of this study is to compare the effectiveness and safety of two hemoglobin F inducer. This is single centered interventional pilot study is to compare the efficacy and safety parameters in beta thalassemia patients. As this is a pilot study, the investigator took a small number of patients. The Sample size was calculated by the World health organization sample size calculator. After screening 39 patients and 24 patients were eligible for enrollment in this study. The main objective was to evaluate safety of both drugs in genetic disorder like thalassemia. for safety evaluation, hematological parameters were evaluated that includes total bilirubin , indirect bilirubin, Serum Glutamate Pyruvate Transaminase, serum glutamic-oxaloacetic transaminase, urea, creatinine and lactate dehydrogenase were monitored . Moreover to evaluate the efficacy of drug, hematological parameters that includes hemoglobin, red blood cells , nucleated red blood cells , reticulocytes count, Red blood cells indices ( mean corpuscular hemoglobin, mean corpuscular volume and mean corpuscular hemoglobin concentration) , white blood cells and platelets were done. Another important parameters to evaluate the efficacy of hemoglobin F inducer is transfusion frequency. Test were done at baseline and after completion of study means after 06 months.
This is a non-randomized, open-label, single-dose study. The aim of this study is to evaluate the safety and efficacy of the treatment with lentiviral vector encoding βA-T87Q-globin gene transduced autologous hematopoietic stem cells transfusion in subjects with β-thalassemia major.
To assesse the efficacy and safety of luspatercept versus placebo in China patients with transfusion-dependent β-thalassaemia.