Active clinical trials for "Thalassemia"

This is a prospective, single-arm, open-label study. Twenty adult patients with transfusion-dependent β -thalassemia will be enrolled to receive Luspatercept with optimal supportive care, including blood transfusion and iron removal, based on the clinician's judgment and practice. The main objective of this study was to evaluate the efficacy and safety of Luspatercept in the treatment of adult patients with transfusion-dependent β -thalassaemia in Chinese clinical practice, and to provide evidence reference for subsequent clinical use.

The goal of this clinical trial is to learn about SP-420 ability to remove iron from organs in subjects with transfusion-dependent β-thalassemia. The main questions it aims to answer are: How efficient is SP-420 in cleaning iron from the liver? How is the safety and tolerability of ascending doses of SP-420? Participants will: Take medication three time weekly Attend up to 20 site visits Undergo MRI scans

The purpose of this study is to evaluate the efficacy, safety and pharmacokinetics of luspatercept plus best supportive care (BSC) versus placebo plus BSC in participants who require regular red blood cell transfusions due to β-thalassemia.

This is a non-randomized, open label, single-site, single-dose, phase 1 study in up to 5 participants (between 5 and 35 years of age, inclusive) with Transfusion-dependent α-thalassemia. The study will evaluate the safety and efficacy of autologous hematopoietic stem cell transplantation (HSCT) using GMCN-508A Drug Product [autologous CD34+ hematopoietic stem cells transduced with GMCN-508A lentiviral vector encoding the human α-globin gene].

The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 in treating β-thalassemia.

This study compares L-arginine Versus Sildenafil as treatment for pulmonary hypertension in Children with Beta Thalassemia

In β-thalassaemia and Sickle Cell Disease (SCD), a significant production of fetal haemoglobin (HbF) may reduce the severity of clinical course and reactivation of γ-globin gene expression in adulthood. HbF induction is one of the best strategies to ameliorate the characteristic symptoms of these diseases. Hydroxyurea (HU) is the only medication, approved by the US Food and Drug Administration, inducing HbF. However, treatments with HU induce sufficient HbF levels in only half of the patients, and side effects including leukopenia and neutropenia are frequently reported. Therefore, novel therapeutic inducers must be identified to develop a personalized treatment in β-thalassaemia and sickle cell anaemia. The availability of new treatments depends on drugs already approved for other indications, and on pharmacokinetics and pharmacovigilance already assessed. Rapamycin (as Sirolimus) is an immunosuppressant agent, approved by the FDA for acute rejection prevention in renal transplant recipients. The ability of this drug to induce γ-globin gene expression in erythroleukemia cell line and erythroid precursors cells (ErPCs) in ß-thalassaemia patients is already known. A clinical investigation on the effects of sirolimus in ß-Thalassaemia aims to evaluate several parameters related to red blood cell status and HbF levels and is a first step for the full clinical development in this new indication.

This is an open label, multi-center study to evaluate the safety and Efficacy of ET-01 Transplantation in subjects with Transfusion Dependent β-Thalassaemia.

This is an open label study to evaluate the safety and efficacy of α-globin Restored Autologous Hematopoietic Stem Cells in α-Thalassemia Major Patients

This is an open label study to evaluate the safety and efficacy of β-globin Restored Autologous Hematopoietic Stem Cells in ß-Thalassemia Major Patients