Combination Therapy of Hydroxyurea With L-Carnitine and Magnesium Chloride in Thalassemia Intermedia...
β-Thalassemia IntermediaThe purpose of this study is determination of the efficacy of combination of hydroxyurea with L-carnitine or magnesium in improving hematologic parameters and cardiac status in patients with β-thalassemia intermedia in comparison with hydroxyurea alone .
A 4-year Extension Study to Core 1-year Study of Iron Chelation Therapy With Deferasirox in β-thalassemia...
Transfusional Iron Overloadβ-thalassemia Major1 moreIn this 4-year extension study the safety, efficacy and and pharmacokinetics of deferasirox in regularly transfused pediatric patients with β-thalassemia major was assessed. Patients who successfully completed the main 1 year trial (NCT00390858) were eligible to continue in this extension trial and receive chelation therapy with deferasirox for up to 4 years.
Extension Study of the Efficacy and Safety of Deferasirox Treatment in Beta-thalassemia Patients...
Beta-thalassemia MajorHemosiderosis2 moreTo allow patients treated with deferasirox in the core study to continue iron chelation therapy for 2 years or until the drug became locally commercially available. To evaluate the long-term safety and efficacy of deferasirox by measuring treatment success, change in liver iron content (LIC) and change in serum ferritin levels. Safety was mainly assessed by incidence of adverse events (AEs)and clinically significant lab parameters.
Iron Balance Study of DFO and GT56-252 in Patients With Transfusional Iron Overload Secondary to...
Beta-ThalassemiaA clinical trial designed to compare the safety and iron excretion properties of desferoxamine (DFO) and deferitrin (GT56-252), an experimental oral iron chelator.
Chelation Therapy of Iron Overload With Pyridoxal Isonicotinoyl Hydrazone
Anemia (Iron-Loading)Beta-Thalassemia4 moreTo demonstrate the safety and effectiveness of orally-administered pyridoxal isonicotinoyl hydrazone (PIH) for the chronic treatment of iron overload.
A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants...
Beta-ThalassemiaThis is a single-arm, multi-site, single-dose, Phase 3 study in approximately 18 participants less than or equal to (<=) 50 years of age with transfusion-dependent β-thalassemia (TDT), who have a β0/β0, β0/IVS-I-110, or IVS-I-110/IVS-I-110 genotype. The study will evaluate the efficacy and safety of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin BB305 Drug Product.
Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD) and Preliminary Efficacy of VIT-2763...
Beta-ThalassemiaNon-transfusion-dependent ThalassemiaThis is a randomised, double-blind, placebo-controlled parallel group trial to investigate the safety, tolerability and efficacy of multiple doses of VIT-2763 versus placebo in participants with non-transfusion-dependent Beta-thalassemia (NTDT).
Hydroxy Urea, Omega 3, Nigella Sativa,Honey on Oxidative Stress and Iron Chelation in Pediatric...
Iron OverloadOxidative Stress1 moreThe aim of the present study is evaluating the strength of combination therapy of hydroxy urea, omega 3, nigella sativa and honey on antioxidant-oxidant status (OXIDATIVE STRESS) in response to reactive oxygen species production (LIPID PEROXIDATION) and their effect on iron intoxication (IRON CHELATION) in pediatric major thalassemia.
Thrombin Generation in Beta-thalassemia Major
Beta Thalassemia Major AnemiaThis 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
Impact of the Preparation Method of Red Cell Concentrates on Transfusion Indices in Thalassemic...
Thalassemia MajorThis study compares the effects of Packed Red Blood Cells (PRBCs) prepared in two different ways on the transfusion indices in beta(ß)-Thalassemia transfusion-dependent patients. The two blood components types derive from the whole blood. In one case, the whole blood is leukoreduced with subsequent plasma removal. In the other case, plasma, buffy coat, and red blood cells (RBCs) are first separated and subsequently, the RBCs leukoreduced. Each type of blood components will be subsequently given to one-half of the patients for a 6-month period and to the other half for other 6-month at the randomization phase, for a total of 12 months of crossed-treatment per patient.