View clinical trials related to Beta-Thalassemia.
Filter by:The aim of the study is to: - Assess the pattern of glucose homeostasis in patients with B thalassemia . - To detect early impairment in glucose metabolism and prediabetic state in B thalassemia patients using continuous glucose monitoring system. - To study the prevalence and type of DM in B thalassemia patients. - A comparative study of standard insulin therapy compared to insulin pump therapy in thalassemic diabetic patients will be done. The study will include screening of 200 children and adolescents who are regularly attending the Hematology Oncology Clinic and fulfilling the inclusion criteria for abnormalities of glucose homeostasis. A pilot study will be done on 15 patients with abnormal glucose tolerance which will include: A-Continuous glucose monitoring system (CGMS) : A glucometer will be given to each patient and will be asked to measure blood glucose before meals and snacks and record the valus in the CGMS for better calibration . B-Therapeutic intervention: Thalassemia patients who proved to have diabetes according to the ADA criteria will be subjected to • Insulin pump will be tried in each diabetic thalassemic patient versus conventional insulin therapy.
This study is looking at the effects of giving early treatment of deferiprone to young children with beta thalassemia who have started receiving regular blood transfusions but have not yet reached the criteria for starting on iron chelation therapy. Half the patients in the study will receive deferiprone, and the other half will receive placebo, for up to 12 months.
This is a single-arm, multi-site, single-dose, Phase 1/2 study to assess ST-400 in 6 subjects with transfusion-dependent β-thalassemia (TDT) who are ≥18 and ≤40 years of age. ST-400 is a type of investigational therapy that consists of gene edited cells. ST-400 is composed of the patient's own blood stem cells which are genetically modified in the laboratory using Sangamo's zinc finger nuclease (ZFN) technology to disrupt a precise and specific sequence of the enhancer of the BCL11A gene (which normally suppresses fetal hemoglobin production in erythrocytes). This process is intended to boost fetal hemoglobin (HbF), which can substitute for reduced or absent adult (defective) hemoglobin. ST-400 is then infused back into the patient after receiving conditioning chemotherapy to make room for the new cells in the bone marrow, with the aim of producing new erythrocytes with increased amounts of HbF. The primary objective is to understand safety and tolerability of ST-400, and secondary objectives are to assess the effects on HbF levels and transfusion requirements.
The study aims to evaluate the effect of local non surgical periodontal therapy on the systemic pro-inflammatory markers in the β-thalassemia (TM-β) patients with chronic periodontitis and systemically healthy demographically matched controls with chronic periodontitis. Both groups will receive non surgical periodontal therapy.
This proof-of-mechanism study is being performed to investigate the safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of multiple oral doses of bitopertin in adults with NTD beta-thalassemia. This study consists of two parts: Part 1 - The main study - 16 weeks in total: Participants will undergo a 6-week dose-escalation period followed by 10 weeks of treatment at the attained target dose. Part 2 - Open Label Extension (OLE) - up to an additional 12 months. Participants will be given the option to enroll into the OLE once the 16-week treatment of Part 1 has been completed. Participants who decide not to enroll in the OLE, at the end of Part 1 will enter a 6-week follow-up period.
This 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.
Every year, 100,000 neonates are born with hemoglobinopathies around the world. Thalassemia is the most common heterogeneous disease of the human being . It is a disease of high prevalence in Mediterranean, Indian, North Chinese, and Pacific populations. Recently, the quantity and quality of the life of these patients have been significantly improved by regular transfusion and iron chelating therapy .
Thalassemia is a blood disorder passed down through families in which the body makes an abnormal form of hemoglobin. There are 2 main types of thalassemia; Alpha & Beta thalassemia. Alpha thalassemia: occurs when a gene or genes related to the alpha globin protein are missing or mutated. Beta-thalassemia syndromes are a group of hereditary blood disorders characterized by reduced or absent beta globin chain synthesis. Beta-thalassemias can be classified into: Silent carrier: completely asymptomatic with normal hematological parameters. Beta-thalassaemia minor (beta-thalassaemia trait): usually asymptomatic; diagnosis is made during a work-up for mild anemia. Beta-thalassaemia intermedia: usually a similar presentation to beta-thalassaemia major; symptoms are usually less pronounced and the course is usually more insidious. Beta-thalassaemia major : In which there is complete absence of hemoglobin A
The study will investigate the relation between erythrocyte glutamine/glutamate ratio and pulmonary hypertension risk in Egyptian thalassemic children in Assiut University Children Hospital
Randomized controlled trial was conducted in post-splenectomy patients aged >12 years. Subjects are randomly assigned to two groups (zinc and placebo). 1.5 mg/kg/day (max 50 mg/day) of Zinc is administered.