View clinical trials related to Beta-Thalassemia.
Filter by:The purpose of this research study is to evaluate the safety of two doses of FBS0701, a new oral iron chelator, and its effectiveness in clearing iron from the liver. FBS0701 is a medication taken by mouth that causes the body to get rid of iron. Iron chelators are used in patients with β-thalassemia and other forms of anemia who experience iron overload - iron increases in the body as a result of regularly required blood transfusions. Patients who qualify will be randomized to receive one of two doses of FBS0701 for up to 24 weeks (6 months) with a total study duration of up to 33 weeks. These patients will be eligible to participate in a dosing extension for up to 72 weeks. The maximum duration of dosing will be up to 96 weeks. The safety of patients will be monitored frequently during the study by physical exams, ECGs, and blood tests. To assess the amount of iron in the liver and heart, each patient must undergo 6 MRI scans during the study. Patients will not need to stay in the hospital for this study but will need to visit the outpatient clinic up to 28 times over the 96 week period. Patients currently taking an iron chelator will be required to stop for a total of up to 26 weeks. The results of this study will help to determine if FBS0701 may be effective as an iron chelator.
An Italian Multicentric randomized, open-label therapeutic trial evaluating the efficacy and safety of Neridronate in the treatment of Osteoporosis in patients with Thalassemia Major and Severe Thalassemia Intermedia. Efficacy and safety of the drug will be evaluated measuring at every visit this parameters: - haematological: Haemochrome - blood chemistry: creatinine, BUN, AST, ALT, Ca, P, proteins electrophoresis, total proteins. The prevalence of ectopic calcification and pseudoxantoma elasticum (PXE)-like syndrome and their follow-up will be evaluated at the beginning of the study vs 24 months through physical examination, abdominal echography and fundus oculi examination. During the trial other known risks factors for osteoporosis will be recorded, including prevalence and incidence of bone fractures and, if executed, Polimorphisms COLIA1. At the beginning of the study and at months 12 and 24 morphometry DXA will be performed to evaluate of the presence of bone deformities. Furthermore data regarding QOL and symptom pain will be evaluated trough administration of scale SF-36. At 12 months an intratrial analisis will be performed on efficacy and safety parameters in order to introduce possible amendments to the study design and to decide the prosecution of the trial During the trial all adverse events will be recorded
Background: - Thalassemia major (TM) is a chronic disorder that affects a person s ability to produce hemoglobin, resulting in anemia. Hemoglobin is a component of red blood cells that carries oxygen and nutrients to cells in the body. As a result, individuals require life-long blood transfusions and extensive medical management. Studies have shown that because of its demanding nature, TM might negatively affect an individual s quality of life, sense of self, and social integration, but little is known about affected individuals overall experiences with and perceptions of TM. - TM is caused by a genetic change in the thalassemia gene. The disease is passed to children by parents who carry one copy of the altered thalassemia gene. The parents are called carriers of the condition and have a 25 percent chance of having a child with TM. It is possible to screen for carriers of TM and use this information for pregnancy planning and management. - TM is common among people from South and South East Asia and is an important public health concern in Singapore. More research is needed to explore the lives of people with TM, and the societal perceptions that exist in Singapore about TM. Objectives: - To describe the familial, social, and professional experiences of individuals with TM. - To investigate the social messages being given out about TM in Singapore and the sources of those messages. - To explore the impact of these experiences, perceptions, and social messages on individuals who have TM. - To explore how the experiences and perceptions of individuals who have TM affect their life, sense of self, social integration, and compliance with medical treatment. Eligibility: - Residents of Singapore who are 14 years of age or older, can speak English, and currently have TM. - Parents of individuals with TM who are 14 years of age or older. Parents must be 21 years of age or older, be able to speak English, and have had caregiving responsibilities for their child at some point. Design: - All participants will have a one-time semi-structured interview, followed by a questionnaire to obtain demographic information. - Interviews will be conducted in Singapore and are expected to last for 30 to 90 minutes. - Individuals with TM will be asked about their own perceptions of TM; familial, social, and professional experiences involving TM; and their perceptions of others views and of social messages related to TM. - Parents of individuals with TM will be asked about their experiences in caring for a child with TM, talking to their child about TM, telling people about their child s TM, and interacting with health care providers.
This study aims to investigate the use of amlodipine, a drug that blocks the uptake of calcium into cells, in the prevention and treatment of iron overload in patients with thalassemia major. Since iron uses the same calcium channels to enter the heart, pancreas and other organs, blocking these channels might help to prevent the accumulation of iron in these tissues. The study will follow 10 patients with thalassemia major: 5 will openly receive amlodipine and 5 will serve as controls, not receiving any additional drugs. Patients will be monitored through one year with an additional year of follow up after the group using amlodipine stops its use. Monitoring will occur through the measurement of blood ferritin as well as live and heart T2* by MRI.
CD34+ stem cell selection in children, adolescents and young adults receiving partially matched family donor or matched unrelated adult donor allogeneic bone marrow or peripheral blood stem cell transplant will be safe and well tolerated and be associated with a low incidence of serious (Grade III/IV) acute and chronic graft versus host disease (GVHD).
The purpose of this research study is to study the safety of increasing doses of FBS0701, and to see how quickly the study medication is absorbed and how quickly it disappears from the bloodstream. FBS0701 is a new, oral iron chelator - a medication taken by mouth that increases the body's elimination of iron. Iron chelators are used in patients who develop iron overload from their transfusions. Four increasing doses of FBS0701 will be tested during this study. The study will start with the lowest dose given to 4 patients (3 mg/kg/day. The next group of 4 patients will receive the next high dose (8mg/kg/day only after the results of the first 4 patients are examined and it is determined safe to continue. Participating patients will take the study medication for 7 days and be followed for 28 days after their last dose to determine if they have any reactions to the study medication - therefore a total of 35 days on study. Patients will need to give up to 17 blood samples over the screening period and first 15 days of the study (a total of about 9 tablespoons). Patients will not need to stay overnight in the clinic but will need to visit the clinic 10 times for screening and on-study visits over the 35 days. Patients currently taking an iron chelator will need to stop that treatment for up to 22 days (up to 5 days before they start the study and for 15 days during the study). The results of this study will be helpful in determining the safety of the drug and the best doses of FBS0701 to be used in the next study which will assess the effectiveness of this new iron chelator.
This study will assess the safety and efficacy of deferasirox in patients with cardiac MRI T2* < 20 msec.
The 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 .
Myocardial iron overload is the leading cause of death in patients with beta-thalassemia major (TM). Therapy with deferoxamine (DFO) combined with deferiprone (DFP) reduces myocardial iron and improves cardiac function. However, the prognosis for TM patients with established cardiac disease switched from DFO monotherapy to combined DFP/DFO chelation is unknown. Twenty-eight TM patients with cardiac disease were enrolled in a prospective study lasting 42±6 months. Fifteen (9 high-ferritin and 6 low-ferritin) were placed on DFP/DFO (DFP, 75 mg/kg t.i.d.; DFO, 40-50 mg/kg over 8-12 h at night 5-7 d/wk), while 13 (5 high- and 8 low-ferritin) received DFO alone. No cardiac events were observed among high-ferritin patients on combination therapy, whereas 4 cardiac events (p=0.0049), including three deaths, occurred in high-ferritin patients on DFO monotherapy. These findings demonstrate that in TM patients with well-established cardiac disease combined iron-chelation therapy with DFP/DFO is superior to DFO monotherapy.
The purpose of this study is to evaluate the safety and tolerability of HQK-1001 administered daily for 8 weeks in subjects with beta thalassemia intermedia