Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo...
Transfusion-dependent Beta-ThalassemiaThis is a multi-center, long-term safety and efficacy follow-up study for subjects with transfusion-dependent β-thalassemia (TDT) who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored parent clinical studies. After completing the parent clinical study (approximately 2 years), eligible subjects will be followed for an additional 13 years for a total of 15 years post-drug product infusion. No investigational drug product will be administered in the study.
Safety and Pharmacokinetic Study of Escalating Doses of SP-420, an Iron Chelator, in Patients With...
Iron OverloadBeta-ThalassemiaThe purpose of this study is to assess safety and amount of the study drug in the blood after increasing doses of SP-420. The study will be conducted in patients with β-thalassemia.
Denosumab Versus Zoledronic Acid in Thalassemia-Induced Osteoporosis
Thalassemia Majors (Beta-Thalassemia Major)OsteoporosisThis study is to compare the two medications Denosumab and Zoledronic Acid For Patients With Beta Thalassemia Major Induced Osteoporosis. Patients with B-thalassemia major induce osteoporosis will undergo baseline assessment of the bone densitometry by Dual-energy X-ray absorptiometry scan as a standard of care by the radiology department, then a blood test for bone specific Alkaline phosphatase and type-1 Carboxy Telopeptide will be measured by the chemistry lab. Patients with B-Thalassemia Major induced osteoporosis, who are 18 years of age or more and willing to participate in the study will be enrolled after consenting by the primary investigator in hematology outpatient clinic. Patients with osteoporosis will receive one of the two medications, at the end of the year Dual-energy X-ray absorptiometry scan will be done to compare the response of the two medications. The potential risks include the drug-related side effects
β-globin Restored Autologous HSC in β-thalassemia Major Patients
β-thalassemia MajorThis is a single center, single arm, open-label study to determine the safety and efficacy of β-globin restored autologous hematopoietic stem cells in β- thalassemia major patients with CVS-654 mutation.
A Study of IMR-687 in Subjects With Beta Thalassemia
β ThalassemiaA Study to Evaluate the Safety and Tolerability of IMR-687 in Subjects with Beta Thalassemia
Combination Therapy Compared With Single-Drug Therapy in Patients With Cardiac Diseases
Cardiovascular DiseasesHeart Diseases1 moreThe purpose of this study is to determine whether left ventricular function improves more rapidly with deferoxamine (DFO) and deferiprone (L1) combination therapy than with DFO monotherapy in patients with thalassemia and decreased ejection fractions. Secondary aims include evaluating changes in myocardial iron burden using T2* and estimating the relative incidence and severity of chelator-induced toxicity.
Apotransferrin in Patients With β-thalassemia
β-thalassemia IntermediaThe aim of the trial is to study the effect of apotransferrin administration in patients suffering from β-thalassemia intermedia in order to restore the erythropoiesis as reflected by enhanced haemoglobin levels or reduced transfusion dependency.
Safety and Efficacy of Deferasirox in Combination With Desferoxamine in β-thalassaemia Patients...
Transfusion-dependent β-thalassemia PatientsCardiac Iron OverloadThe primary efficacy endpoint of this interventional study was to evaluate the number of patients achieving a complete response (CR), defined as patients switching from intensive deferasirox -DFO treatment, at any time point during the 24 months of study, to deferasirox monotherapy based on improvement in the cardiac magnetic resonance imaging (MRI) T2* value to >10ms, and continue to maintain their MRI T2* to values >10 msec.
Stem Cell Transplantation After Reduced-Dose Chemotherapy for Patients With Sickle Cell Disease...
HemoglobinopathiesAnemia4 moreThe purpose of this study is to find out if using a lower dose of chemotherapy before stem cell transplantation can cure patients of sickle cell anemia or thalassemia while causing fewer severe side effects than conventional high dose chemotherapy with transplantation.
HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity
Sickle Cell DiseaseBeta Thalassemia-MajorThis study will evaluate the use of reduced intensity conditioning regimen in patients with high risk hemoglobinopathy Sickle Cell and B-Thalassemia Major in combination with standard immunosuppressive medications, followed by a routine stem cell transplant in order to assess whether or not it is as effective as myeloablative high dose chemotherapy and transplant.