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Clinical Trials for beta-Thalassemia

• Clinical Observation of Luspatercept in Treatment of Chinese Adult β-thalassaemia Patients With TD β-thalassemia
• Thalidomide and Hydroxyurea Combination in β-Thalassemia Patients
• Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
• NAC Effect on Iron Overload and Blood Transfusion in β-thalassemia Major
• A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)
• A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia
• Clinical Study to Evaluate the Possible Efficacy and Safety of L- Carnitine and Sildenafil in Children Having Beta Thalassemia With Increased Tricuspid Regurgitant Jet Velocity
• Safety and Efficacy Evaluation of BRL-101 in Subjects With Transfusion-Dependent β-Thalassemia
• A Study to Evaluate Luspatercept (ACE-536) in Chinese Participants Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia.
• The Safety and Efficiency of Luspatercept in Chinese Adults With Transfusion Dependent β-thalassemia: a Real-world Study
• Addition of JSP191 (C-kit Antibody) to Non-myeloablative Hematopoietic Cell Transplantation For Sickle Cell Disease and Beta-Thalassemia
• Clinical Experience of Thalidomide in Thalassemic Patients
• Efficacy and Safety Study of Multiple Doses of VIT-2763 in Adults With Transfusion-dependent Beta-thalassemia
• Pre-transplant Immunosuppression and Donor Stem Cell Transplant for the Treatment of Severe Hemoglobinopathies
• A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
• A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
• A Study Investigate the Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Response of SLN124 in Adults With Alpha/Beta-thalassaemia and Very Low- and Low-risk Myelodysplastic Syndrome
• The BENeFiTS Trial in Beta Thalassemia Intermedia
• A Study of IMR-687 in Subjects With Beta Thalassemia
• Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD) and Preliminary Efficacy of VIT-2763 in β-thalassaemia
• PK of SOF/LED in HCV - Infected Adolescents With Haematological Disorders
• Hydroxy Urea, Omega 3, Nigella Sativa,Honey on Oxidative Stress and Iron Chelation in Pediatric Major Thalassemia
• The Effect of N-acetylcysteine on Oxidative Stress Status and Iron Overload in Thalassemia Major
• Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients (THALA-RAP)
• Study in Beta-thalassaemia or Myelodysplastic Syndrome Patients to Investigate the Safety and Tolerability of SLN124
• Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants Who Require Regular RBC Transfusions Due to Beta (β)-Thalassemia.
• AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion
• Phase 2a Pilot Study of NBMI Treatment in Patients With Beta Thalassemia Major, Requiring Iron Chelation
• A Study to Evaluate Long-term Safety in Subjects Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials
• Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS TMPRSS6-LRx
• Safety Study for Beta Thalassemia Subjects on PTG-300
• Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study
• Hyalornic Acid Level in β-Thalassemic Children Treated for Hepatitis C Virus
• The Effect of Alpha-tocopherol in Hemolysis and Oxidative Stress Marker on the Red Cell Membrane Beta-thalassemia Major
• Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients
• Zinc Supplementation in Patients With β-Thalassemia Major Complicated With Diabetes Mellitus
• Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects With Chronic Anemia
• SP-420 in Subjects With Transfusion-dependent Beta-Thalassemia or Other Rare Anemias
• Pharmacokinetics of Oral Hydroxyurea Solution
• the Efficacy and Safety of Sugammadex in Children 0-2 Years Old
• T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias
• Pilot Study to Assess the Safety, PK and Iron Chelating Activity of DST-0509 (Deferasirox) in Thalassemia Patients Refractory to Chelation
• Safety and Efficacy of Early Treatment With Deferiprone in Infants and Young Children
• L-arginine Versus Sildenafil in Children With Beta Thalassemia Associated With Pulmonary Hypertension
• A Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Patients With Transfusion-Dependent Beta Thalassemia
• Periodontal Therapy in Thalassemia Major Patients
• A Study of Bitopertin (RO4917838) in Adults With Non-Transfusion-Dependent (NTD) Beta-Thalassemia
• The Efficacy and Safety of HYDROXYUREA in Management of Beta Thalassemia Patients in Karachi Pakistan
• Monitoring of Chimerism After Transplantation in Patients With β Thalassemia Major and the Treatment Strategies for the Reduction of Chimerism
• Denosumab Versus Zoledronic Acid in Thalassemia-Induced Osteoporosis
• The Potential Hepatoprotective Effect of Metformin in Patients With Beta Thalassemia Major
• A Study of EPEG in Beta Thalassemia Patients
• Benefits of Nigella Sativa in Children With Beta Thalassemia Major
• Study of The Therapeutic Benefits of Al-hijamah in Children With Beta Thalassemia Major
• Effect of Spirulina Compared to Amlodipine on Cardiac Iron Overload in Children With Beta Thalassemia
• An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia
• Denosumab vs Placebo in Patients With Thalassemia Major and Osteoporosis
• Amlodipine as Adjuvant Treatment to Iron Chelation for Prevention of Cardiac Iron Overload in Thalassemia Patients
• HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity
• Efficacy of Basiliximab in the Prevention of Acute Graft-versus-host Disease in Unrelated Allogeneic Hematopoietic Stem Cell Transplantation Therapy for Thalassemia Major
• Safety and Pharmacokinetic Study of Escalating Doses of SP-420, an Iron Chelator, in Patients With β-Thalassemia
• ACE-536 Extension Study - Beta Thalassemia
• Clinical Trial of Deferasirox Combination Treatment With Deferiprone In Thalassaemia Patients
• Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies
• An Algorithm to Start Iron Chelation in Minimally Transfused Young Beta-thalassemia Major Patients
• A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease
• Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure
• Role of Vitamin C to Augment Iron Chelation With DFP or DFX
• Study of Efficacy and Safety of INC424 in Regularly Transfused Patients With Thalassemia.
• Efficacy and Safety of Efficacy and Safety of Continued Iron Chelation Therapy In Poly-transfused Thalassemia Patients With Low Serum Ferritin (< 500 ng/ml)
• Treatment of Iron Overload Requiring Chelation Therapy
• Bone Marrow Transplant With Abatacept for Non-Malignant Diseases
• Effect of Metoprolol on Thalassemia Cardiomyopathy
• Immunomodulatory Effects of Silymarin in Patients With Beta-Thalassemia Major
• Study to Evaluate the Effects of ACE-536 in Patients With Beta-thalassemia
• An Open Label Study to Evaluate the Pharmacokinetics, Safety, Tolerability and Efficacy of Deferasirox Administered to Chinese Patients With β-thalassemia Major Aged From 2 to Less Than 6 Years Old
• Combination Deferasirox and Deferiprone for Severe Iron Overload in Thalassemia
• Trial of HQK-1001 in Beta Thalassemia Intermedia in Lebanon
• Therapeutic Effect and Safety of Combined Hydroxyurea With Recombinant Human Erythropoietin.
• Post Hematopoietic Stem Cell Transplantation
• Study of SDMB (2,2 Dimethylbutyrate, Sodium Salt) in Beta Thalassemia Intermedia in Thailand
• Effect of Antioxidant Cocktail in Beta-thalassemia/Hb E Patients
• Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With Beta( β)- Thalassemia.
• Study Of Efficacy,Safety of Combined Deferasirox and Deferiprone Versus Combined Deferiprone and Desferal In Conditions of Iron Overload
• Safety and Efficacy of Deferasirox in Combination With Desferoxamine in β-thalassaemia Patients With Severe Cardiac Iron Overload
• Amlodipine in the Prevention and Treatment of Iron Overload in Patients With Thalassemia Major
• Safety, Efficacy and Pharmacokinetics of an Oral Iron Chelator Given for a Year to Pediatric Patients With Iron Overload
• A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
• The Effect of Vitamin D Supplementation on Calcium Excretion in Thalassemia: a Dose Response Study
• Traditional Chinese Medicine in the Supportive Management of Anaemic and Cytopenic (Leukopenia, Thrombocytopenia) Haematological Disorders
• Evaluating the Safety and Effectiveness of Mozobil Mobilization in Adults With Beta-Thalassemia Major
• Safety and Pharmacodynamic Study of an Oral Iron Chelator Given for 6 Months to Patients With Iron Overload
• Efficacy and Safety of Neridronate (Nerixia®)to Treat Osteoporosis in Patients With TM and TI
• Amlodipine Use in the Prevention and Treatment of Iron Overload in Patients With Thalassemia Major
• CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant
• Safety and Pharmacokinetic Study of Escalating Multiple Doses of an Iron Chelator in Patients With Iron Overload
• Zoledronic Acid for the Prevention of Bone Loss Post-bone Marrow Transplantation for Thalassemia Major Patients
• Therapeutic Effects of Silymarin in Patients With B-thalassemia Major
• Haploidentical PBMC Transplant for Severe Congenital Anemias
• Effect of Deferasirox on Patients With Cardiac MRI T2* < 20 Msec
• Combination Therapy of Hydroxyurea With L-Carnitine and Magnesium Chloride in Thalassemia Intermedia
• Intensive Combined Chelation Therapy for Iron-Induced Cardiac Disease in Patients With Thalassemia Major
• Phase 1/2 Study of HQK-1001 in Patients With Beta Thalassemia
• Pilot Study for Patients With Poor Response to Deferasirox
• Efficacy Study of the Use of Sequential DFP-DFO Versus DFP
• Evaluating the Safety and Effectiveness of Decitabine in People With Thalassemia Intermedia
• Evaluating the Efficacy of Deferasirox in Transfusion Dependent Chronic Anaemias (Myelodysplastic Syndrome, Beta-thalassaemia Patients) With Chronic Iron Overload
• Phase I Study to Examine the Effect of Deferasirox on Renal Hemodynamics in β-thalassemia Patients With Transfusional Iron Overload
• Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment
• Stem Cell Transplant in Sickle Cell Disease and Thalassemia
• A 4-year Extension Study to Core 1-year Study of Iron Chelation Therapy With Deferasirox in β-thalassemia Major Pediatric Patients With Transfusional Iron Overload.
• Evaluating the Safety of G-CSF Mobilization in Individuals With Beta Thalassemia Major
• Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Rare Chronic Anemia Patients
• Extension Study of the Efficacy and Safety of Deferasirox Treatment in Beta-thalassemia Patients With Transfusional Hemosiderosis (Study Amended to 2-year Duration)
• An Extension Study of Iron Chelation Therapy With Deferasirox (ICL670) in β-thalassemia Patients With Transfusional Iron Overload
• A Study of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis
• Combination Therapy Compared With Single-Drug Therapy in Patients With Cardiac Diseases
• Efficacy Study in Removing Excess Iron From the Heart
• Combined Chelation Treatment With Deferiprone and Deferoxamine in Thalassemia Major
• Iron Balance Study of DFO and GT56-252 in Patients With Transfusional Iron Overload Secondary to Beta-Thalassemia
• Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias
• Safety & Efficacy of ICL670 vs. Deferoxamine in Beta-thalassemia Patients With Iron Overload Due to Blood Transfusions
• Stem Cell Transplantation After Reduced-Dose Chemotherapy for Patients With Sickle Cell Disease or Thalassemia
• Cord Blood Transplantation for Sickle Cell Anemia and Thalassemia
• 5-Azacytidine and Phenylbutyrate to Treat Severe Thalassemia
• Phase II Study of Arginine Butyrate With or Without Epoetin Alfa in Patients With Thalassemia Intermedia
• 5-Azacytidine and Phenylbutyrate to Treat Severe Thalassemia
• Study of Allogeneic Bone Marrow Transplantation Using Matched, Related Donors in Patients With Nonmalignant Hematologic Disorders
• Hydroxyurea to Treat Beta-Thalassemia (Cooley's Anemia)
• Thalassemia (Cooley's Anemia) Clinical Research Network (TCRN)
• Evaluation of Subcutaneous Desferrioxamine as Treatment for Transfusional Hemochromatosis
• Chelation Therapy of Iron Overload With Pyridoxal Isonicotinoyl Hydrazone