Thalassemia



A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (β)-Thalassemia


Condition:   Thalassemia
Interventions:   Drug: Luspatercept;   Other: Placebo;   Other: Best Supportive Care (BSC)
Sponsors:   Celgene;   Acceleron Pharma, Inc.
Not yet recruiting


Gene Therapy for Beta-Thalassemia Major Using Autologous Hematopoietic Stem Cell Genetically Modified


Condition:   Beta Thalassemia Major
Intervention:   Genetic: Autologous CD34+ cells genetically modified
Sponsors:   Nanfang Hospital of Southern Medical University;   Guangdong Yike Gene Science and Technology CO.,Ltd
Not yet recruiting


Long-term Safety and Efficacy Follow-up of Subjects Treated With GSK2696277 for Transfusion Dependent Beta-thalassemia in San Raffaele Telethon Institute of Gene Therapy-Beta Thalassemia (TIGET-BTHAL) Study


Condition:   Beta Thalassaemia
Intervention:   Other: Safety and Efficacy assessments
Sponsors:   GlaxoSmithKline;   San Raffaele Telethon Institute of Gene Therapy (SR-TIGET)
Recruiting


A Study of Bitopertin (RO4917838) in Adults With Non-Transfusion-Dependent Beta-Thalassemia


Condition:   Beta-Thalassemia
Intervention:   Drug: Bitopertin
Sponsor:   Hoffmann-La Roche
Recruiting


Adiponectin, IL-6 and hsC-RP in Relation to Carotid Intima-media Thickness in B-thalassemia Patients


Condition:   Thalassemia
Interventions:   Diagnostic Test: Interleukin-6;   Diagnostic Test: HsC-RP;   Diagnostic Test: Adiponectin level;   Diagnostic Test: Carotid intima media thickness
Sponsor:   Assiut University
Not yet recruiting


Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major


Condition:   Thalassemia Major
Interventions:   Drug: Busulfan;   Drug: Cyclophosphamide;   Drug: Fludarabine;   Drug: Mycophenolate mofetil;   Drug: Tacrolimus;   Drug: Methotrexate;   Drug: Thymoglobulin;   Drug: Basiliximab
Sponsor:   First Affiliated Hospital of Guangxi Medical University
Recruiting


Evaluation of Nutritional Status in Thalassemia Major Patients in Assiut Children Hospital


Condition:   Thalassemia Major
Intervention:  
Sponsor:   Assiut University
Not yet recruiting


Monitoring of Chimerism After Transplantation in Patients With β Thalassemia Major and the Treatment Strategies for the Reduction of Chimerism


Condition:   Beta Thalassemia Major
Interventions:   Drug: Interleukin-2;   Drug: Donor Regulatory T-Lymphocytes
Sponsor:   First Affiliated Hospital of Guangxi Medical University
Recruiting


To Document the Burden of Illness on the Quality of Life and the Impact on Healthcare Utilization in (Beta) β-thalassemia Subjects Who Are Transfusion Dependent (TD) and Non-transfusion Dependent (NTD) Receiving Standard of Care


Condition:   Beta-Thalassemia
Interventions:   Other: Quality of Life (QOL) questionnaires;   Other: Healthcare Resource Utilization
Sponsor:   Celgene
Completed


Effects of Spirulina on Cardiac Functions in Children With Beta Thalassemia Major


Condition:   Beta Thalassemia
Intervention:   Other: spirulina
Sponsor:   Tanta University
Active, not recruiting


Gene Therapy for Transfusion Dependent Beta-thalassemia


Condition:   Beta-Thalassemia
Intervention:   Genetic: Autologous hematopoietic stem cells genetically modified with GLOBE lentiviral vector encoding for the human beta-globin gene
Sponsors:   IRCCS San Raffaele;   Fondazione Telethon
Recruiting


Reproductive Capacity and Iron Burden in Thalassemia


Condition:   THALASSEMIA MAJOR
Interventions:   Other: Blood Draw/Semen Exam;   Other: Retrospective data/Chart Review/Relevant Clinical Results;   Other: Pituitary MRI
Sponsors:   Children's Hospital & Research Center Oakland;   Novartis Pharmaceuticals
Recruiting


ACE-536 Extension Study - Beta Thalassemia


Condition:   Beta-Thalassemia
Intervention:   Drug: ACE-536
Sponsor:   Acceleron Pharma, Inc.
Active, not recruiting


Effect of Deferasirox on Endocrine Complications in Subjects With Transfusion Dependent Thalassemia


Condition:   Thalassemia (Transfusion Delendent)
Intervention:   Drug: deferasirox
Sponsor:   Novartis Pharmaceuticals
Withdrawn


Study of Efficacy and Safety of INC424 in Regularly Transfused Patients With Thalassemia.


Condition:   Thalassemia Major
Intervention:   Drug: ruxolitinib
Sponsor:   Novartis Pharmaceuticals
Completed


Deferasirox BID (Twice a Day) in Transfusion Dependent Thalassemia Patients With Inadequate Response to High Doses


Condition:   Focus on Transfusion Dependent Thalassemia Patients Who Are Inadequate Responders to Deferasirox > 35mg/kgQD
Intervention:   Drug: Deferasirox
Sponsor:   Novartis Pharmaceuticals
Withdrawn


Study to Evaluate the Effects of ACE-536 in Patients With Beta-thalassemia


Condition:   B-Thalassemia
Intervention:   Drug: ACE-536
Sponsor:   Acceleron Pharma, Inc.
Completed


A Study Evaluating the Safety and Efficacy of the LentiGlobin® BB305 Drug Product in Beta-Thalassemia Major Subjects


Condition:   Beta-thalassemia Major
Intervention:   Genetic: LentiGlobin® BB305 Drug Product
Sponsor:   bluebird bio
Active, not recruiting


Study to Evaluate Efficacy and Safety of S303 Treated Red Blood Cells (RBCs)in Subjects With Thalassemia Major Requiring Chronic RBC Transfusion


Condition:   Thalassemia Major
Interventions:   Biological: S-303 Treated Red Blood Cells (RBCs);   Biological: Conventional, untreated Red Blood Cells
Sponsor:   Cerus Corporation
Active, not recruiting


Efficacy and Safety Study of Deferasirox in Patients With Non-transfusion Dependent Thalassemia


Condition:   Non-transfusion Dependent Thalassemia
Intervention:   Drug: ICL670 deferasirox
Sponsor:   Novartis Pharmaceuticals
Active, not recruiting


Effect of Antioxidant Cocktail in Beta-thalassemia/Hb E Patients


Condition:   Thalassemia
Interventions:   Drug: curcuminoids, N-acetylcysteine, deferiprone and vitamin E;   Drug: Curcuminoids and alpha-tocopherol
Sponsors:   Mahidol University;   ChaingMai University
Completed


Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With Beta( β)- Thalassemia.


Conditions:   Beta Thalassemia Major;   Beta Thalassemia Intermedia
Interventions:   Drug: Sotatercept 0.1mg/kg;   Drug: Sotatercept 0.3mg/kg;   Drug: Sotatercept 0.5mg/kg;   Drug: Sotatercept 0.75mg/kg;   Drug: Sotatercept 1.0mg/kg;   Drug: Sotatercept 1.5mg/kg
Sponsor:   Celgene
Active, not recruiting


Perceptions of Thalassemia Major in Singapore: An Exploratory Study of Stigma


Condition:   Thalassemia Major
Intervention:  
Sponsor:   National Human Genome Research Institute (NHGRI)
Completed


Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia


Condition:   Severe Thalassemia
Intervention:   Drug: Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan
Sponsors:   Washington University School of Medicine;   New England Research Institutes;   Pediatric Blood and Marrow Transplant Consortium
Completed


Genetic Factors Affecting the Severity of Beta Thalassemia


Condition:   Beta Thalassemia
Intervention:  
Sponsor:   Deborah Rund
Completed

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