Thalassemia



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


Condition:   Thalassemia Major
Intervention:  
Sponsor:   Assiut University
Not yet recruiting - verified May 2017


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 - verified May 2017


A Study of EPEG in Beta Thalassemia Patients


Condition:   Beta-Thalassemia
Intervention:   Drug: EPEG (Pegylated erythropoeitin)
Sponsor:   Prolong Pharmaceuticals
Not yet recruiting - verified October 2016


A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Subjects With Transfusion-Dependent β-Thalassemia


Condition:   Beta-Thalassemia
Intervention:   Genetic: LentiGlobin BB305 Drug Product
Sponsor:   bluebird bio
Recruiting - verified April 2017


Benefits of Nigella Sativa in Children With Beta Thalassemia Major


Condition:   Nigella Sativa With Beta Thalassemia Major
Intervention:   Drug: nigella sativ
Sponsor:   Tanta University
Recruiting - verified June 2016


Effect of Spirulina Compared to Amlodipine on Cardiac Iron Overload in Children With Beta Thalassemia


Condition:   Beta Thalassemia Major
Interventions:   Drug: Spirulina;   Drug: Amlodipine
Sponsor:   Tanta University
Active, not recruiting - verified January 2016


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 - verified April 2017


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


Condition:   Beta Thalassemia
Intervention:   Other: spirulina
Sponsor:   Tanta University
Active, not recruiting - verified April 2017


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 - verified June 2016


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 - verified June 2016


ACE-536 Extension Study - Beta Thalassemia


Condition:   Beta-Thalassemia
Intervention:   Drug: ACE-536
Sponsor:   Acceleron Pharma, Inc.
Active, not recruiting - verified December 2016


Haploidentical Hematopoietic Stem Cell Transplantation for Children With Sickle Cell Disease and Thalassemia Using CD34+ Positive Selected Grafts


Conditions:   Sickle Cell-thalassemia Disease;   Thalassemia
Intervention:   Drug: peripheral blood stem cell graft that are CD34+ selected
Sponsors:   Catherine Bollard;   Children's Research Institute
Recruiting - verified October 2016


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


Condition:   Thalassemia (Transfusion Delendent)
Intervention:   Drug: deferasirox
Sponsor:   Novartis Pharmaceuticals
Withdrawn - verified March 2015


Allograft for Sickle Cell Disease and Thalassemia


Condition:   Sickle Cell Disease and Thalassemia
Intervention:   Biological: Donor Stem Cell Transplantation
Sponsor:   University of Texas Southwestern Medical Center
Recruiting - verified June 2016


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 - verified January 2014


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 - verified December 2016


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 - verified May 2017


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 - verified December 2016


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


Condition:   β-thalassemia Major
Intervention:   Drug: Deferasirox
Sponsor:   Novartis Pharmaceuticals
Withdrawn - verified August 2014


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 - verified July 2016


Combination Deferasirox and Deferiprone for Severe Iron Overload in Thalassemia


Condition:   Thalassemia Major With Severe Transfusional Iron Overload
Intervention:   Drug: Deferasirox and deferiprone
Sponsors:   Children's Hospital of Philadelphia;   The Cooley’s Anemia Foundation,;   Ann & Robert H Lurie Children's Hospital of Chicago
Active, not recruiting - verified February 2017


ß-Thalassemia Major With Autologous CD34+ Hematopoietic Progenitor Cells Transduced With TNS9.3.55 a Lentiviral Vector Encoding the Normal Human ß-Globin Gene


Condition:   Confirmed Diagnosis of ß-thalassemia Major
Intervention:   Genetic: Autologous CD34+ cells transduced with TNS9.3.55
Sponsor:   Memorial Sloan Kettering Cancer Center
Active, not recruiting - verified August 2016


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 - verified January 2017


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


Condition:   Thalassemia Major
Intervention:  
Sponsor:   National Human Genome Research Institute (NHGRI)
Completed - verified January 7, 2016


Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLO


Condition:   Thalassemia
Interventions:   Drug: Busulfan;   Drug: Fludarabine;   Drug: Campath 1H;   Drug: Cyclophosphamide;   Drug: MESNA
Sponsors:   Baylor College of Medicine;   Texas Children's Hospital
Terminated - verified July 2016

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