beta-Thalassemia
A disorder characterized by reduced synthesis of the beta chains of hemoglobin. There is retardation of hemoglobin A synthesis in the heterozygous form (thalassemia minor), which is asymptomatic, while in the homozygous form (thalassemia major, Cooley's anemia, Mediterranean anemia, erythroblastic anemia), which can result in severe complications and even death, hemoglobin A synthesis is absent.
Drugs by status
Approved for this indication (4)
Phase 4 trials (21)
Phase 3 trials (23)
Phase 2 trials (53)
- azacitidine
- plerixafor
- apotransferrin
- cd3,cd19 depleted hematopoietic stem cells
- pyridoxal isonicotinoyl hydrazone
- deferitazole
- prednisone
- emeramide
- lymphocyte immune globulin, anti-thymocyte globulin (equine)
- hemopoietic stem cell
- hydroxychloroquine
- icosapent ethyl
- Tovinontrine
- Filgrastim
- neridronic acid
- allogeneic stem cells
- lidocaine
- bitopertin
- sirolimus
- sodium 2,2-dimethylbutyrate
- sotatercept
- sp-420
- abatacept
- alemtuzumab
- bortezomib
- allogeneic cord blood
- autologous cd34+ cells
- autologous hematopoietic stem cells genetically modified with globe lentiviral vector encoding for the human beta-globin gene
- umbilical cord mesenchymal stem cells
- cd45-depleted allogenic peripheral blood stem cell
- +23 more
Phase 1 trials (13)
Other trials (12)
- decitabine
- vitamin e
- lymphocytes
- allogeneic hematopoietic stem cells
- stem cells
- taurine
- donor regulatory t lymphocytes
- allogeneic bone marrow
- haplo-identical hematopoietic stem cells
- sargramostim
- cytarabine
- Autologous hematopoeitic stem cells and mesenchymal stem cells transduced with lentiviral vector carrying the FANCA gene ex vivo
Organizations
Organization Involved with Phase 4 Indications (26)
- Apotex
- Azienda Ospedaliera V. Cervello
- Cairo University
- University of London
- Cornell University
- Harvard University
- Guangxi Medical University
- Hacettepe University
- Hainan People's Hospital
- Nanjing University
- Ospedale Microcitemico
- Tanta University
- University of Athens
- Fujian Medical University
- Peking University
- Xiamen University
- University of Cagliari
- University of Indonesia
- Jiao Tong University
- University of Turin
- Guizhou Medical University
- CORDA, The Heart Charity
- The UK Thalassemia Society
- People's Liberation Army of China
- The First People's Hospital of Yunan Province
- Liuzhou General Hospital
Organization Involved with Phase 3 Indications (17)
- Acceleron Pharma, Inc.
- Celgene
- Cerus Corporation
- Hamad Medical Corporation
- Madaus Inc
- bluebird bio
- Fundação de Amparo à Pesquisa do Estado de São Paulo
- Federal Research Institute of Pediatric Hematology, Oncology and Immunology
- University of Arizona
- Ain Shams University
- Ege University
- University of Campinas
- Beni Suef University
- American University of Beirut
- Isfahan University
- Ospedale Regionale per le Microcitemie azienda
- Maternity and Children Hospital, Makkah
Organization Involved with Phase 2 Indications (71)
- Imara, Inc.
- Shire
- Vertex
- Roche
- Genzyme
- Bioverativ Therapeutics Inc.
- EmeraMed
- Ionis Pharmaceuticals, Inc.
- La Jolla Pharmaceutical Company
- Nova Laboratories Limited
- Protagonist Therapeutics
- Vifor Inc.
- Fondazione Telethon
- National Institutes of Health (NIH)
- National Cancer Institute (NCI)
- National Heart, Lung, and Blood Institute (NHLBI)
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- Laikο General Hospital, Athens
- University of Pennsylvania
- University of Pittsburgh
- Central South University
- Boston University
- Case Western Reserve University
- Duke University
- Mahidol University
- New England Research Institutes
- New York Medical College
- Northwell Health
- Northwestern University
- +41 more
Organization Involved with Phase 1 Indications (7)
Organization Involved with Other Experimental Indications (10)