286. Hereditary sideroblastic anemia Disease details / Clinical trials / Drug dev / DR info
Clinical trials : 7 / Drugs : 25 - (DrugBank : 10) / Drug target genes : 19 - Drug target pathways : 64
Drugs, Primary sponsors and Trial info (Enrollment year, Phase, Trial ID, Countries)
ALA
Department of Hematolgy and Rheumatology, Tohoku University Graduate School of Medicine
2016 - JPRN-UMIN000023490 Japan
Allogeneic bone marrow transplantation
Fred Hutchinson Cancer Center
2022 Phase 2 NCT04965597 United States
Aminolevulinic acid
Department of Hematology and Oncology, Graduate School of Medicine, Kyoto University
2016 - JPRN-UMIN000021407 Japan
Biospecimen collection
Fred Hutchinson Cancer Center
2022 Phase 2 NCT04965597 United States
Bone marrow aspiration
Fred Hutchinson Cancer Center
2022 Phase 2 NCT04965597 United States
Bone marrow biopsy
Fred Hutchinson Cancer Center
2022 Phase 2 NCT04965597 United States
Computed tomography
Fred Hutchinson Cancer Center
2022 Phase 2 NCT04965597 United States
Deferasirox
NOVARTIS FARMA
2010 - EUCTR2010-021062-29-IT Greece;Italy;United Kingdom
Novartis Pharma Services AG
2011 - EUCTR2010-021062-29-GR Greece;Italy;United Kingdom
2010 - EUCTR2010-021062-29-GB Greece;Italy;United Kingdom
2008 Phase 2 EUCTR2007-000766-20-GB Italy;United Kingdom
Deferoxamine
NOVARTIS FARMA
2010 - EUCTR2010-021062-29-IT Greece;Italy;United Kingdom
Novartis Pharma Services AG
2008 Phase 2 EUCTR2007-000766-20-GB Italy;United Kingdom
Deferoxamine mesilate
Novartis Pharma Services AG
2011 - EUCTR2010-021062-29-GR Greece;Italy;United Kingdom
2010 - EUCTR2010-021062-29-GB Greece;Italy;United Kingdom
Desferal
NOVARTIS FARMA
2010 - EUCTR2010-021062-29-IT Greece;Italy;United Kingdom
Novartis Pharma Services AG
2011 - EUCTR2010-021062-29-GR Greece;Italy;United Kingdom
2008 Phase 2 EUCTR2007-000766-20-GB Italy;United Kingdom
DFO
Novartis Pharma Services AG
2011 - EUCTR2010-021062-29-GR Greece;Italy;United Kingdom
2010 - EUCTR2010-021062-29-GB Greece;Italy;United Kingdom
2008 Phase 2 EUCTR2007-000766-20-GB Italy;United Kingdom
Echocardiography
Fred Hutchinson Cancer Center
2022 Phase 2 NCT04965597 United States
Exjade
NOVARTIS FARMA
2010 - EUCTR2010-021062-29-IT Greece;Italy;United Kingdom
Novartis Pharma Services AG
2011 - EUCTR2010-021062-29-GR Greece;Italy;United Kingdom
2010 - EUCTR2010-021062-29-GB Greece;Italy;United Kingdom
2008 Phase 2 EUCTR2007-000766-20-GB Italy;United Kingdom
Fludarabine phosphate
Fred Hutchinson Cancer Center
2022 Phase 2 NCT04965597 United States
ICL670
Novartis Pharma Services AG
2011 - EUCTR2010-021062-29-GR Greece;Italy;United Kingdom
2010 - EUCTR2010-021062-29-GB Greece;Italy;United Kingdom
2008 Phase 2 EUCTR2007-000766-20-GB Italy;United Kingdom
Lapine T-lymphocyte immune globulin
Fred Hutchinson Cancer Center
2022 Phase 2 NCT04965597 United States
Methotrexate
Fred Hutchinson Cancer Center
2022 Phase 2 NCT04965597 United States
Multigated acquisition scan
Fred Hutchinson Cancer Center
2022 Phase 2 NCT04965597 United States
Peripheral blood stem cell transplantation
Fred Hutchinson Cancer Center
2022 Phase 2 NCT04965597 United States
Phosphate
Department of Hematolgy and Rheumatology, Tohoku University Graduate School of Medicine
2016 - JPRN-UMIN000023490 Japan
Department of Hematology and Oncology, Graduate School of Medicine, Kyoto University
2016 - JPRN-UMIN000021407 Japan
Quality-OF-life assessment
Fred Hutchinson Cancer Center
2022 Phase 2 NCT04965597 United States
Tacrolimus
Fred Hutchinson Cancer Center
2022 Phase 2 NCT04965597 United States
Treosulfan
Fred Hutchinson Cancer Center
2022 Phase 2 NCT04965597 United States
X-RAY imaging
Fred Hutchinson Cancer Center
2022 Phase 2 NCT04965597 United States
Department of Hematolgy and Rheumatology, Tohoku University Graduate School of Medicine
2016 - JPRN-UMIN000023490 Japan
Allogeneic bone marrow transplantation
Fred Hutchinson Cancer Center
2022 Phase 2 NCT04965597 United States
Aminolevulinic acid
Department of Hematology and Oncology, Graduate School of Medicine, Kyoto University
2016 - JPRN-UMIN000021407 Japan
Biospecimen collection
Fred Hutchinson Cancer Center
2022 Phase 2 NCT04965597 United States
Bone marrow aspiration
Fred Hutchinson Cancer Center
2022 Phase 2 NCT04965597 United States
Bone marrow biopsy
Fred Hutchinson Cancer Center
2022 Phase 2 NCT04965597 United States
Computed tomography
Fred Hutchinson Cancer Center
2022 Phase 2 NCT04965597 United States
Deferasirox
NOVARTIS FARMA
2010 - EUCTR2010-021062-29-IT Greece;Italy;United Kingdom
Novartis Pharma Services AG
2011 - EUCTR2010-021062-29-GR Greece;Italy;United Kingdom
2010 - EUCTR2010-021062-29-GB Greece;Italy;United Kingdom
2008 Phase 2 EUCTR2007-000766-20-GB Italy;United Kingdom
Deferoxamine
NOVARTIS FARMA
2010 - EUCTR2010-021062-29-IT Greece;Italy;United Kingdom
Novartis Pharma Services AG
2008 Phase 2 EUCTR2007-000766-20-GB Italy;United Kingdom
Deferoxamine mesilate
Novartis Pharma Services AG
2011 - EUCTR2010-021062-29-GR Greece;Italy;United Kingdom
2010 - EUCTR2010-021062-29-GB Greece;Italy;United Kingdom
Desferal
NOVARTIS FARMA
2010 - EUCTR2010-021062-29-IT Greece;Italy;United Kingdom
Novartis Pharma Services AG
2011 - EUCTR2010-021062-29-GR Greece;Italy;United Kingdom
2008 Phase 2 EUCTR2007-000766-20-GB Italy;United Kingdom
DFO
Novartis Pharma Services AG
2011 - EUCTR2010-021062-29-GR Greece;Italy;United Kingdom
2010 - EUCTR2010-021062-29-GB Greece;Italy;United Kingdom
2008 Phase 2 EUCTR2007-000766-20-GB Italy;United Kingdom
Echocardiography
Fred Hutchinson Cancer Center
2022 Phase 2 NCT04965597 United States
Exjade
NOVARTIS FARMA
2010 - EUCTR2010-021062-29-IT Greece;Italy;United Kingdom
Novartis Pharma Services AG
2011 - EUCTR2010-021062-29-GR Greece;Italy;United Kingdom
2010 - EUCTR2010-021062-29-GB Greece;Italy;United Kingdom
2008 Phase 2 EUCTR2007-000766-20-GB Italy;United Kingdom
Fludarabine phosphate
Fred Hutchinson Cancer Center
2022 Phase 2 NCT04965597 United States
ICL670
Novartis Pharma Services AG
2011 - EUCTR2010-021062-29-GR Greece;Italy;United Kingdom
2010 - EUCTR2010-021062-29-GB Greece;Italy;United Kingdom
2008 Phase 2 EUCTR2007-000766-20-GB Italy;United Kingdom
Lapine T-lymphocyte immune globulin
Fred Hutchinson Cancer Center
2022 Phase 2 NCT04965597 United States
Methotrexate
Fred Hutchinson Cancer Center
2022 Phase 2 NCT04965597 United States
Multigated acquisition scan
Fred Hutchinson Cancer Center
2022 Phase 2 NCT04965597 United States
Peripheral blood stem cell transplantation
Fred Hutchinson Cancer Center
2022 Phase 2 NCT04965597 United States
Phosphate
Department of Hematolgy and Rheumatology, Tohoku University Graduate School of Medicine
2016 - JPRN-UMIN000023490 Japan
Department of Hematology and Oncology, Graduate School of Medicine, Kyoto University
2016 - JPRN-UMIN000021407 Japan
Quality-OF-life assessment
Fred Hutchinson Cancer Center
2022 Phase 2 NCT04965597 United States
Tacrolimus
Fred Hutchinson Cancer Center
2022 Phase 2 NCT04965597 United States
Treosulfan
Fred Hutchinson Cancer Center
2022 Phase 2 NCT04965597 United States
X-RAY imaging
Fred Hutchinson Cancer Center
2022 Phase 2 NCT04965597 United States