284. ダイアモンド・ブラックファン貧血 Disease details / Clinical trials / Drug dev / DR info

Abatacept
   Emory University
      2014   Phase 1   NCT01917708   United States
Alefacept
   Emory University
      2010   -   NCT01319851   United States
Alemtuzumab
   Paul Szabolcs
      2018   Phase 1/Phase 2   NCT03653338   United States
   University of California, San Francisco
      2005   Phase 2   NCT00301834   United States
Allogeneic bone marrow transplantation
   Fred Hutchinson Cancer Center
      2022   Phase 2   NCT04965597   United States
   University of California, San Francisco
      2005   Phase 2   NCT00301834   United States
      2000   Phase 1/Phase 2   NCT00305708   United States
Allogeneic hematopoietic stem cell transplantation
   Roswell Park Cancer Institute
      2012   Phase 2   NCT01529827   United States
   University of California, San Francisco
      2005   Phase 2   NCT00301834   United States
Alpha beta depletion
   Mitchell Cairo
      2021   Phase 2   NCT04099966   United States
ANTI-thymocyte globulin
   Masonic Cancer Center, University of Minnesota
      2000   Phase 2/Phase 3   NCT00176878   United States
      1999   -   NCT00290628   United States
   University of California, San Francisco
      2000   Phase 1/Phase 2   NCT00305708   United States
Antithymocyte globulin
   National Heart, Lung, and Blood Institute (NHLBI)
      1998   Phase 2   NCT00001749   United States
Biospecimen collection
   Fred Hutchinson Cancer Center
      2022   Phase 2   NCT04965597   United States
Bitopertin
   National Heart, Lung, and Blood Institute (NHLBI)
      2023   Phase 1/Phase 2   NCT05828108   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
Bone marrow transplantation
   Stanford University
      2009   Phase 2   NCT00957931   United States
BPX-501 T cells
   Bellicum Pharmaceuticals
      2014   Phase 1/Phase 2   NCT02065869   Germany;Italy;Spain;United Kingdom;United States
Busulfan
   Masonic Cancer Center, University of Minnesota
      2000   Phase 2/Phase 3   NCT00176878   United States
      1999   -   NCT00290628   United States
   University of California, San Francisco
      2005   Phase 2   NCT00301834   United States
      2000   Phase 1/Phase 2   NCT00305708   United States
Busulfan, cyclophosphamide, ATG, gcsf
   Masonic Cancer Center, University of Minnesota
      2002   Phase 2/Phase 3   NCT00176852   United States
Busulfan, fludarabine, ATG, TLI
   Masonic Cancer Center, University of Minnesota
      2002   Phase 2/Phase 3   NCT00176852   United States
Campath, fludarabine, cyclophosphamide
   Masonic Cancer Center, University of Minnesota
      2002   Phase 2/Phase 3   NCT00176852   United States
CD3/CD19 depleted leukocytes
   Paul Szabolcs
      2018   Phase 1/Phase 2   NCT03653338   United States
CD34 stem cell selection therapy
   Diane George
      2013   Phase 1/Phase 2   NCT01966367   United States
CD45RA depleted leukocytes
   Paul Szabolcs
      2018   Phase 1/Phase 2   NCT03653338   United States
Computed tomography
   Fred Hutchinson Cancer Center
      2022   Phase 2   NCT04965597   United States
Cyclophosphamide
   Masonic Cancer Center, University of Minnesota
      1999   -   NCT00290628   United States
Cyclophosphamide dose level 1
   Children's Hospital Los Angeles
      2007   Phase 2   NCT02512679   -
Cyclophosphamide dose level 2
   Children's Hospital Los Angeles
      2007   Phase 2   NCT02512679   -
Cyclophosphamide dose level 3
   Children's Hospital Los Angeles
      2007   Phase 2   NCT02512679   -
Cyclophosphamide dose level 4
   Children's Hospital Los Angeles
      2007   Phase 2   NCT02512679   -
Cyclosporine
   Masonic Cancer Center, University of Minnesota
      1999   -   NCT00290628   United States
   National Heart, Lung, and Blood Institute (NHLBI)
      1998   Phase 2   NCT00001749   United States
   University of California, San Francisco
      2005   Phase 2   NCT00301834   United States
Daclizumab
   Neal Young, M.D.
      1999   Phase 2   NCT00001962   United States
Deferasirox
   Novartis
      2007   Phase 4   NCT00673608   Australia
   Novartis Pharma Services AG
      2008   Phase 2   EUCTR2007-000766-20-GB   Italy;United Kingdom
   Novartis Pharmaceuticals
      2005   Phase 3   NCT00235391   Australia;Belgium;Canada;Germany;Greece;Italy;Netherlands;Spain;Taiwan;Thailand;Turkey;United Kingdom;United States
Deferitazole
   Shire Development LLC
      2012   Phase 2   EUCTR2011-006322-25-GB   Canada;Egypt;Italy;Lebanon;Thailand;Turkey;United Kingdom;United States
Deferoxamine
   Novartis Pharma Services AG
      2008   Phase 2   EUCTR2007-000766-20-GB   Italy;United Kingdom
Desferal
   Novartis Pharma Services AG
      2008   Phase 2   EUCTR2007-000766-20-GB   Italy;United Kingdom
DFO
   Novartis Pharma Services AG
      2008   Phase 2   EUCTR2007-000766-20-GB   Italy;United Kingdom
Echocardiography
   Fred Hutchinson Cancer Center
      2022   Phase 2   NCT04965597   United States
Eltrombopag
   National Heart, Lung, and Blood Institute (NHLBI)
      2020   Phase 1/Phase 2   NCT04269889   United States
Enriched hematopoetic stem cell infusion
   Talaris Therapeutics Inc.
      2011   Phase 1/Phase 2   NCT01419704   United States
Exjade
   Novartis Pharma Services AG
      2008   Phase 2   EUCTR2007-000766-20-GB   Italy;United Kingdom
FBS0701 capsule
   FERROKIN BIOSCIENCES INC.
      2012   -   EUCTR2011-006322-25-IT   Italy;United Kingdom;United States
      2012   -   EUCTR2011-005675-16-IT   Canada;Italy;Lebanon;Turkey;United States
      2010   -   EUCTR2010-019645-25-IT   Italy;United Kingdom
FBS0701/SSP004184
   FERROKIN BIOSCIENCES INC.
      2012   -   EUCTR2011-006322-25-IT   Italy;United Kingdom;United States
Filgrastim
   Masonic Cancer Center, University of Minnesota
      1999   -   NCT00290628   United States
Fludarabine
   Paul Szabolcs
      2018   Phase 1/Phase 2   NCT03653338   United States
Fludarabine monophosphate
   Masonic Cancer Center, University of Minnesota
      2000   Phase 2/Phase 3   NCT00176878   United States
Fludarabine phosphate
   Fred Hutchinson Cancer Center
      2022   Phase 2   NCT04965597   United States
   Roswell Park Cancer Institute
      2012   Phase 2   NCT01529827   United States
   University of California, San Francisco
      2005   Phase 2   NCT00301834   United States
      2000   Phase 1/Phase 2   NCT00305708   United States
G-CSF
   National Heart, Lung, and Blood Institute (NHLBI)
      2001   Phase 2   NCT00011505   United States
Human placental derived stem cell
   New York Medical College
      2013   Phase 1   NCT01586455   United States
Hydroxyurea
   Paul Szabolcs
      2018   Phase 1/Phase 2   NCT03653338   United States
ICL670
   Novartis Pharma Services AG
      2008   Phase 2   EUCTR2007-000766-20-GB   Italy;United Kingdom
Iron chelating agents
   FERROKIN BIOSCIENCES INC.
      2012   -   EUCTR2011-006322-25-IT   Italy;United Kingdom;United States
      2012   -   EUCTR2011-005675-16-IT   Canada;Italy;Lebanon;Turkey;United States
L-leucine
   Federal Scientific Clinical Centre of Pediatric Hematology, Oncology and Immunology named after Dmitry Rogache
      2014   Phase 2   NCT02386267   Russian Federation
Laboratory biomarker analysis
   Roswell Park Cancer Institute
      2012   Phase 2   NCT01529827   United States
Lapine T-lymphocyte immune globulin
   Fred Hutchinson Cancer Center
      2022   Phase 2   NCT04965597   United States
Lenalidomide
   Jason Robert Gotlib
      2009   Phase 1   NCT01034592   United States
Leucine
   Northwell Health
      2013   Phase 1/Phase 2   NCT01362595   United States
Leukapheresis
   National Heart, Lung, and Blood Institute (NHLBI)
      2001   Phase 2   NCT00011505   United States
Magnesium hydroxide
   Shire Development LLC
      2012   Phase 2   EUCTR2011-006322-25-GB   Canada;Egypt;Italy;Lebanon;Thailand;Turkey;United Kingdom;United States
      2010   Phase 2   EUCTR2010-019645-25-GB   Italy;Thailand;Turkey;United Kingdom;United States
Melphalan
   Masonic Cancer Center, University of Minnesota
      1999   -   NCT00290628   United States
   Roswell Park Cancer Institute
      2012   Phase 2   NCT01529827   United States
Mesenchymal stromal cells
   Stanford University
      2009   Phase 2   NCT00957931   United States
Methotrexate
   Fred Hutchinson Cancer Center
      2022   Phase 2   NCT04965597   United States
   Roswell Park Cancer Institute
      2012   Phase 2   NCT01529827   United States
   University of California, San Francisco
      2005   Phase 2   NCT00301834   United States
Methylprednisolone
   Masonic Cancer Center, University of Minnesota
      1999   -   NCT00290628   United States
   University of California, San Francisco
      2005   Phase 2   NCT00301834   United States
Multigated acquisition scan
   Fred Hutchinson Cancer Center
      2022   Phase 2   NCT04965597   United States
Mycophenolate mofetil
   Masonic Cancer Center, University of Minnesota
      1999   -   NCT00290628   United States
   Roswell Park Cancer Institute
      2012   Phase 2   NCT01529827   United States
Myeloablative preparative regimen
   Masonic Cancer Center, University of Minnesota
      2014   -   NCT02179359   United States
Peripheral blood stem cell transplantation
   Fred Hutchinson Cancer Center
      2022   Phase 2   NCT04965597   United States
   Roswell Park Cancer Institute
      2012   Phase 2   NCT01529827   United States
   University of California, San Francisco
      2005   Phase 2   NCT00301834   United States
      2000   Phase 1/Phase 2   NCT00305708   United States
Quality-OF-life assessment
   Fred Hutchinson Cancer Center
      2022   Phase 2   NCT04965597   United States
Radiation therapy
   Masonic Cancer Center, University of Minnesota
      1999   -   NCT00290628   United States
   University of California, San Francisco
      2000   Phase 1/Phase 2   NCT00305708   United States
Reduced intensity preparative regimen
   Masonic Cancer Center, University of Minnesota
      2014   -   NCT02179359   United States
Reduced toxicity ablative regimen
   Masonic Cancer Center, University of Minnesota
      2014   -   NCT02179359   United States
Rimiducid
   Bellicum Pharmaceuticals
      2017   Phase 1/Phase 2   NCT03733249   Italy;Saudi Arabia;United Kingdom
      2014   Phase 1/Phase 2   NCT02065869   Germany;Italy;Spain;United Kingdom;United States
Rituximab
   National Heart, Lung, and Blood Institute (NHLBI)
      2005   Phase 2   NCT00229619   United States
   Paul Szabolcs
      2018   Phase 1/Phase 2   NCT03653338   United States
Rivogenlecleucel
   Bellicum Pharmaceuticals
      2017   Phase 1/Phase 2   NCT03733249   Italy;Saudi Arabia;United Kingdom
Sotatercept with prednisone boost
   Northwell Health
      2012   Phase 1/Phase 2   NCT01464164   United States
SSP-004184 100 MG capsule
   Shire Development LLC
      2010   Phase 2   EUCTR2010-019645-25-GB   Italy;Thailand;Turkey;United Kingdom;United States
SSP-004184 250 MG capsule
   Shire Development LLC
      2010   Phase 2   EUCTR2010-019645-25-GB   Italy;Thailand;Turkey;United Kingdom;United States
SSP-004184 375 MG capsule
   Shire Development LLC
      2010   Phase 2   EUCTR2010-019645-25-GB   Italy;Thailand;Turkey;United Kingdom;United States
SSP-004184 50 MG capsule
   Shire Development LLC
      2010   Phase 2   EUCTR2010-019645-25-GB   Italy;Thailand;Turkey;United Kingdom;United States
SSP-004184 500 MG capsule
   Shire Development LLC
      2010   Phase 2   EUCTR2010-019645-25-GB   Italy;Thailand;Turkey;United Kingdom;United States
SSP-004184AQ 100 MG capsule
   Shire Development LLC
      2012   Phase 2   EUCTR2011-006322-25-GB   Canada;Egypt;Italy;Lebanon;Thailand;Turkey;United Kingdom;United States
SSP-004184AQ 200 MG capsule
   Shire Development LLC
      2012   Phase 2   EUCTR2011-006322-25-GB   Canada;Egypt;Italy;Lebanon;Thailand;Turkey;United Kingdom;United States
SSP-004184AQ 250 MG capsule
   Shire Development LLC
      2012   Phase 2   EUCTR2011-006322-25-GB   Canada;Egypt;Italy;Lebanon;Thailand;Turkey;United Kingdom;United States
SSP-004184AQ 375 MG capsule
   Shire Development LLC
      2012   Phase 2   EUCTR2011-006322-25-GB   Canada;Egypt;Italy;Lebanon;Thailand;Turkey;United Kingdom;United States
SSP-004184AQ 50 MG capsule
   Shire Development LLC
      2012   Phase 2   EUCTR2011-006322-25-GB   Canada;Egypt;Italy;Lebanon;Thailand;Turkey;United Kingdom;United States
Stem cell infusion
   Masonic Cancer Center, University of Minnesota
      2002   Phase 2/Phase 3   NCT00176852   United States
Stem cell transplant
   Masonic Cancer Center, University of Minnesota
      2000   Phase 2/Phase 3   NCT00176878   United States
Tacrolimus
   Fred Hutchinson Cancer Center
      2022   Phase 2   NCT04965597   United States
   Roswell Park Cancer Institute
      2012   Phase 2   NCT01529827   United States
Thiotepa
   Paul Szabolcs
      2018   Phase 1/Phase 2   NCT03653338   United States
Thiotepa--escalated dose
   University of Florida
      2018   Phase 1/Phase 2   NCT03513328   United States
Thiotepa--single daily dose
   University of Florida
      2018   Phase 1/Phase 2   NCT03513328   United States
Total body irradiation
   Masonic Cancer Center, University of Minnesota
      2002   Phase 2/Phase 3   NCT00176852   United States
Total lymphoid irradiation
   Masonic Cancer Center, University of Minnesota
      2000   Phase 2/Phase 3   NCT00176878   United States
Total-body irradiation
   Roswell Park Cancer Institute
      2012   Phase 2   NCT01529827   United States
Treosulfan
   Fred Hutchinson Cancer Center
      2022   Phase 2   NCT04965597   United States
Trifluoperazine
   Adrianna Vlachos
      2019   Phase 1/Phase 2   NCT03966053   United States
Umbilical cord blood transplantation
   Masonic Cancer Center, University of Minnesota
      1999   -   NCT00290628   United States
   University of California, San Francisco
      2005   Phase 2   NCT00301834   United States
      2000   Phase 1/Phase 2   NCT00305708   United States
X-RAY imaging
   Fred Hutchinson Cancer Center
      2022   Phase 2   NCT04965597   United States