284. Diamond-Blackfan anemia Disease details / Clinical trials / Drug dev / DR info


Clinical trials : 36 Drugs : 95 - (DrugBank : 34) / Drug target genes : 23 - Drug target pathways : 126

Drugs and their primary sponsors and trial info
(S)-4,5-dihydro-2-[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazo
   Shire Development LLC
      2010   Phase 2   EUCTR2010-019645-25-GB   Italy;Thailand;Turkey;United Kingdom;United States;
(S)-4,5-dihydro-2-[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide
   Shire Development LLC
      2010   Phase 2   EUCTR2010-019645-25-GB   Italy;Thailand;Turkey;United Kingdom;United States;
(S)-4,5-dihydro-2[hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-m
   Shire Development LLC
      2012   Phase 2   EUCTR2011-006322-25-GB   Canada;Egypt;Italy;Lebanon;Thailand;Turkey;United Kingdom;United States;
(S)-4,5-dihydro-2[hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide
   Shire Development LLC
      2012   Phase 2   EUCTR2011-006322-25-GB   Canada;Egypt;Italy;Lebanon;Thailand;Turkey;United Kingdom;United States;
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
   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
      2020   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;
BPX-501 T cells
   Bellicum Pharmaceuticals
      2014   Phase 2   NCT02065869   Germany;Italy;Spain;United Kingdom;United States;
Bone marrow transplantation
   Stanford University
      2009   Phase 2   NCT00957931   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;
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;
Campath, Fludarabine, Cyclophosphamide
   Masonic Cancer Center, University of Minnesota
      2002   Phase 2/Phase 3   NCT00176852   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;
DFO
   Novartis Pharma Services AG
      2008   Phase 2   EUCTR2007-000766-20-GB   Italy;United Kingdom;
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;
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;
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;
FBS0701 Capsule
   FERROKIN BIOSCIENCES INC.
      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
   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   -   EUCTR2011-005675-16-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;
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;
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
   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;
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
   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;
Radiation therapy
   Masonic Cancer Center, University of Minnesota
      1999   -   NCT00290628   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 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;
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;
Sotatercept with prednisone boost
   Northwell Health
      2012   Phase 1/Phase 2   NCT01464164   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
   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 lymphoid irradiation
   Masonic Cancer Center, University of Minnesota
      2000   Phase 2/Phase 3   NCT00176878   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;