285. ファンコニ貧血 Disease details / Clinical trials / Drug dev / DR info

AMD3100
   Children's Hospital Medical Center, Cincinnati
      2007   Phase 1/Phase 2   NCT00479115   United States;
AP1903
   Bellicum Pharmaceuticals, Inc.
      2017   Phase 2   EUCTR2014-000584-41-ES   Italy;Spain;United Kingdom;
      2017   Phase 1;Phase 2   EUCTR2016-003226-16-IT   Italy;
      2014   Phase 1;Phase 2   EUCTR2014-000584-41-IT   Italy;Spain;United Kingdom;
AP1903 A594
   Bellicum Pharmaceuticals, Inc.
      -   Phase 2   EUCTR2014-000584-41-GB   Italy;Spain;United Kingdom;
ATG
   Medical College of Wisconsin
      2009   Phase 2   NCT01071239   United States;
Abatacept
   Emory University
      2014   Phase 1   NCT01917708   United States;
Alefacept
   Emory University
      2010   -   NCT01319851   United States;
Allogeneic Bone Marrow Transplantation
   Fred Hutchinson Cancer Research Center
      2007   Phase 2   NCT00453388   Brazil;United States;
Allogeneic bone marrow transplantation
   Fred Hutchinson Cancer Research Center
      2000   Phase 1   NCT00093743   United States;
      1998   Phase 1   NCT00317876   Brazil;United States;
   Roswell Park Cancer Institute
      2002   Phase 2   NCT00053989   United States;
   University of California, San Francisco
      2000   Phase 1/Phase 2   NCT00305708   United States;
Allogeneic hematopoietic stem cell transplantation
   Fred Hutchinson Cancer Research Center
      2000   Phase 1   NCT00093743   United States;
   Roswell Park Cancer Institute
      2009   -   NCT00856388   United States;
AmCell CliniMACs
   Children's Hospital Medical Center, Cincinnati
      2007   Phase 1/Phase 2   NCT00479115   United States;
Anti-CD45
   Baylor College of Medicine
      2002   Phase 1   NCT00586274   United States;
      2001   Phase 1/Phase 2   NCT00590460   United States;
Anti-Thymocyte Globulin
   Masonic Cancer Center, University of Minnesota
      2000   Phase 1/Phase 2   NCT00630253   United States;
Anti-Thymocyte Globulin (Rabbit)
   Memorial Sloan Kettering Cancer Center
      2018   Phase 2   NCT03600909   United States;
Anti-thymocyte globulin
   Masonic Cancer Center, University of Minnesota
      2002   Phase 2   NCT00258427   United States;
      1999   -   NCT00290628   United States;
   Milton S. Hershey Medical Center
      2003   Phase 2   NCT00084695   United States;
   Roswell Park Cancer Institute
      2009   -   NCT00856388   United States;
      2002   Phase 2   NCT00053989   United States;
   University of California, San Francisco
      2000   Phase 1/Phase 2   NCT00305708   United States;
BPX-501 T cells
   Bellicum Pharmaceuticals
      2014   Phase 2   NCT02065869   Germany;Italy;Spain;United Kingdom;United States;
BPX-501 cells
   Bellicum Pharmaceuticals, Inc.
      2017   Phase 2   EUCTR2014-000584-41-ES   Italy;Spain;United Kingdom;
      2014   Phase 1;Phase 2   EUCTR2014-000584-41-IT   Italy;Spain;United Kingdom;
      -   Phase 2   EUCTR2014-000584-41-GB   Italy;Spain;United Kingdom;
Bone Marrow Aspiration
   Fred Hutchinson Cancer Research Center
      2012   Phase 1   NCT01331018   United States;
   Hospital Infantil Universitario Niño Jesús, Madrid, Spain
      2016   Phase 1/Phase 2   NCT03157804   Spain;
Bone Marrow Transplantation
   Masonic Cancer Center, University of Minnesota
      2006   Phase 2/Phase 3   NCT00352976   United States;
Busulfan
   Children's Hospital Medical Center, Cincinnati
      2014   Phase 2   NCT02143830   United States;
   Masonic Cancer Center, University of Minnesota
      2018   Phase 2   NCT03579875   United States;
      2002   Phase 2   NCT00258427   United States;
      1999   -   NCT00290628   United States;
   Medical College of Wisconsin
      2009   Phase 2   NCT01071239   United States;
   Memorial Sloan Kettering Cancer Center
      2018   Phase 2   NCT03600909   United States;
   Milton S. Hershey Medical Center
      2003   Phase 2   NCT00084695   United States;
   University of California, San Francisco
      2000   Phase 1/Phase 2   NCT00305708   United States;
Busulfan, fludarabine, & cyclophosphamide with immunosuppression with ATG and cyclosporine.
   Memorial Sloan Kettering Cancer Center
      2009   Phase 2   NCT00987480   United States;
CAMPATH-1H
   Baylor College of Medicine
      2001   Phase 1/Phase 2   NCT00590460   United States;
CD34 selected haploidentical PBSCT
   Baylor College of Medicine
      2002   Phase 1   NCT00586274   United States;
CD34+ selected cells
   Neena Kapoor, M.D.
      2011   -   NCT02127905   United States;
CD34+CELLS
   Rocket Pharmaceuticals, Inc.
      2020   Phase 2   EUCTR2018-002502-31-GB   Spain;United Kingdom;
      2018   Phase 2   EUCTR2018-002502-31-ES   Spain;
Campath 1h
   Baylor College of Medicine
      2002   Phase 1   NCT00586274   United States;
Chemotherapy
   Children's Hospital Medical Center, Cincinnati
      2009   Phase 2   NCT01082133   United States;
CliniMACS Prodigy System
   Rajni Agarwal
      2021   Phase 1/Phase 2   NCT04784052   United States;
CliniMACS device
   Memorial Sloan Kettering Cancer Center
      2009   Phase 2   NCT00987480   United States;
CliniMACs device
   Medical College of Wisconsin
      2009   Phase 2   NCT01071239   United States;
Cyclophosphamide
   Children's Hospital Medical Center, Cincinnati
      2014   Phase 2   NCT02143830   United States;
   Fred Hutchinson Cancer Research Center
      2007   Phase 2   NCT00453388   Brazil;United States;
      1998   Phase 1   NCT00317876   Brazil;United States;
   Masonic Cancer Center, University of Minnesota
      2006   Phase 2/Phase 3   NCT00352976   United States;
      2002   Phase 2   NCT00258427   United States;
      2000   Phase 1/Phase 2   NCT00630253   United States;
      1999   -   NCT00290628   United States;
   Medical College of Wisconsin
      2009   Phase 2   NCT01071239   United States;
   Memorial Sloan Kettering Cancer Center
      2018   Phase 2   NCT03600909   United States;
      2001   -   NCT00595127   United States;
   Milton S. Hershey Medical Center
      2003   Phase 2   NCT00084695   United States;
   Rajni Agarwal
      2021   Phase 1/Phase 2   NCT04784052   United States;
   Roswell Park Cancer Institute
      2002   Phase 2   NCT00053989   United States;
Cyclophosphamide (CY) (Plan 1)
   Masonic Cancer Center, University of Minnesota
      2018   Phase 2   NCT03579875   United States;
Cyclophosphamide (CY) (Plan 2)
   Masonic Cancer Center, University of Minnesota
      2018   Phase 2   NCT03579875   United States;
Cyclophosphamide 30
   Columbia University
      2002   Phase 2/Phase 3   NCT01019876   United States;
Cyclophosphamide 40
   Columbia University
      2002   Phase 2/Phase 3   NCT01019876   United States;
Cyclophosphamide, Fludarabine
   Masonic Cancer Center, University of Minnesota
      2004   Phase 1/Phase 2   NCT00167206   United States;
Cyclosporine
   Fred Hutchinson Cancer Research Center
      2007   Phase 2   NCT00453388   Brazil;United States;
      2000   Phase 1   NCT00093743   United States;
      1998   Phase 1   NCT00317876   Brazil;United States;
   Masonic Cancer Center, University of Minnesota
      2000   Phase 1/Phase 2   NCT00630253   United States;
      1999   -   NCT00290628   United States;
Danazol
   Boston Children's Hospital
      2009   Phase 1/Phase 2   NCT01001598   United States;
Depleted Stem Cell Transplant
   Rajni Agarwal
      2021   Phase 1/Phase 2   NCT04784052   United States;
Donor mobilized PBSC infusion
   Masonic Cancer Center, University of Minnesota
      2018   Phase 2   NCT03579875   United States;
ENBREL*SC 4FL 25MG+4SIR 1ML
   ISTITUTO GIANNINA GASLINI
      2005   -   EUCTR2004-004914-18-IT   Italy;
Eltrombopag
   National Heart, Lung, and Blood Institute (NHLBI)
      2018   Phase 2   NCT03206086   United States;
Etanercept
   Children's Hospital Medical Center, Cincinnati
      2005   Early Phase 1   NCT00965666   United States;
   ISTITUTO GIANNINA GASLINI
      2005   -   EUCTR2004-004914-18-IT   Italy;
FP-045
   Foresee Pharmaceuticals Co., Ltd.
      2021   Phase 1/Phase 2   NCT04522375   -
Filgrastim
   ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
      -   Phase 2   EUCTR2014-005264-14-FR   France;
   Fred Hutchinson Cancer Research Center
      2012   Phase 1   NCT01331018   United States;
   Great Ormond Street Hospital NHS foundation Trust
      2015   Phase 2   EUCTR2014-004272-29-GB   France;Spain;United Kingdom;
   Hospital Infantil Universitario Niño Jesús, Madrid, Spain
      2016   Phase 1/Phase 2   NCT03157804   Spain;
   Hospital Universitari Vall d'Hebron Research Institute
      2013   Phase 2   NCT02931071   Spain;
   Masonic Cancer Center, University of Minnesota
      2002   Phase 2   NCT00258427   United States;
      2000   Phase 1/Phase 2   NCT00630253   United States;
      1999   -   NCT00290628   United States;
Fludarabine
   Baylor College of Medicine
      2002   Phase 1   NCT00586274   United States;
      2001   Phase 1/Phase 2   NCT00590460   United States;
   Children's Hospital Medical Center, Cincinnati
      2014   Phase 2   NCT02143830   United States;
   Columbia University
      2002   Phase 2/Phase 3   NCT01019876   United States;
   Masonic Cancer Center, University of Minnesota
      2006   Phase 2/Phase 3   NCT00352976   United States;
      2000   Phase 1/Phase 2   NCT00630253   United States;
   Medical College of Wisconsin
      2009   Phase 2   NCT01071239   United States;
   Memorial Sloan Kettering Cancer Center
      2018   Phase 2   NCT03600909   United States;
      2001   -   NCT00595127   United States;
   Rajni Agarwal
      2021   Phase 1/Phase 2   NCT04784052   United States;
   University of California, San Francisco
      2011   -   NCT01316549   United States;
Fludarabine (FLU)
   Masonic Cancer Center, University of Minnesota
      2018   Phase 2   NCT03579875   United States;
Fludarabine Phosphate
   Fred Hutchinson Cancer Research Center
      2007   Phase 2   NCT00453388   Brazil;United States;
Fludarabine phosphate
   Fred Hutchinson Cancer Research Center
      2000   Phase 1   NCT00093743   United States;
   Masonic Cancer Center, University of Minnesota
      2002   Phase 2   NCT00258427   United States;
   Milton S. Hershey Medical Center
      2003   Phase 2   NCT00084695   United States;
   Roswell Park Cancer Institute
      2009   -   NCT00856388   United States;
      2002   Phase 2   NCT00053989   United States;
   University of California, San Francisco
      2000   Phase 1/Phase 2   NCT00305708   United States;
G-CSF
   ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
      -   Phase 2   EUCTR2014-005264-14-FR   France;
   Assistance Publique - Hôpitaux de Paris
      2017   Phase 1/Phase 2   NCT02678533   France;
   Children's Hospital Medical Center, Cincinnati
      2014   Phase 2   NCT02143830   United States;
   Masonic Cancer Center, University of Minnesota
      2018   Phase 2   NCT03579875   United States;
   Memorial Sloan Kettering Cancer Center
      2018   Phase 2   NCT03600909   United States;
Genetically Engineered Hematopoietic Stem Progenitor Cells
   Fred Hutchinson Cancer Research Center
      2012   Phase 1   NCT01331018   United States;
Genetically Engineered Hematopoietic Stem/Progenitor Cells
   Hospital Infantil Universitario Niño Jesús, Madrid, Spain
      2016   Phase 1/Phase 2   NCT03157804   Spain;
Graft-versus-tumor induction therapy
   Roswell Park Cancer Institute
      2002   Phase 2   NCT00053989   United States;
Hematopoietic Stem Cell Transplant
   Masonic Cancer Center, University of Minnesota
      2004   Phase 1/Phase 2   NCT00167206   United States;
Hematopoietic Stem Cell Transplantation
   Masonic Cancer Center, University of Minnesota
      2000   Phase 1/Phase 2   NCT00630253   United States;
Hematopoietic stem cell transplantation
   Masonic Cancer Center, University of Minnesota
      2002   Phase 2   NCT00258427   United States;
IV administration of Genetically Engineered Hematopoietic Stem/Progenitors Cells (HSPCs)
   Hospital Infantil Universitario Niño Jesús, Madrid, Spain
      2016   Phase 1/Phase 2   NCT03157804   Spain;
JSP191
   Rajni Agarwal
      2021   Phase 1/Phase 2   NCT04784052   United States;
Leukapheresis
   Fred Hutchinson Cancer Research Center
      2012   Phase 1   NCT01331018   United States;
Melphalan
   Masonic Cancer Center, University of Minnesota
      1999   -   NCT00290628   United States;
   Milton S. Hershey Medical Center
      2003   Phase 2   NCT00084695   United States;
   Roswell Park Cancer Institute
      2009   -   NCT00856388   United States;
Metformin HCl
   Boston Children's Hospital
      2018   Phase 2   NCT03398824   United States;
Methotrexate
   Fred Hutchinson Cancer Research Center
      1998   Phase 1   NCT00317876   Brazil;United States;
Methylprednisolone
   Fred Hutchinson Cancer Research Center
      2012   Phase 1   NCT01331018   United States;
   Masonic Cancer Center, University of Minnesota
      2002   Phase 2   NCT00258427   United States;
      2000   Phase 1/Phase 2   NCT00630253   United States;
      1999   -   NCT00290628   United States;
   Milton S. Hershey Medical Center
      2003   Phase 2   NCT00084695   United States;
   Roswell Park Cancer Institute
      2002   Phase 2   NCT00053989   United States;
Methylprednisolone (MP)
   Masonic Cancer Center, University of Minnesota
      2018   Phase 2   NCT03579875   United States;
Miltenyi CliniMACS
   Children's Hospital Medical Center, Cincinnati
      2009   Phase 2   NCT01082133   United States;
Mozobil
   ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
      -   Phase 2   EUCTR2014-005264-14-FR   France;
   Great Ormond Street Hospital NHS foundation Trust
      2015   Phase 2   EUCTR2014-004272-29-GB   France;Spain;United Kingdom;
Mycophenolate mofetil
   Fred Hutchinson Cancer Research Center
      2000   Phase 1   NCT00093743   United States;
   Masonic Cancer Center, University of Minnesota
      1999   -   NCT00290628   United States;
   Roswell Park Cancer Institute
      2002   Phase 2   NCT00053989   United States;
Neupogen
   Great Ormond Street Hospital NHS foundation Trust
      2015   Phase 2   EUCTR2014-004272-29-GB   France;Spain;United Kingdom;
Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation
   Fred Hutchinson Cancer Research Center
      2007   Phase 2   NCT00453388   Brazil;United States;
Nonmyeloablative allogeneic hematopoietic stem cell transplantation
   Fred Hutchinson Cancer Research Center
      1998   Phase 1   NCT00317876   Brazil;United States;
Olaparib
   Academic and Community Cancer Research United
      2020   Phase 2   NCT04042831   United States;
   Baptist Health South Florida
      2020   Phase 2   NCT04483544   United States;
Oxandrolone
   Children's Hospital Medical Center, Cincinnati
      2004   Phase 1   NCT00243399   United States;
Pembrolizumab
   Baptist Health South Florida
      2020   Phase 2   NCT04483544   United States;
Peripheral blood stem cell
   Children's Hospital Medical Center, Cincinnati
      2014   Phase 2   NCT02143830   United States;
Peripheral blood stem cell transplantation
   Fred Hutchinson Cancer Research Center
      2000   Phase 1   NCT00093743   United States;
   Roswell Park Cancer Institute
      2002   Phase 2   NCT00053989   United States;
   University of California, San Francisco
      2000   Phase 1/Phase 2   NCT00305708   United States;
Plerixafor
   Assistance Publique - Hôpitaux de Paris
      2017   Phase 1/Phase 2   NCT02678533   France;
   Fred Hutchinson Cancer Research Center
      2012   Phase 1   NCT01331018   United States;
   Great Ormond Street Hospital NHS foundation Trust
      2015   Phase 2   EUCTR2014-004272-29-GB   France;Spain;United Kingdom;
   Hospital Infantil Universitario Niño Jesús, Madrid, Spain
      2016   Phase 1/Phase 2   NCT03157804   Spain;
   Hospital Universitari Vall d'Hebron Research Institute
      2013   Phase 2   NCT02931071   Spain;
Plérixafor
   ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
      -   Phase 2   EUCTR2014-005264-14-FR   France;
Prednisone
   Fred Hutchinson Cancer Research Center
      2012   Phase 1   NCT01331018   United States;
Quercetin (dietary supplement)
   Children's Hospital Medical Center, Cincinnati
      2018   Phase 2   NCT03476330   United States;
      2012   Phase 1   NCT01720147   United States;
RP-L102
   Rocket Pharmaceuticals Inc.
      2020   Phase 2   NCT04248439   United States;
      2019   Phase 2   NCT04069533   Spain;
      2019   Phase 1   NCT03814408   United States;
   Rocket Pharmaceuticals, Inc.
      2020   Phase 2   EUCTR2018-002502-31-GB   Spain;United Kingdom;
      2018   Phase 2   EUCTR2018-002502-31-ES   Spain;
Rabbit ATG
   Children's Hospital Medical Center, Cincinnati
      2014   Phase 2   NCT02143830   United States;
Rabbit Anti-Thymoglobulin (rATG)
   Rajni Agarwal
      2021   Phase 1/Phase 2   NCT04784052   United States;
Radiation therapy
   Masonic Cancer Center, University of Minnesota
      1999   -   NCT00290628   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;
   Bellicum Pharmaceuticals, Inc.
      2017   Phase 2   EUCTR2014-000584-41-ES   Italy;Spain;United Kingdom;
      -   Phase 2   EUCTR2014-000584-41-GB   Italy;Spain;United Kingdom;
Rituximab
   Masonic Cancer Center, University of Minnesota
      2018   Phase 2   NCT03579875   United States;
   Rajni Agarwal
      2021   Phase 1/Phase 2   NCT04784052   United States;
Rivogenlecleucel
   Bellicum Pharmaceuticals
      2017   Phase 1/Phase 2   NCT03733249   Italy;Saudi Arabia;United Kingdom;
   Bellicum Pharmaceuticals, Inc.
      -   Phase 2   EUCTR2014-000584-41-GB   Italy;Spain;United Kingdom;
Rivogenleucleucel
   Bellicum Pharmaceuticals, Inc.
      2017   Phase 2   EUCTR2014-000584-41-ES   Italy;Spain;United Kingdom;
Sargramostim
   Roswell Park Cancer Institute
      2002   Phase 2   NCT00053989   United States;
Sirolimus
   Masonic Cancer Center, University of Minnesota
      2006   Phase 2/Phase 3   NCT00352976   United States;
Stem cell infusion
   Baylor College of Medicine
      2001   Phase 1/Phase 2   NCT00590460   United States;
T cell infusion
   Baylor College of Medicine
      2002   Phase 1   NCT00586274   United States;
Tacrolimus
   Roswell Park Cancer Institute
      2002   Phase 2   NCT00053989   United States;
The CliniMACS device
   Memorial Sloan Kettering Cancer Center
      2018   Phase 2   NCT03600909   United States;
Therapeutic allogeneic lymphocytes
   Roswell Park Cancer Institute
      2002   Phase 2   NCT00053989   United States;
Thiotepa
   University of California, San Francisco
      2017   -   NCT03609840   United States;
Thymic Shielding During Radiation
   Masonic Cancer Center, University of Minnesota
      2004   Phase 1/Phase 2   NCT00167206   United States;
Total Body Irradiation
   Masonic Cancer Center, University of Minnesota
      2006   Phase 2/Phase 3   NCT00352976   United States;
      2004   Phase 1/Phase 2   NCT00167206   United States;
Total Body Irradiation (TBI) (Plan 1)
   Masonic Cancer Center, University of Minnesota
      2018   Phase 2   NCT03579875   United States;
Transduced CD34+ Cells
   National Heart, Lung, and Blood Institute (NHLBI)
      1993   Phase 1   NCT00001399   United States;
Umbilical cord blood transplantation
   Masonic Cancer Center, University of Minnesota
      1999   -   NCT00290628   United States;
   Roswell Park Cancer Institute
      2002   Phase 2   NCT00053989   United States;
   University of California, San Francisco
      2000   Phase 1/Phase 2   NCT00305708   United States;
Zarzio
   ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
      -   Phase 2   EUCTR2014-005264-14-FR   France;