DR info

臨床試験数 : 61 / 薬物数 : 90 - (DrugBank : 31) / 標的遺伝子数 : 23 - 標的パスウェイ数 : 126

薬物ごとの開発者（Primary Sponsor）、臨床試験情報（抜粋）です。

5-[4-[2-(5-(1-hydroxyethyl)-2- pyridinyl)ethoxy]benzyl]-2,4- thiazolidinedione hydrochloride
   Minoryx Therapeutics S.L.
      2020   Phase 2   EUCTR2019-000654-59-FR   France;Germany;Spain;United States;
      2019   Phase 2   EUCTR2019-000654-59-ES   Germany;Spain;
      2019   Phase 2   EUCTR2019-000654-59-DE   Argentina;France;Germany;Spain;United States;
5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride
   Minoryx Therapeutics S.L.
      2018   Phase 2;Phase 3   EUCTR2017-000748-16-PL   France;Germany;Hungary;Italy;Netherlands;Poland;Spain;United Kingdom;United States;
      2018   Phase 2;Phase 3   EUCTR2017-000748-16-DE   France;Germany;Hungary;Italy;Netherlands;Poland;Spain;United Kingdom;United States;
      2017   Phase 2;Phase 3   EUCTR2017-000748-16-NL   France;Germany;Hungary;Italy;Netherlands;Poland;Spain;United Kingdom;United States;
      2017   Phase 2;Phase 3   EUCTR2017-000748-16-HU   France;Germany;Hungary;Italy;Netherlands;Spain;United Kingdom;United States;
      2017   Phase 2;Phase 3   EUCTR2017-000748-16-GB   France;Germany;Hungary;Italy;Netherlands;Poland;Spain;United Kingdom;United States;
      2017   Phase 2;Phase 3   EUCTR2017-000748-16-ES   France;Germany;Hungary;Italy;Netherlands;Spain;United Kingdom;United States;
5-[[4-[2-[5-(1-HYDROXYETHYL)-2-PYRIDINYL]ETHOXY]PHENYL]METHYL]-2,4-THIAZOLIDINEDIONE HYDROCHLORIDE
   Minoryx Therapeutics S.L.
      2020   Phase 2   EUCTR2019-000654-59-FR   France;Germany;Spain;United States;
      2019   Phase 2   EUCTR2019-000654-59-ES   Germany;Spain;
      2019   Phase 2   EUCTR2019-000654-59-DE   Argentina;France;Germany;Spain;United States;
AUTOLOGOUS CD34+ CELLS TRANSDUCED WITH LENTI-D VECTOR ENCODING ABCD1 CDNA
   bluebird bio, Inc
      2021   Phase 3   EUCTR2015-002805-13-NL   Argentina;Australia;Brazil;France;Netherlands;United Kingdom;United States;
   bluebird bio, Inc.
      2018   Phase 2;Phase 3   EUCTR2011-001953-10-DE   France;Germany;United Kingdom;United States;
      -   Phase 3   EUCTR2015-002805-13-FR   Algeria;Argentina;Australia;France;United Kingdom;United States;
Albumin solution
   Onofre, Aurora Pujol, M.D.
      2020   Phase 2/Phase 3   NCT04303416   Spain;
Albunorm® ,
   Aurora Pujol Onofre
      2020   Phase 2   EUCTR2019-004733-17-ES   Spain;
Alemtuzumab
   Masonic Cancer Center, University of Minnesota
      2006   Phase 2   NCT00383448   United States;
Allogeneic stem cell transplantation
   Masonic Cancer Center, University of Minnesota
      2009   Phase 2   NCT01043640   United States;
Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA
   BLUEBIRD BIO, INC.
      2019   Phase 3   EUCTR2018-001145-14-IT   France;Germany;Italy;Netherlands;United Kingdom;United States;
   bluebird bio, Inc.
      2020   Phase 3   EUCTR2018-001145-14-DE   France;Germany;Italy;Netherlands;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-001145-14-NL   France;Germany;Italy;Netherlands;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-001145-14-GB   France;Germany;Italy;Netherlands;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-001145-14-FR   France;Germany;Italy;Netherlands;United Kingdom;United States;
Bezafibrate
   Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
      2010   -   NCT01165060   Netherlands;
Busulfan
   Masonic Cancer Center, University of Minnesota
      2009   Phase 2   NCT01043640   United States;
Busulfan, Cyclophosphamide, Antithymocyte Globulin
   Masonic Cancer Center, University of Minnesota
      1995   Phase 2/Phase 3   NCT00176904   United States;
CALD HR-D (High-Risk, Regimen C)
   Masonic Cancer Center, University of Minnesota
      2014   Phase 2   NCT02171104   United States;
CALD HR-D (High-Risk, Regimen D)
   Masonic Cancer Center, University of Minnesota
      2014   Phase 2   NCT02171104   United States;
CALD SR-A (Standard-Risk, Regimen A)
   Masonic Cancer Center, University of Minnesota
      2014   Phase 2   NCT02171104   United States;
CALD SR-B (Standard-Risk, Regimen B)
   Masonic Cancer Center, University of Minnesota
      2014   Phase 2   NCT02171104   United States;
Campath-1H
   Masonic Cancer Center, University of Minnesota
      2009   Phase 2   NCT01043640   United States;
Chenodeoxycholic acid
   Children's Hospital Research Foundation University of Cincinnati
      1997   -   NCT00004442   -
Cholic Acids
   Travere Therapeutics, Inc.
      1992   Phase 3   NCT00007020   United States;
Clofarabine
   Masonic Cancer Center, University of Minnesota
      2006   Phase 2   NCT00383448   United States;
Cyclophosphamide
   Masonic Cancer Center, University of Minnesota
      2009   Phase 2   NCT01043640   United States;
Cyclosporine A
   Masonic Cancer Center, University of Minnesota
      2009   Phase 2   NCT01043640   United States;
      2006   Phase 2   NCT00383448   United States;
D-BIOTIN
   MEDDAY SAS
      2014   Phase 2;Phase 3   EUCTR2014-000698-38-ES   Spain;
DUOC-01
   Joanne Kurtzberg, MD
      2014   Phase 1   NCT02254863   United States;
Elivaldogene autotemcel
   BLUEBIRD BIO, INC.
      2019   Phase 3   EUCTR2018-001145-14-IT   France;Germany;Italy;Netherlands;United Kingdom;United States;
   bluebird bio, Inc
      2021   Phase 3   EUCTR2015-002805-13-NL   Argentina;Australia;Brazil;France;Netherlands;United Kingdom;United States;
   bluebird bio, Inc.
      2020   Phase 3   EUCTR2018-001145-14-DE   France;Germany;Italy;Netherlands;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-001145-14-NL   France;Germany;Italy;Netherlands;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-001145-14-GB   France;Germany;Italy;Netherlands;United Kingdom;United States;
Elivaldogenum tavalentivecum
   bluebird bio, Inc.
      -   Phase 3   EUCTR2015-002805-13-FR   Algeria;Argentina;Australia;France;United Kingdom;United States;
Glyceryl trierucate/glyceryl trioleate
   Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
      1998   Phase 2   NCT00004418   United States;
Hematopoietic Stem Cell Transplantation
   Masonic Cancer Center, University of Minnesota
      2006   Phase 2   NCT00383448   United States;
Hematopoietic stem cell infusion
   Talaris Therapeutics Inc.
      2011   Phase 1/Phase 2   NCT01372228   United States;
Human Placental Derived Stem Cell
   New York Medical College
      2013   Phase 1   NCT01586455   United States;
Hydroxyurea
   Masonic Cancer Center, University of Minnesota
      2006   Phase 2   NCT00383448   United States;
IMD Preparative Regimen
   Masonic Cancer Center, University of Minnesota
      2014   Phase 2   NCT02171104   United States;
Interferon beta
   FDA Office of Orphan Products Development
      1998   -   NCT00004450   -
Lenti-D Drug Product
   BLUEBIRD BIO, INC.
      2019   Phase 3   EUCTR2018-001145-14-IT   France;Germany;Italy;Netherlands;United Kingdom;United States;
   bluebird bio, Inc
      2021   Phase 3   EUCTR2015-002805-13-NL   Argentina;Australia;Brazil;France;Netherlands;United Kingdom;United States;
   bluebird bio, Inc.
      2020   Phase 3   EUCTR2018-001145-14-DE   France;Germany;Italy;Netherlands;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-001145-14-NL   France;Germany;Italy;Netherlands;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-001145-14-GB   France;Germany;Italy;Netherlands;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-001145-14-FR   France;Germany;Italy;Netherlands;United Kingdom;United States;
      2018   Phase 2;Phase 3   EUCTR2011-001953-10-DE   France;Germany;United Kingdom;United States;
      2015   Phase 1;Phase 2;Phase 3   EUCTR2011-001953-10-FR   Argentina;Australia;France;Germany;United Kingdom;United States;
      -   Phase 3   EUCTR2015-002805-13-FR   Algeria;Argentina;Australia;France;United Kingdom;United States;
Leriglitazone
   Minoryx Therapeutics S.L.
      2020   Phase 2   EUCTR2019-000654-59-FR   France;Germany;Spain;United States;
      2019   Phase 2   EUCTR2019-000654-59-ES   Germany;Spain;
      2019   Phase 2   EUCTR2019-000654-59-DE   Argentina;France;Germany;Spain;United States;
Lipoic acid
   Onofre, Aurora Pujol, M.D.
      2011   Phase 2   NCT01495260   Spain;
Lorenzo's Oil
   University of Minnesota
      2011   -   NCT02233257   United States;
Lorenzo's oil
   Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
      2005   Phase 3   NCT00545597   United States;
MD1003
   MEDDAY SAS
      2014   Phase 2;Phase 3   EUCTR2014-000698-38-ES   Spain;
MD1003 100 mg capsule
   MedDay Pharmaceuticals SA
      2014   Phase 2/Phase 3   NCT02961803   France;Germany;Spain;
MIN-102
   Minoryx Therapeutics S.L.
      2018   Phase 2;Phase 3   EUCTR2017-000748-16-PL   France;Germany;Hungary;Italy;Netherlands;Poland;Spain;United Kingdom;United States;
      2018   Phase 2;Phase 3   EUCTR2017-000748-16-DE   France;Germany;Hungary;Italy;Netherlands;Poland;Spain;United Kingdom;United States;
      2017   Phase 2;Phase 3   EUCTR2017-000748-16-NL   France;Germany;Hungary;Italy;Netherlands;Poland;Spain;United Kingdom;United States;
      2017   Phase 2;Phase 3   EUCTR2017-000748-16-HU   France;Germany;Hungary;Italy;Netherlands;Spain;United Kingdom;United States;
      2017   Phase 2;Phase 3   EUCTR2017-000748-16-GB   France;Germany;Hungary;Italy;Netherlands;Poland;Spain;United Kingdom;United States;
      2017   Phase 2;Phase 3   EUCTR2017-000748-16-ES   France;Germany;Hungary;Italy;Netherlands;Spain;United Kingdom;United States;
   Minoryx Therapeutics, S.L.
      2019   Phase 2   NCT04528706   France;Germany;Spain;
      2017   Phase 2/Phase 3   NCT03231878   France;Germany;Hungary;Italy;Netherlands;Spain;United Kingdom;United States;
Melphalan
   Masonic Cancer Center, University of Minnesota
      2006   Phase 2   NCT00383448   United States;
Mesenchymal Stem Cells
   Masonic Cancer Center, University of Minnesota
      2015   Phase 1   NCT02410239   -
Modified cobratoxin
   ReceptoPharm Inc.
      2006   Phase 3   EUCTR2004-002200-14-GB   United Kingdom;
Mycophenylate mofetil
   Masonic Cancer Center, University of Minnesota
      2006   Phase 2   NCT00383448   United States;
N-acetylcysteine
   Onofre, Aurora Pujol, M.D.
      2011   Phase 2   NCT01495260   Spain;
NV1205
   NeuroVia, Inc.
      -   Phase 1   EUCTR2017-001684-21-FR   Argentina;Australia;Chile;Colombia;France;Russian Federation;Ukraine;United Kingdom;
   Neurov Acquisition LLC
      2018   Phase 1   EUCTR2017-001684-21-GB   Argentina;Australia;Chile;Colombia;France;Russian Federation;Ukraine;United Kingdom;
Osteopetrosis Haploidentical Only Preparative Regimen
   Masonic Cancer Center, University of Minnesota
      2014   Phase 2   NCT02171104   United States;
Osteopetrosis Only Preparative Regimen
   Masonic Cancer Center, University of Minnesota
      2014   Phase 2   NCT02171104   United States;
PXL065
   Poxel SA
      2022   Phase 2   NCT05200104   -
PXL770
   Poxel SA
      2022   Phase 2   NCT05146284   -
Pioglitazone
   Onofre, Aurora Pujol, M.D.
      2016   Phase 2   NCT03864523   Spain;
Pramipexole
   Massachusetts General Hospital
      2021   Phase 4   NCT05003648   Netherlands;United States;
QTXEW2-04-AF, EW2, GC-1, QRX-431
   NeuroVia, Inc.
      -   Phase 1   EUCTR2017-001684-21-FR   Argentina;Australia;Chile;Colombia;France;Russian Federation;Ukraine;United Kingdom;
   Neurov Acquisition LLC
      2018   Phase 1   EUCTR2017-001684-21-GB   Argentina;Australia;Chile;Colombia;France;Russian Federation;Ukraine;United Kingdom;
RPI78M for injection
   ReceptoPharm Inc.
      2006   Phase 3   EUCTR2004-002200-14-GB   United Kingdom;
Sobetirome
   NeuroVia, Inc.
      -   Phase 1   EUCTR2017-001684-21-FR   Argentina;Australia;Chile;Colombia;France;Russian Federation;Ukraine;United Kingdom;
   Neurov Acquisition LLC
      2018   Phase 1   EUCTR2017-001684-21-GB   Argentina;Australia;Chile;Colombia;France;Russian Federation;Ukraine;United Kingdom;
   Thomas S. Scanlan
      2013   Phase 1   NCT01787578   United States;
Sobetirome (NV1205)
   NeuroVia, Inc.
      2018   Phase 1/Phase 2   NCT03196765   Argentina;Australia;Chile;Colombia;France;Russian Federation;Ukraine;United Kingdom;
Stem Cell Transplant
   Masonic Cancer Center, University of Minnesota
      1995   Phase 2/Phase 3   NCT00176904   United States;
Stem Cell Transplantation
   Masonic Cancer Center, University of Minnesota
      2014   Phase 2   NCT02171104   United States;
Thalidomide
   FDA Office of Orphan Products Development
      1998   -   NCT00004450   -
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
      2006   Phase 2   NCT00383448   United States;
Ursodiol
   Children's Hospital Research Foundation University of Cincinnati
      1997   -   NCT00004442   -
VK0214
   Viking Therapeutics, Inc.
      2021   Phase 1   NCT04973657   United States;
Vitamin D3
   Stanford University
      2016   Phase 1   NCT02595489   United States;
Vitamin E
   Onofre, Aurora Pujol, M.D.
      2011   Phase 2   NCT01495260   Spain;