20. 副腎白質ジストロフィー Disease details / Clinical trials / Drug dev / DR info

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
      2017   Phase 2;Phase 3   EUCTR2017-000748-16-IT   France;Germany;Hungary;Italy;Netherlands;Poland;Spain;United Kingdom;United States
   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;Poland;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
Albumin
   Aurora Pujol Onofre
      2020   Phase 2   EUCTR2019-004733-17-ES   Spain
Albumin solution
   Onofre, Aurora Pujol, M.D.
      2020   Phase 2/Phase 3   NCT04303416   Spain
Alemtuzumab
   Masonic Cancer Center, University of Minnesota
      2006   Phase 2   NCT00383448   United States
ALLO-hsct
   bluebird bio
      2015   -   NCT02204904   Argentina;Canada;Germany;Italy;Netherlands;United Kingdom;United States
Allogeneic stem cell transplantation
   Masonic Cancer Center, University of Minnesota
      2009   Phase 2   NCT01043640   United States
Autologous CD34+ cells transduced with lenti-D vector encoding ABCD1 cdna
   BLUEBIRD BIO, INC.
      2022   Phase 3   EUCTR2015-002805-13-IT   Argentina;Australia;Brazil;Canada;France;Germany;Italy;Netherlands;United Kingdom;United States
   bluebird bio, Inc
      2021   Phase 3   EUCTR2015-002805-13-NL   Argentina;Australia;Brazil;France;Germany;Italy;Netherlands;United Kingdom;United States
      2020   Phase 3   EUCTR2015-002805-13-DE   Argentina;Australia;Brazil;Canada;France;Germany;Italy;Netherlands;United Kingdom;United States
   bluebird bio, Inc.
      2018   Phase 2;Phase 3   EUCTR2011-001953-10-DE   France;Germany;United Kingdom;United States
      2017   Phase 3   EUCTR2015-002805-13-FR   Algeria;Argentina;Australia;France;Germany;Italy;Netherlands;United Kingdom;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
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 acid
   Children's Hospital Research Foundation University of Cincinnati
      1997   -   NCT00004442   -
Cholic acids
   Mirum Pharmaceuticals, 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
Dimethyl fumarate
   Pujol, Aurora, M.D.
      2024   Phase 2/Phase 3   NCT06513533   Spain
DUOC-01
   Joanne Kurtzberg, MD
      2014   Phase 1   NCT02254863   United States
Elivaldogene autotemcel
   BLUEBIRD BIO, INC.
      2022   Phase 3   EUCTR2015-002805-13-IT   Argentina;Australia;Brazil;Canada;France;Germany;Italy;Netherlands;United Kingdom;United States
      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;Germany;Italy;Netherlands;United Kingdom;United States
      2020   Phase 3   EUCTR2015-002805-13-DE   Argentina;Australia;Brazil;Canada;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
Elivaldogenum tavalentivecum
   bluebird bio, Inc.
      2017   Phase 3   EUCTR2015-002805-13-FR   Algeria;Argentina;Australia;France;Germany;Italy;Netherlands;United Kingdom;United States
Glyceryl trierucate
   FDA Office of Orphan Products Development
      1998   -   NCT00004450   -
Glyceryl trierucate/glyceryl trioleate
   Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
      1998   Phase 2   NCT00004418   United States
Glyceryl trioleate
   FDA Office of Orphan Products Development
      1998   -   NCT00004450   -
Hematopoietic stem cell infusion
   Talaris Therapeutics Inc.
      2011   Phase 1/Phase 2   NCT01372228   United States
Hematopoietic stem cell transplantation
   Masonic Cancer Center, University of Minnesota
      2006   Phase 2   NCT00383448   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
Imitation procedure
   SwanBio Therapeutics, Inc.
      2022   Phase 1/Phase 2   NCT05394064   Netherlands;United States
Interferon beta
   FDA Office of Orphan Products Development
      1998   -   NCT00004450   -
Intrathecal and intravenous LV gene therapy
   Shenzhen Geno-Immune Medical Institute
      2018   -   NCT03727555   China
Lenti-D
   bluebird bio
      2019   Phase 3   NCT03852498   France;Germany;Italy;Netherlands;United Kingdom;United States
Lenti-D drug product
   BLUEBIRD BIO, INC.
      2022   Phase 3   EUCTR2015-002805-13-IT   Argentina;Australia;Brazil;Canada;France;Germany;Italy;Netherlands;United Kingdom;United States
      2019   Phase 3   EUCTR2018-001145-14-IT   France;Germany;Italy;Netherlands;United Kingdom;United States
   bluebird bio
      2013   Phase 2/Phase 3   NCT01896102   Argentina;Australia;France;Germany;United Kingdom;United States
   bluebird bio, Inc
      2021   Phase 3   EUCTR2015-002805-13-NL   Argentina;Australia;Brazil;France;Germany;Italy;Netherlands;United Kingdom;United States
      2020   Phase 3   EUCTR2015-002805-13-DE   Argentina;Australia;Brazil;Canada;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
      2018   Phase 2;Phase 3   EUCTR2011-001953-10-DE   France;Germany;United Kingdom;United States
      2017   Phase 3   EUCTR2015-002805-13-FR   Algeria;Argentina;Australia;France;Germany;Italy;Netherlands;United Kingdom;United States
      2015   Phase 1;Phase 2;Phase 3   EUCTR2011-001953-10-FR   Argentina;Australia;France;Germany;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
   Minoryx Therapeutics, S.L.
      2023   Phase 3   NCT05819866   Argentina;Brazil;France;Germany;India;Spain;United Kingdom;United States
Lipoic acid
   Onofre, Aurora Pujol, M.D.
      2011   Phase 2   NCT01495260   Spain
Lorenzo'S OIL
   Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
      2005   Phase 3   NCT00545597   United States
   University of Minnesota
      2011   -   NCT02233257   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
Melphalan
   Masonic Cancer Center, University of Minnesota
      2006   Phase 2   NCT00383448   United States
Mesenchymal stem cells
   Masonic Cancer Center, University of Minnesota
      2023   Phase 1   NCT06030648   United States
      2015   Phase 1   NCT02410239   -
MIN-102
   MINORYX THERAPEUTICS
      2017   Phase 2;Phase 3   EUCTR2017-000748-16-IT   France;Germany;Hungary;Italy;Netherlands;Poland;Spain;United Kingdom;United States
   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
      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;Poland;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   Argentina;France;Germany;Spain
      2017   Phase 2/Phase 3   NCT03231878   France;Germany;Hungary;Italy;Netherlands;Spain;United Kingdom;United States
Modified cobratoxin
   ReceptoPharm Inc.
      2006   Phase 3   EUCTR2004-002200-14-GB   United Kingdom
Mycophenolate mofetil
   Masonic Cancer Center, University of Minnesota
      2009   Phase 2   NCT01043640   United States
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
NO interventional drug product utilized IN this follow-UP study
   bluebird bio
      2016   -   NCT02698579   Algeria;Argentina;Australia;Brazil;France;Germany;Italy;Netherlands;United Kingdom;United States
NV1205
   NeuroVia, Inc.
      2018   Phase 1/Phase 2   NCT03196765   Argentina;Australia;Chile;Colombia;France;Russian Federation;Ukraine;United Kingdom
      -   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
Pioglitazone
   AURORA PUJOL ONOFRE
      2013   -   EUCTR2011-006113-34-ES   Spain
   Onofre, Aurora Pujol, M.D.
      2016   Phase 2   NCT03864523   Spain
Plerixafor
   bluebird bio, Inc.
      2020   Phase 3   EUCTR2018-001145-14-DE   France;Germany;Italy;Netherlands;United Kingdom;United States
Pramipexole
   Massachusetts General Hospital
      2023   Phase 4   NCT05003648   Netherlands;United States
PXL065
   Poxel SA
      2023   Phase 2   NCT05200104   -
PXL770
   Poxel S.A.
      2022   Phase 2   EUCTR2021-006223-18-NL   Germany;Netherlands
      2022   Phase 2   EUCTR2021-006223-18-DE   Germany;Netherlands
   Poxel SA
      2023   Phase 2   NCT05146284   -
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
SBT101
   SwanBio Therapeutics, Inc.
      2022   Phase 1/Phase 2   NCT05394064   Netherlands;United States
Sobetirome
   NeuroVia, Inc.
      2018   Phase 1/Phase 2   NCT03196765   Argentina;Australia;Chile;Colombia;France;Russian Federation;Ukraine;United Kingdom
      -   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
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
Transduced CD34+ hematopoietic stem cell
   Shenzhen Second People's Hospital
      2015   Phase 1/Phase 2   NCT02559830   China
Ursodiol
   Children's Hospital Research Foundation University of Cincinnati
      1997   -   NCT00004442   -
Vitamin D3
   Stanford University
      2016   Phase 1   NCT02595489   United States
Vitamin E
   Onofre, Aurora Pujol, M.D.
      2011   Phase 2   NCT01495260   Spain
VK0214
   Viking Therapeutics, Inc.
      2021   Phase 1   NCT04973657   France;Germany;Italy;United Kingdom;United States