18. 脊髄小脳変性症（多系統萎縮症を除く。） Disease details / Clinical trials / Drug dev / DR info

Aavrh.10hfxn, serotype RH.10 adeno-associated virus (AAV) gene transfer vector expressing THE cdna coding FOR human FXN
   Weill Medical College of Cornell University
      2022   Phase 1   NCT05302271   United States
Alpha-tocotrienol quinone
   PTC Therapeutics, Inc.
      2023   Phase 3   EUCTR2022-002668-65-FR   Australia;Brazil;Canada;France;Germany;Italy;New Zealand;Spain;United States
      -   Phase 3   EUCTR2022-002668-65-DE   Australia;Brazil;Canada;France;Germany;Italy;New Zealand;Spain;United States
Alpha-tocotrienolquinone
   PTC Therapeutics, Inc.
      2021   Phase 2;Phase 3   EUCTR2020-002812-36-FR   Australia;Brazil;Canada;France;Germany;Italy;New Zealand;Spain;United States
      2021   Phase 2;Phase 3   EUCTR2020-002812-36-ES   Australia;Brazil;Canada;France;Germany;Italy;Spain;United States
      2021   Phase 2;Phase 3   EUCTR2020-002812-36-DE   Australia;Brazil;Canada;France;Germany;Italy;New Zealand;Spain;United States
Artesunate
   INSERM
      2021   Phase 1;Phase 2   EUCTR2021-000171-36-FR   France
Artesunate oral product
   Institut National de la Santé Et de la Recherche Médicale, France
      2022   Phase 1/Phase 2   NCT04921930   France
ASP2016
   Astellas Gene Therapies
      2024   Phase 1   NCT06483802   United States
Baclofen
   DAIICHI SANKYO COMPANY, LIMITED
      2005   Phase 4   JPRN-jRCT1080220031   -
Balance training BY means OF conventional rehabilitation
   IRCCS Eugenio Medea
      2025   -   NCT06692296   Italy
Balance training over stabilometric platform
   IRCCS Eugenio Medea
      2025   -   NCT06692296   Italy
Binocrit*1SIR 40000UI/1ML
   UNIVERSITA' DEGLI STUDI DI NAPOLI FEDERICO II
      2012   Phase 2   EUCTR2011-006156-37-IT   Italy
Blood analysis FOR frataxin'S level measurement
   Berta Alemany
      2021   Phase 4   NCT04801303   Spain
Blood analysis FOR hypercalcemia'S control
   Berta Alemany
      2021   Phase 4   NCT04801303   Spain
Bupropion & citalopram
   University of Colorado, Denver
      2012   Phase 4   NCT01716221   -
Cabaletta FOR IV infusion once weekly during 24 weeks
   Bioblast Pharma Ltd.
      2014   Phase 2   NCT02147886   Israel
Calcitriol
   Berta Alemany
      2021   Phase 4   NCT04801303   Spain
CTI-1601
   Larimar Therapeutics, Inc.
      2024   Phase 2   NCT06447025   United States
      2022   Phase 2   NCT05579691   United States
      2020   Phase 1   NCT04519567   United States
      2019   Phase 1   NCT04176991   United States
Deferiprone
   APOPHARMA INC., TORONTO, ONTARIO
      2009   -   EUCTR2009-010865-22-IT   Belgium;France;Italy;Spain
      2008   -   EUCTR2007-003331-23-IT   Belgium;France;Italy;Spain;United Kingdom
DT-216
   Design Therapeutics
      2022   Phase 1   NCT05285540   United States
   Design Therapeutics, Inc.
      2022   Phase 1   NCT05573698   United States
DT-216p2
   Design Therapeutics, Inc.
      2025   Phase 1/Phase 2   NCT06874010   Australia
      2025   Phase 1   NCT06772870   Australia
EGB 761 (R) 120 MG
   BEAUFOUR IPSEN PHARMA
      2008   Phase 2   EUCTR2007-005371-34-FR   France
EGB 761 120 MG
   Ipsen
      2008   Phase 2   NCT00824512   France
Elamipretide
   Children's Hospital of Philadelphia
      2022   Phase 1/Phase 2   NCT05168774   United States
Epoeitin beta
   Medizinische Universität Innsbruck, Univ.-Klinik für Neurologie
      2009   Phase 2   EUCTR2008-000040-13-AT   Austria
Epoetin alfa
   Federico II University
      2013   Phase 2   NCT01493973   Italy
   UNIVERSITA' DEGLI STUDI DI NAPOLI FEDERICO II
      2012   Phase 2   EUCTR2011-006156-37-IT   Italy
Eprex*1FL 40000UI/ML 1ML
   ISTITUTO NEUROLOGICO CARLO BESTA
      2007   -   EUCTR2007-003357-85-IT   Italy
Eprex*1SIR 10000UI 1ML
   UNIVERSITA' DEGLI STUDI DI NAPOLI FEDERICO II
      2012   Phase 2   EUCTR2011-006156-37-IT   Italy
Eprex*1SIR 40000UI/ML 1ML
   UNIVERSITA' DEGLI STUDI DI NAPOLI FEDERICO II
      2012   Phase 2   EUCTR2011-006156-37-IT   Italy
Erythropoietin
   ISTITUTO NEUROLOGICO CARLO BESTA
      2007   -   EUCTR2007-003357-85-IT   Italy
Etravirina
   ASSOCIAZIONE LA NOSTRA FAMIGLIA - SEZIONE SCIENTIFICA I.R.C.C.S. E.MEDEA
      2020   Phase 2   EUCTR2019-002618-38-IT   Italy
Etravirine tablets
   IRCCS Eugenio Medea
      2020   Phase 2   NCT04273165   Italy
EU1/97/031-032
   Medizinische Universität Innsbruck, Univ.-Klinik für Neurologie
      2009   Phase 2   EUCTR2008-000040-13-AT   Austria
EU1/97/031/031-032
   Dr. Sylvia Boesch
      2006   Phase 2   EUCTR2005-005938-12-AT   Austria
Exercise intervention
   Children's Hospital of Philadelphia
      2020   -   NCT04192136   United States
Flumazenil
   Okayama University Graduate School of Medicine, Dentistry and Pharmaceutical Sciences
      2013   -   JPRN-UMIN000011111   Japan
Gamma interferon
   Azienda Policlinico Umberto I
      2013   Phase 2   NCT02035020   Italy
   IRCCS Eugenio Medea
      2016   Phase 2   NCT03888664   -
Ginkgo biloba
   BEAUFOUR IPSEN PHARMA
      2008   Phase 2   EUCTR2007-005371-34-FR   France
Granocyte
   University of Bristol
      2018   Phase 2   EUCTR2017-003084-34-GB   United Kingdom
H-116PI-DE.PDF
   Medizinische Universität Innsbruck, Univ.-Klinik für Neurologie
      2009   Phase 2   EUCTR2008-000040-13-AT   Austria
High dose LX2006
   Lexeo Therapeutics
      2022   Phase 1/Phase 2   NCT05445323   Canada;United States
Idebenone
   National Institute of Neurological Disorders and Stroke (NINDS)
      2005   Phase 2   NCT00229632   United States
      2004   Phase 1   NCT00078481   United States
      2001   Phase 1   NCT00015808   United States
Imukin - 6 flaconi 0.5 ML 100 MCG
   ASSOCIAZIONE LA NOSTRA FAMIGLIA - SEZIONE SCIENTIFICA I.R.C.C.S. E.MEDEA
      2016   Phase 2   EUCTR2015-002432-40-IT   Italy
Imukin*SC 6F 0,5ML 100MCG
   AZIENDA UNIVERSITARIA POLICLINICO UMBERTO I DI ROMA
      2013   -   EUCTR2012-001881-14-IT   Italy
Intelence - 200 MG - compressa -USO orale - flacone (hdpe) 60 compresse
   ASSOCIAZIONE LA NOSTRA FAMIGLIA - SEZIONE SCIENTIFICA I.R.C.C.S. E.MEDEA
      2020   Phase 2   EUCTR2019-002618-38-IT   Italy
Interferon gamma-1B
   AZIENDA UNIVERSITARIA POLICLINICO UMBERTO I DI ROMA
      2013   -   EUCTR2012-001881-14-IT   Italy
   Children's Hospital of Philadelphia
      2013   Phase 2   NCT01965327   United States
Iron chelating intervention
   Assistance Publique - Hôpitaux de Paris
      2005   Phase 1/Phase 2   NCT00224640   France
KPS-0373
   Kissei Pharmaceutical Co., Ltd.
      2016   Phase 3   NCT02889302   Japan
      2011   Phase 2   NCT01384435   Japan
      2009   Phase 2   NCT01004016   Japan
      2004   Phase 2   NCT00863538   Japan
KPS-0373, high dose
   Kissei Pharmaceutical Co., Ltd.
      2013   Phase 3   NCT01970137   Japan
      2013   Phase 3   NCT01970124   Japan
      2013   Phase 3   NCT01970111   Japan
      2013   Phase 3   NCT01970098   Japan
KPS-0373, LOW dose
   Kissei Pharmaceutical Co., Ltd.
      2013   Phase 3   NCT01970137   Japan
      2013   Phase 3   NCT01970124   Japan
      2013   Phase 3   NCT01970111   Japan
      2013   Phase 3   NCT01970098   Japan
Lenograstim
   University of Bristol
      2018   Phase 2   EUCTR2017-003084-34-GB   United Kingdom
Lithium carbonate
   Hospital de Clinicas de Porto Alegre
      2011   Phase 2/Phase 3   NCT01096082   Brazil
LOW dose LX2006
   Lexeo Therapeutics
      2022   Phase 1/Phase 2   NCT05445323   Canada;United States
LU AA24493
   H. Lundbeck A/S
      2009   -   EUCTR2008-003662-25-AT   Austria;Germany;Italy
      -   -   EUCTR2008-003662-25-DE   Austria;Germany;Italy
   H.Lundbeck A/S
      2009   -   EUCTR2008-003662-25-IT   Austria;Germany;Italy
Methylprednisolone
   Children's Hospital of Philadelphia
      2015   Early Phase 1   NCT02424435   United States
MIB-626
   Metro International Biotech, LLC
      2021   Phase 2   NCT04817111   United States
MID dose LX2006
   Lexeo Therapeutics
      2022   Phase 1/Phase 2   NCT05445323   Canada;United States
MIN-102
   Minoryx Therapeutics, S.L.
      2019   Phase 2   NCT03917225   Belgium;France;Germany;Spain
NEO-recormon
   Dr. Sylvia Boesch
      2006   Phase 2   EUCTR2005-005938-12-AT   Austria
Neorecormon
   Medizinische Universität Innsbruck, Univ.-Klinik für Neurologie
      2009   Phase 2   EUCTR2008-000040-13-AT   Austria
Nicotinamide
   RWTH Aachen University
      2023   Phase 2   NCT03761511   Austria;France;Germany;Italy;Spain;United Kingdom
   RWTH Aachen University represented by the Center for Translational & Clinical Research Aachen (CTC-A)
      2018   Phase 2;Phase 3   EUCTR2017-002163-17-DE   Austria;Germany;Spain;United Kingdom
   RWTH Aachen University represented by the Rector himself, represented by the Dean of the Medical Faculty
      2020   Phase 2;Phase 3   EUCTR2017-002163-17-ES   Austria;Germany;Spain;United Kingdom
      2019   Phase 2;Phase 3   EUCTR2017-002163-17-AT   Austria;Germany
   RWTH Aachen University, Center for Translational & Clinical Research Aachen (CTC-A)
      2020   Phase 2;Phase 3   EUCTR2017-002163-17-GB   Austria;Germany;Spain;United Kingdom
Nicotinamide riboside
   Children's Hospital of Philadelphia
      2020   -   NCT04192136   United States
Nomlabofusp
   Larimar Therapeutics, Inc.
      2024   Phase 1   NCT06681766   United States
Omaveloxolone
   Biogen
      2026   -   NCT06628687   United States
      2025   Phase 3   NCT06953583   Australia;Austria;Brazil;Canada;Denmark;France;Germany;Ireland;Italy;Netherlands;Spain;United Kingdom;United States
      2024   -   NCT06623890   Austria;Belgium;Czechia;Germany;Italy;Netherlands;United States
      2024   Phase 1   NCT06054893   United States
Omaveloxolone capsules, 10 MG
   Reata, a wholly owned subsidiary of Biogen
      2015   Phase 2   NCT02255435   Australia;Austria;Brazil;Italy;United Kingdom;United States
Omaveloxolone capsules, 150 MG
   Reata, a wholly owned subsidiary of Biogen
      2015   Phase 2   NCT02255435   Australia;Austria;Brazil;Italy;United Kingdom;United States
Omaveloxolone capsules, 160 MG
   Reata, a wholly owned subsidiary of Biogen
      2015   Phase 2   NCT02255435   Australia;Austria;Brazil;Italy;United Kingdom;United States
Omaveloxolone capsules, 2.5 MG
   Reata, a wholly owned subsidiary of Biogen
      2015   Phase 2   NCT02255435   Australia;Austria;Brazil;Italy;United Kingdom;United States
Omaveloxolone capsules, 20 MG
   Reata, a wholly owned subsidiary of Biogen
      2015   Phase 2   NCT02255435   Australia;Austria;Brazil;Italy;United Kingdom;United States
Omaveloxolone capsules, 300 MG
   Reata, a wholly owned subsidiary of Biogen
      2015   Phase 2   NCT02255435   Australia;Austria;Brazil;Italy;United Kingdom;United States
Omaveloxolone capsules, 40 MG
   Reata, a wholly owned subsidiary of Biogen
      2015   Phase 2   NCT02255435   Australia;Austria;Brazil;Italy;United Kingdom;United States
Omaveloxolone capsules, 5 MG
   Reata, a wholly owned subsidiary of Biogen
      2015   Phase 2   NCT02255435   Australia;Austria;Brazil;Italy;United Kingdom;United States
Omaveloxolone capsules, 80 MG
   Reata, a wholly owned subsidiary of Biogen
      2015   Phase 2   NCT02255435   Australia;Austria;Brazil;Italy;United Kingdom;United States
Prednisolone
   Astellas Gene Therapies
      2024   Phase 1   NCT06483802   United States
Prednisone
   Weill Medical College of Cornell University
      2022   Phase 1   NCT05302271   United States
PTC743
   PTC THERAPEUTICS INC.
      2023   Phase 3   EUCTR2022-002668-65-IT   Australia;Brazil;Canada;France;Germany;Italy;New Zealand;Spain;United States
      2021   Phase 2;Phase 3   EUCTR2020-002812-36-IT   Australia;Brazil;Canada;France;Germany;Italy;Spain;United States
   PTC Therapeutics, Inc.
      2023   Phase 3   EUCTR2022-002668-65-FR   Australia;Brazil;Canada;France;Germany;Italy;New Zealand;Spain;United States
      2021   Phase 2;Phase 3   EUCTR2020-002812-36-FR   Australia;Brazil;Canada;France;Germany;Italy;New Zealand;Spain;United States
      2021   Phase 2;Phase 3   EUCTR2020-002812-36-ES   Australia;Brazil;Canada;France;Germany;Italy;Spain;United States
      2021   Phase 2;Phase 3   EUCTR2020-002812-36-DE   Australia;Brazil;Canada;France;Germany;Italy;New Zealand;Spain;United States
      -   Phase 3   EUCTR2022-002668-65-DE   Australia;Brazil;Canada;France;Germany;Italy;New Zealand;Spain;United States
Resveratrol
   Murdoch Childrens Research Institute
      2019   Phase 2   NCT03933163   Australia
      2011   Phase 1/Phase 2   NCT01339884   Australia
RHUG-CSF
   University of Bristol
      2018   Phase 2   EUCTR2017-003084-34-GB   United Kingdom
Rokan (R) novo 120 MG
   BEAUFOUR IPSEN PHARMA
      2008   Phase 2   EUCTR2007-005371-34-FR   France
Rosuvastatin
   Children's Hospital of Philadelphia
      2016   Early Phase 1   NCT02705547   United States
Saline
   Design Therapeutics, Inc.
      2025   Phase 1   NCT06772870   Australia
SUB09246MIG
   RWTH Aachen University, Center for Translational & Clinical Research Aachen (CTC-A)
      2020   Phase 2;Phase 3   EUCTR2017-002163-17-GB   Austria;Germany;Spain;United Kingdom
TAK-831
   Neurocrine Biosciences
      2017   Phase 2   NCT03214588   United States
Varenicline
   Niigata University
      2013   Phase 1,2   JPRN-UMIN000011560   Japan
Vatiquinone
   PTC THERAPEUTICS INC.
      2023   Phase 3   EUCTR2022-002668-65-IT   Australia;Brazil;Canada;France;Germany;Italy;New Zealand;Spain;United States
      2021   Phase 2;Phase 3   EUCTR2020-002812-36-IT   Australia;Brazil;Canada;France;Germany;Italy;Spain;United States
   PTC Therapeutics
      2022   Phase 3   NCT05515536   Australia;Brazil;Canada;France;Germany;Italy;New Zealand;Spain;United States
      2022   Phase 2   NCT05485987   United States
      2020   Phase 2/Phase 3   NCT04577352   Australia;Brazil;Canada;France;Germany;Italy;New Zealand;Spain;United States
   PTC Therapeutics, Inc.
      2023   Phase 3   EUCTR2022-002668-65-FR   Australia;Brazil;Canada;France;Germany;Italy;New Zealand;Spain;United States
      2021   Phase 2;Phase 3   EUCTR2020-002812-36-FR   Australia;Brazil;Canada;France;Germany;Italy;New Zealand;Spain;United States
      2021   Phase 2;Phase 3   EUCTR2020-002812-36-ES   Australia;Brazil;Canada;France;Germany;Italy;Spain;United States
      2021   Phase 2;Phase 3   EUCTR2020-002812-36-DE   Australia;Brazil;Canada;France;Germany;Italy;New Zealand;Spain;United States
      -   Phase 3   EUCTR2022-002668-65-DE   Australia;Brazil;Canada;France;Germany;Italy;New Zealand;Spain;United States