21. ミトコンドリア病 Disease details / Clinical trials / Drug dev / DR info

2-isopropyl-3H-naphtho[2,1-D]imidazole-4,5-dione
   Abliva AB
      2023   Phase 3   EUCTR2021-002846-33-BE   Belgium;Canada;Denmark;France;Germany;Italy;Spain;Turkey;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002846-33-DK   Belgium;Canada;Denmark;France;Germany;Italy;Spain;Turkey;United Kingdom;United States
2-{[(8Z)-13-[(methylcarbamoyl)formamido]- tridec-8-EN-1-YL]OXY}acetic acid
   OMEICOS Therapeutics GmbH
      2023   Phase 2   EUCTR2022-003307-16-IT   Germany;Italy
2-{[(8Z)-13-[(methylcarbamoyl)formamido]-tridec-8-EN-1-YL]OXY}acetic acid
   OMEICOS Therapeutics GmbH
      2023   Phase 2   EUCTR2022-003307-16-DE   Germany;Italy
ABI-009
   Aadi, LLC
      2022   Phase 2   NCT03747328   -
ALFA-tocotrienolo chinone
   PTC THERAPEUTICS INC.
      2022   Phase 3   EUCTR2022-000375-39-IT   Australia;Canada;France;Italy;Poland;Spain;Sweden;United Kingdom;United States
Alpha-tocotrienol quinone
   PTC THERAPEUTICS, INC.
      2023   Phase 3   EUCTR2022-000375-39-FR   France;Italy;Japan;Poland;Spain;Sweden;United Kingdom;United States
      2023   Phase 3   EUCTR2022-000375-39-ES   France;Italy;Poland;Spain;Sweden;United Kingdom;United States
      2021   Phase 2;Phase 3   EUCTR2020-002100-39-FR   France;Germany;Italy;Poland;Spain;Sweden;United Kingdom;United States
      2021   Phase 2;Phase 3   EUCTR2020-002100-39-ES   France;Germany;Italy;Spain;United Kingdom;United States
      -   Phase 3   EUCTR2022-000375-39-SE   France;Italy;Japan;Poland;Spain;Sweden;United Kingdom;United States
      -   Phase 3   EUCTR2022-000375-39-PL   France;Italy;Japan;Poland;Spain;Sweden;United Kingdom;United States
      -   Phase 2;Phase 3   EUCTR2020-002100-39-SE   Australia;Canada;France;Germany;Italy;Japan;Poland;Russian Federation;Spain;Sweden;United Kingdom;United States
      -   Phase 2;Phase 3   EUCTR2020-002100-39-PL   France;Germany;Italy;Poland;Spain;Sweden;United Kingdom;United States
Arginine and citrulline supplementations
   Baylor College of Medicine
      2009   Phase 0   NCT01339494   United States
Blood drawing
   Charite University, Berlin, Germany
      2020   -   NCT06967831   Germany
Bupropion
   Abliva AB
      2020   Phase 1   NCT04643249   United Kingdom
Caffeine
   Abliva AB
      2020   Phase 1   NCT04643249   United Kingdom
Cardiopulmonary exercise testing
   Children's Hospital of Philadelphia
      2023   Phase 1   NCT05569122   United States
CD34+ cells enriched with MNV-BLD
   Minovia Therapeutics Ltd.
      2019   Phase 1/Phase 2   NCT03384420   Israel
Coenzymeq10
   University of Florida
      2007   Phase 3   NCT00432744   Canada;United States
Creatine chemical exchange saturation transfer (crcest) imaging sequence
   Children's Hospital of Philadelphia
      2021   -   NCT04734626   United States
Cysteamine bitartrate
   Amgen
      2015   Phase 2   NCT02473445   United States
      2014   Phase 2   NCT02023866   United States
Dexmedetomidine
   The University of Texas Health Science Center, Houston
      2014   -   NCT02053766   United States
Dextromethorphan
   Abliva AB
      2020   Phase 1   NCT04643249   United Kingdom
Dichloroacetate
   Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
      2000   Phase 2   NCT00068913   United States
Elamipretide
   STEALTH BIOTHERAPEUTICS
      2022   Phase 3   EUCTR2021-003907-16-IT   Australia;Denmark;Finland;Germany;Hungary;Italy;Netherlands;Norway;Spain;United Kingdom;United States
   Stealth BioTherapeutics Inc.
      2022   Phase 3   NCT05162768   Australia;Germany;Hungary;Italy;Netherlands;New Zealand;Norway;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2021-003907-16-NO   Australia;Denmark;Finland;Germany;Hungary;Italy;Netherlands;Norway;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2021-003907-16-NL   Australia;Germany;Hungary;Italy;Netherlands;New Zealand;Norway;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2021-003907-16-HU   Australia;Denmark;Finland;Germany;Hungary;Italy;Netherlands;Norway;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2021-003907-16-ES   Australia;Denmark;Finland;Germany;Hungary;Italy;Netherlands;Norway;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2021-003907-16-DE   Australia;Germany;Hungary;Italy;Netherlands;New Zealand;Norway;Spain;United Kingdom;United States
      2016   Phase 2   NCT02976038   United States
      2016   Phase 2   NCT02805790   United States
      2015   Phase 1/Phase 2   NCT02367014   United States
EPI-743
   Edison Pharmaceuticals Inc
      2014   Phase 2   NCT02104336   United States
   National Human Genome Research Institute (NHGRI)
      2012   Phase 1/Phase 2   NCT01642056   United States
   PTC Therapeutics
      2014   Phase 2   NCT02352896   United States
EPI-743 15 MG/KG
   PTC Therapeutics
      2012   Phase 2   NCT01721733   United States
EPI-743 5 MG/KG
   PTC Therapeutics
      2012   Phase 2   NCT01721733   United States
EPI743
   PTC THERAPEUTICS INC.
      2022   Phase 3   EUCTR2022-000375-39-IT   Australia;Canada;France;Italy;Poland;Spain;Sweden;United Kingdom;United States
   PTC THERAPEUTICS, INC.
      2023   Phase 3   EUCTR2022-000375-39-FR   France;Italy;Japan;Poland;Spain;Sweden;United Kingdom;United States
      2023   Phase 3   EUCTR2022-000375-39-ES   France;Italy;Poland;Spain;Sweden;United Kingdom;United States
      2021   Phase 2;Phase 3   EUCTR2020-002100-39-FR   France;Germany;Italy;Poland;Spain;Sweden;United Kingdom;United States
      2021   Phase 2;Phase 3   EUCTR2020-002100-39-ES   France;Germany;Italy;Spain;United Kingdom;United States
      -   Phase 3   EUCTR2022-000375-39-SE   France;Italy;Japan;Poland;Spain;Sweden;United Kingdom;United States
      -   Phase 3   EUCTR2022-000375-39-PL   France;Italy;Japan;Poland;Spain;Sweden;United Kingdom;United States
      -   Phase 2;Phase 3   EUCTR2020-002100-39-SE   Australia;Canada;France;Germany;Italy;Japan;Poland;Russian Federation;Spain;Sweden;United Kingdom;United States
      -   Phase 2;Phase 3   EUCTR2020-002100-39-PL   France;Germany;Italy;Poland;Spain;Sweden;United Kingdom;United States
Exercise pedal
   Children's Hospital of Philadelphia
      2023   Phase 1   NCT05569122   United States
Feburane
   FONDAZIONE IRCCS ISTITUTO NEUROLOGICO CARLO BESTA
      2019   Phase 2   EUCTR2018-001094-25-IT   Italy
Flurbiprofen
   Abliva AB
      2020   Phase 1   NCT04643249   United Kingdom
Generation OF ipscs
   Charite University, Berlin, Germany
      2020   -   NCT06967831   Germany
Gentle jogger
   Children's Hospital of Philadelphia
      2023   Phase 1   NCT05569122   United States
Glutamine oral supplement
   Hospital Universitario 12 de Octubre
      2021   -   NCT05255328   Spain
Glutamine oral supplementation
   Jesús González de la Aleja Tejera
      2021   -   NCT04948138   Spain
Glycerol tributyrate
   George Washington University
      2025   Phase 1/Phase 2   NCT06792500   United States
Idebenone
   Michio Hirano
      2009   Phase 2   NCT00887562   United States
IW-6463 tablets
   Tisento Therapeutics
      2020   Phase 2   NCT04475549   United States
Ketogenic diet
   Second Affiliated Hospital, School of Medicine, Zhejiang University
      2023   -   NCT06013397   China
KH176
   Khondrion B.V.
      2022   Phase 2   EUCTR2020-000832-23-DK   Denmark;Germany;Netherlands;United Kingdom
      2021   Phase 2   EUCTR2020-003124-16-NL   Czechia;Netherlands
      2021   Phase 2   EUCTR2020-000832-23-NL   Denmark;Germany;Netherlands;United Kingdom
      2021   Phase 2   EUCTR2019-000599-40-DK   Denmark;Germany;Netherlands;United Kingdom
      2020   Phase 2   EUCTR2019-000599-40-GB   Denmark;Germany;Netherlands;United Kingdom
      2020   Phase 2   EUCTR2019-000599-40-DE   Denmark;Germany;Netherlands;United Kingdom
      2019   Phase 2   EUCTR2019-000599-40-NL   Germany;Netherlands;United Kingdom
   Khondrion BV
      2019   Phase 2   NCT04165239   Denmark;Germany;Netherlands;United Kingdom
      2016   Phase 2   NCT02909400   Netherlands
      2016   Phase 2   EUCTR2016-001696-79-NL   Netherlands
      2015   Phase 1   NCT02544217   Belgium
KL1333
   Abliva AB
      2023   Phase 3   EUCTR2021-002846-33-BE   Belgium;Canada;Denmark;France;Germany;Italy;Spain;Turkey;United Kingdom;United States
      2022   Phase 2   NCT05650229   Belgium;Denmark;France;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2021-002846-33-DK   Belgium;Canada;Denmark;France;Germany;Italy;Spain;Turkey;United Kingdom;United States
      2020   Phase 1   NCT04643249   United Kingdom
      2019   Phase 1   NCT03888716   United Kingdom
KL1333 100 MG
   Yungjin Pharm. Co., Ltd.
      2017   Phase 1   NCT03056209   Korea, Republic of
KL1333 200 MG
   Yungjin Pharm. Co., Ltd.
      2017   Phase 1   NCT03056209   Korea, Republic of
KL1333 25 MG
   Yungjin Pharm. Co., Ltd.
      2017   Phase 1   NCT03056209   Korea, Republic of
KL1333 400 MG
   Yungjin Pharm. Co., Ltd.
      2017   Phase 1   NCT03056209   Korea, Republic of
KL1333 50 MG
   Yungjin Pharm. Co., Ltd.
      2017   Phase 1   NCT03056209   Korea, Republic of
KL1333 600 MG
   Yungjin Pharm. Co., Ltd.
      2017   Phase 1   NCT03056209   Korea, Republic of
KL1333 800 MG
   Yungjin Pharm. Co., Ltd.
      2017   Phase 1   NCT03056209   Korea, Republic of
L-arginine
   Kurume UniversityYasutoshi Koga
      2008   Phase 3   JPRN-jRCT2091220023   Japan
   The Hospital for Sick Children
      2012   Phase 2   NCT01603446   Canada
   Yasutoshi Koga, Department of Pediatrics and Child Health, Kurume University School of Medicine
      2009   Phase 3   JPRN-jRCT1091220025   Japan
L-citrulline
   Baylor College of Medicine
      2021   Phase 1   NCT03952234   United States
Lactose
   Japan Agency of Medical Research and Development
      2016   -   JPRN-UMIN000025772   Japan
LOW residue diet intervention
   Newcastle University
      2017   -   NCT03388528   United Kingdom
Lumason® contrast agent
   Children's Hospital of Philadelphia
      2023   Phase 1   NCT05569122   United States
Medium-chain triglycerides
   The University of Texas Health Science Center, Houston
      2010   Phase 0   NCT01252979   United States
Midazolam injection
   Abliva AB
      2020   Phase 1   NCT04643249   United Kingdom
MNV-201
   Minovia Therapeutics Ltd.
      2023   Phase 1   NCT06017869   Israel
MTP-131
   STEALTH BIOTHERAPEUTICS
      2022   Phase 3   EUCTR2021-003907-16-IT   Australia;Denmark;Finland;Germany;Hungary;Italy;Netherlands;Norway;Spain;United Kingdom;United States
   Stealth BioTherapeutics Inc.
      2022   Phase 3   EUCTR2021-003907-16-NO   Australia;Denmark;Finland;Germany;Hungary;Italy;Netherlands;Norway;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2021-003907-16-NL   Australia;Germany;Hungary;Italy;Netherlands;New Zealand;Norway;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2021-003907-16-HU   Australia;Denmark;Finland;Germany;Hungary;Italy;Netherlands;Norway;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2021-003907-16-ES   Australia;Denmark;Finland;Germany;Hungary;Italy;Netherlands;Norway;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2021-003907-16-DE   Australia;Germany;Hungary;Italy;Netherlands;New Zealand;Norway;Spain;United Kingdom;United States
N-acetylcysteine
   Michio Hirano, MD
      2023   Phase 1   NCT05241262   United States
Nicotinamide riboside
   Cambridge University Hospitals NHS Foundation Trust
      2017   -   NCT03432871   United Kingdom
OFF-label compassionate drug USE
   Charite University, Berlin, Germany
      2020   -   NCT06967831   Germany
Omeprazole
   Abliva AB
      2020   Phase 1   NCT04643249   United Kingdom
OMT-28
   OMEICOS Therapeutics GmbH
      2023   Phase 2   EUCTR2022-003307-16-IT   Germany;Italy
      2023   Phase 2   EUCTR2022-003307-16-DE   Germany;Italy
   Omeicos Therapeutics GmbH
      2023   Phase 2   NCT05972954   Germany;Italy;Netherlands
Oral administration OF 100 MG KH176 twice daily
   Khondrion BV
      2021   Phase 2   NCT04604548   Denmark;Germany;Netherlands;United Kingdom
PGZ BED
   Children's Hospital of Philadelphia
      2023   Phase 1   NCT05569122   United States
Pheburane
   FONDAZIONE IRCCS ISTITUTO NEUROLOGICO CARLO BESTA
      2019   Phase 2   EUCTR2018-001094-25-IT   Italy
Probucol
   RiboNova Inc.
      2025   -   NCT06931834   United States
Propofol
   The University of Texas Health Science Center, Houston
      2014   -   NCT02053766   United States
PTC743
   PTC THERAPEUTICS INC.
      2022   Phase 3   EUCTR2022-000375-39-IT   Australia;Canada;France;Italy;Poland;Spain;Sweden;United Kingdom;United States
      2021   Phase 2;Phase 3   EUCTR2020-002100-39-IT   France;Germany;Italy;Spain;Sweden;United Kingdom;United States
   PTC THERAPEUTICS, INC.
      2023   Phase 3   EUCTR2022-000375-39-FR   France;Italy;Japan;Poland;Spain;Sweden;United Kingdom;United States
      2023   Phase 3   EUCTR2022-000375-39-ES   France;Italy;Poland;Spain;Sweden;United Kingdom;United States
      2021   Phase 2;Phase 3   EUCTR2020-002100-39-FR   France;Germany;Italy;Poland;Spain;Sweden;United Kingdom;United States
      2021   Phase 2;Phase 3   EUCTR2020-002100-39-ES   France;Germany;Italy;Spain;United Kingdom;United States
      -   Phase 3   EUCTR2022-000375-39-SE   France;Italy;Japan;Poland;Spain;Sweden;United Kingdom;United States
      -   Phase 3   EUCTR2022-000375-39-PL   France;Italy;Japan;Poland;Spain;Sweden;United Kingdom;United States
      -   Phase 2;Phase 3   EUCTR2020-002100-39-SE   Australia;Canada;France;Germany;Italy;Japan;Poland;Russian Federation;Spain;Sweden;United Kingdom;United States
      -   Phase 2;Phase 3   EUCTR2020-002100-39-PL   France;Germany;Italy;Poland;Spain;Sweden;United Kingdom;United States
   Vinay Penematsa
      2023   Phase 3   JPRN-jRCT2011230045   France;Italy;Japan;Poland;Spain;UK;US
Reagila
   Semmelweis University, STIA-POC-2020
      2021   Phase 4   EUCTR2021-001268-58-HU   Hungary
Repaglinide
   Abliva AB
      2020   Phase 1   NCT04643249   United Kingdom
Sevoflurane
   The University of Texas Health Science Center, Houston
      2014   -   NCT02053766   United States
   d sessler
      2006   -   NCT01001585   United States
Sirolimus
   Matthew Demczko
      2025   Phase 2   NCT06843811   United States
Skin biopsy
   Charite University, Berlin, Germany
      2020   -   NCT06967831   Germany
Sonlicromanol
   Khondrion B.V.
      2022   Phase 2   EUCTR2020-000832-23-DK   Denmark;Germany;Netherlands;United Kingdom
      2021   Phase 2   EUCTR2020-003124-16-NL   Czechia;Netherlands
      2021   Phase 2   EUCTR2020-000832-23-NL   Denmark;Germany;Netherlands;United Kingdom
   Khondrion BV
      2025   Phase 3   NCT06451757   -
      2021   Phase 2   NCT04846036   Netherlands
Taurine
   Department of Neurology, Kawasaki Medical School
      2012   -   JPRN-UMIN000009358   Japan
      2011   -   JPRN-UMIN000005592   Japan
   Kawasaki Medical School
      2017   -   JPRN-UMIN000029376   Japan
   Kawasaki Medical School Hospital
      2015   Phase 3   JPRN-UMIN000019195   Japan
      2014   Phase 3   JPRN-UMIN000015538   Japan
      2013   Phase 3   JPRN-UMIN000011908   Japan
   Sunada Yoshihide
      2017   Phase 3   JPRN-jRCTs061180015   -
TRI-O-acetyluridine
   Repligen Corporation
      2003   Phase 1   NCT00060515   United States
TTI-0102
   Thiogenesis Therapeutics, Inc.
      2025   Phase 2   NCT06644534   France;Netherlands
Vatiquinone
   PTC THERAPEUTICS INC.
      2022   Phase 3   EUCTR2022-000375-39-IT   Australia;Canada;France;Italy;Poland;Spain;Sweden;United Kingdom;United States
      2021   Phase 2;Phase 3   EUCTR2020-002100-39-IT   France;Germany;Italy;Spain;Sweden;United Kingdom;United States
   PTC THERAPEUTICS, INC.
      2023   Phase 3   EUCTR2022-000375-39-FR   France;Italy;Japan;Poland;Spain;Sweden;United Kingdom;United States
      2023   Phase 3   EUCTR2022-000375-39-ES   France;Italy;Poland;Spain;Sweden;United Kingdom;United States
      2021   Phase 2;Phase 3   EUCTR2020-002100-39-FR   France;Germany;Italy;Poland;Spain;Sweden;United Kingdom;United States
      2021   Phase 2;Phase 3   EUCTR2020-002100-39-ES   France;Germany;Italy;Spain;United Kingdom;United States
      -   Phase 3   EUCTR2022-000375-39-SE   France;Italy;Japan;Poland;Spain;Sweden;United Kingdom;United States
      -   Phase 3   EUCTR2022-000375-39-PL   France;Italy;Japan;Poland;Spain;Sweden;United Kingdom;United States
      -   Phase 2;Phase 3   EUCTR2020-002100-39-SE   Australia;Canada;France;Germany;Italy;Japan;Poland;Russian Federation;Spain;Sweden;United Kingdom;United States
      -   Phase 2;Phase 3   EUCTR2020-002100-39-PL   France;Germany;Italy;Poland;Spain;Sweden;United Kingdom;United States
   PTC Therapeutics
      2022   Phase 3   NCT05218655   France;Italy;Japan;Poland;Spain;United Kingdom;United States
      2020   Phase 2/Phase 3   NCT04378075   Canada;France;Italy;Japan;Poland;Spain;Sweden;United Kingdom;United States
   Vinay Penematsa
      2023   Phase 3   JPRN-jRCT2011230045   France;Italy;Japan;Poland;Spain;UK;US
      2022   Phase 2-3   JPRN-jRCT2011210075   Canada;France;Italy;Japan;Poland;Spain;Sweden;UK;USA
Zagociguat 15MG
   Tisento Therapeutics
      2025   Phase 2   NCT06961344   -
      2024   Phase 2   NCT06402123   Australia;Canada;Germany;Italy;United Kingdom;United States
Zagociguat 30MG
   Tisento Therapeutics
      2024   Phase 2   NCT06402123   Australia;Canada;Germany;Italy;United Kingdom;United States