302. レーベル遺伝性視神経症 Disease details / Clinical trials / Drug dev / DR info
臨床試験数 : 41 / 薬物数 : 22 - (DrugBank : 9) / 標的遺伝子数 : 5 - 標的パスウェイ数 : 33
薬物、開発者(Primary Sponsor)、臨床試験情報(症例登録開始年(Enrollment year)、フェーズ、ID、実施国)
Béfizal
Hôpital Necker-Enfants Malades
2019 Phase 2/Phase 3 NCT04561466 France
Cyclosporine
University Hospital, Angers
2011 Phase 2 NCT02176733 France
Glycerol tributyrate
George Washington University
2025 Phase 1/Phase 2 NCT06792500 United States
GS010
GENSIGHT BIOLOGICS
2019 Phase 3 EUCTR2017-002187-40-NL Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-IT Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-GB Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-FR Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-ES Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-BE Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 4 EUCTR2017-002153-11-IT France;Germany;Italy;United Kingdom;United States
2018 Phase 3 EUCTR2017-002153-11-GB France;Germany;Italy;United Kingdom;United States
2016 Phase 3 EUCTR2015-001266-26-IT France;Germany;Italy;United Kingdom;United States
2016 Phase 3 EUCTR2015-001265-11-IT France;Germany;Italy;United Kingdom;United States
GENSIGHT-BIOLOGICS
2016 Phase 3 EUCTR2015-001266-26-DE France;Germany;Italy;United Kingdom;United States
2016 Phase 3 EUCTR2015-001265-11-DE France;Germany;Italy;United Kingdom;United States
2015 Phase 3 EUCTR2015-001266-26-GB France;Germany;Italy;United Kingdom;United States
2015 Phase 3 EUCTR2015-001265-11-GB France;Germany;Italy;United Kingdom;United States
2013 Phase 1;Phase 2 EUCTR2013-001405-90-FR France
GenSight Biologics
2019 - NCT03672968 -
2014 Phase 1/Phase 2 NCT02064569 France
Idebenone
Beijing Tongren Hospital, CMU
2018 - ChiCTR-IPR-17013821 China
Hyogo College of Medicine
2013 Phase 1,2 JPRN-UMIN000017939 Japan
SANTHERA PHARMACEUTICALS
- Phase 4 EUCTR2015-004405-16-IT Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain;United Kingdom
Santhera Pharmaceuticals
2016 Phase 4 NCT02774005 Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain;United Kingdom;United States
2016 - NCT02771379 Austria;France;Germany;Greece;Italy;Netherlands
Santhera Pharmaceuticals (Switzerland) Limited
2017 Phase 4 EUCTR2015-004405-16-BG Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain;United Kingdom;United States
2016 Phase 4 EUCTR2015-004405-16-PT Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain;United Kingdom;United States
2016 Phase 4 EUCTR2015-004405-16-ES Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain
2016 Phase 4 EUCTR2015-004405-16-DE Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain;United Kingdom;United States
2016 Phase 4 EUCTR2015-004405-16-BE Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain;United Kingdom;United States
2016 Phase 4 EUCTR2015-004405-16-AT Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain;United Kingdom;United States
- Phase 4 EUCTR2015-004405-16-PL Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain;United Kingdom;United States
IEM-EP
Region Stockholm
2008 - NCT06376279 -
Injection needle
Neurophth Therapeutics Inc
2023 Phase 1/Phase 2 NCT05293626 United States
KH176
Khondrion BV
2015 Phase 1 NCT02544217 Belgium
Lenadogene nolparvovec
GENSIGHT BIOLOGICS
2019 Phase 3 EUCTR2017-002187-40-NL Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-IT Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-GB Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-FR Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-ES Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-BE Belgium;France;Italy;Netherlands;Spain;United Kingdom
Mecobalamin
Beijing Tongren Hospital, CMU
2018 - ChiCTR-IPR-17013821 China
Nadh
GENSIGHT BIOLOGICS
2019 Phase 3 EUCTR2017-002187-40-NL Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-GB Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-FR Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-ES Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-BE Belgium;France;Italy;Netherlands;Spain;United Kingdom
NFS-02 injection
Neurophth Therapeutics Inc
2023 Phase 1/Phase 2 NCT05820152 China;United States
NR082 injection
Neurophth Therapeutics Inc
2023 Phase 1/Phase 2 NCT05293626 United States
Wuhan Neurophth Biotechnology Limited Company
2021 Phase 2/Phase 3 NCT04912843 China
Pending
GENSIGHT BIOLOGICS
2016 Phase 3 EUCTR2015-001265-11-IT France;Germany;Italy;United Kingdom;United States
GENSIGHT-BIOLOGICS
2016 Phase 3 EUCTR2015-001266-26-DE France;Germany;Italy;United Kingdom;United States
2016 Phase 3 EUCTR2015-001265-11-DE France;Germany;Italy;United Kingdom;United States
2015 Phase 3 EUCTR2015-001266-26-GB France;Germany;Italy;United Kingdom;United States
2015 Phase 3 EUCTR2015-001265-11-GB France;Germany;Italy;United Kingdom;United States
RAAV2-ND1
Taihe Hospital, Hubei University of Medicine, Shiyan, Hubei Province, China
2021 Phase 0 ChiCTR2000041574 China
RAAV2-ND4
Bin Li
2018 - NCT03428178 China
2011 - NCT01267422 China
Huazhong University of Science and Technology
2017 Phase 2/Phase 3 NCT03153293 China
RAAV2/2-ND4 vector
GENSIGHT BIOLOGICS
2016 Phase 3 EUCTR2015-001266-26-IT France;Germany;Italy;United Kingdom;United States
2016 Phase 3 EUCTR2015-001265-11-IT France;Germany;Italy;United Kingdom;United States
GENSIGHT-BIOLOGICS
2016 Phase 3 EUCTR2015-001266-26-DE France;Germany;Italy;United Kingdom;United States
2016 Phase 3 EUCTR2015-001265-11-DE France;Germany;Italy;United Kingdom;United States
2015 Phase 3 EUCTR2015-001266-26-GB France;Germany;Italy;United Kingdom;United States
2015 Phase 3 EUCTR2015-001265-11-GB France;Germany;Italy;United Kingdom;United States
2013 Phase 1;Phase 2 EUCTR2013-001405-90-FR France
Raxone
SANTHERA PHARMACEUTICALS
- Phase 4 EUCTR2015-004405-16-IT Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain;United Kingdom
Santhera Pharmaceuticals (Switzerland) Limited
2017 Phase 4 EUCTR2015-004405-16-BG Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain;United Kingdom;United States
2016 Phase 4 EUCTR2015-004405-16-PT Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain;United Kingdom;United States
2016 Phase 4 EUCTR2015-004405-16-ES Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain
2016 Phase 4 EUCTR2015-004405-16-DE Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain;United Kingdom;United States
2016 Phase 4 EUCTR2015-004405-16-BE Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain;United Kingdom;United States
2016 Phase 4 EUCTR2015-004405-16-AT Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain;United Kingdom;United States
- Phase 4 EUCTR2015-004405-16-PL Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain;United Kingdom;United States
Recombinant AAV vector serotype 2 containing THE human wild type mitochondrial ND4 gene
GENSIGHT BIOLOGICS
2019 Phase 3 EUCTR2017-002187-40-NL Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-GB Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-FR Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-ES Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-BE Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 4 EUCTR2017-002153-11-IT France;Germany;Italy;United Kingdom;United States
2018 Phase 3 EUCTR2017-002153-11-GB France;Germany;Italy;United Kingdom;United States
2016 Phase 3 EUCTR2015-001266-26-IT France;Germany;Italy;United Kingdom;United States
2016 Phase 3 EUCTR2015-001265-11-IT France;Germany;Italy;United Kingdom;United States
GENSIGHT-BIOLOGICS
2016 Phase 3 EUCTR2015-001266-26-DE France;Germany;Italy;United Kingdom;United States
2016 Phase 3 EUCTR2015-001265-11-DE France;Germany;Italy;United Kingdom;United States
2015 Phase 3 EUCTR2015-001266-26-GB France;Germany;Italy;United Kingdom;United States
2015 Phase 3 EUCTR2015-001265-11-GB France;Germany;Italy;United Kingdom;United States
2013 Phase 1;Phase 2 EUCTR2013-001405-90-FR France
Recombinant AAV vector serotype 2 containing THE human wild typemitochondrial ND4 gene
GENSIGHT BIOLOGICS
2018 Phase 3 EUCTR2017-002187-40-IT Belgium;France;Italy;Netherlands;Spain;United Kingdom
Sham injection
Wuhan Neurophth Biotechnology Limited Company
2021 Phase 2/Phase 3 NCT04912843 China
Vitamin B1
Beijing Tongren Hospital, CMU
2018 - ChiCTR-IPR-17013821 China
Hôpital Necker-Enfants Malades
2019 Phase 2/Phase 3 NCT04561466 France
Cyclosporine
University Hospital, Angers
2011 Phase 2 NCT02176733 France
Glycerol tributyrate
George Washington University
2025 Phase 1/Phase 2 NCT06792500 United States
GS010
GENSIGHT BIOLOGICS
2019 Phase 3 EUCTR2017-002187-40-NL Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-IT Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-GB Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-FR Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-ES Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-BE Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 4 EUCTR2017-002153-11-IT France;Germany;Italy;United Kingdom;United States
2018 Phase 3 EUCTR2017-002153-11-GB France;Germany;Italy;United Kingdom;United States
2016 Phase 3 EUCTR2015-001266-26-IT France;Germany;Italy;United Kingdom;United States
2016 Phase 3 EUCTR2015-001265-11-IT France;Germany;Italy;United Kingdom;United States
GENSIGHT-BIOLOGICS
2016 Phase 3 EUCTR2015-001266-26-DE France;Germany;Italy;United Kingdom;United States
2016 Phase 3 EUCTR2015-001265-11-DE France;Germany;Italy;United Kingdom;United States
2015 Phase 3 EUCTR2015-001266-26-GB France;Germany;Italy;United Kingdom;United States
2015 Phase 3 EUCTR2015-001265-11-GB France;Germany;Italy;United Kingdom;United States
2013 Phase 1;Phase 2 EUCTR2013-001405-90-FR France
GenSight Biologics
2019 - NCT03672968 -
2014 Phase 1/Phase 2 NCT02064569 France
Idebenone
Beijing Tongren Hospital, CMU
2018 - ChiCTR-IPR-17013821 China
Hyogo College of Medicine
2013 Phase 1,2 JPRN-UMIN000017939 Japan
SANTHERA PHARMACEUTICALS
- Phase 4 EUCTR2015-004405-16-IT Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain;United Kingdom
Santhera Pharmaceuticals
2016 Phase 4 NCT02774005 Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain;United Kingdom;United States
2016 - NCT02771379 Austria;France;Germany;Greece;Italy;Netherlands
Santhera Pharmaceuticals (Switzerland) Limited
2017 Phase 4 EUCTR2015-004405-16-BG Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain;United Kingdom;United States
2016 Phase 4 EUCTR2015-004405-16-PT Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain;United Kingdom;United States
2016 Phase 4 EUCTR2015-004405-16-ES Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain
2016 Phase 4 EUCTR2015-004405-16-DE Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain;United Kingdom;United States
2016 Phase 4 EUCTR2015-004405-16-BE Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain;United Kingdom;United States
2016 Phase 4 EUCTR2015-004405-16-AT Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain;United Kingdom;United States
- Phase 4 EUCTR2015-004405-16-PL Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain;United Kingdom;United States
IEM-EP
Region Stockholm
2008 - NCT06376279 -
Injection needle
Neurophth Therapeutics Inc
2023 Phase 1/Phase 2 NCT05293626 United States
KH176
Khondrion BV
2015 Phase 1 NCT02544217 Belgium
Lenadogene nolparvovec
GENSIGHT BIOLOGICS
2019 Phase 3 EUCTR2017-002187-40-NL Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-IT Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-GB Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-FR Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-ES Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-BE Belgium;France;Italy;Netherlands;Spain;United Kingdom
Mecobalamin
Beijing Tongren Hospital, CMU
2018 - ChiCTR-IPR-17013821 China
Nadh
GENSIGHT BIOLOGICS
2019 Phase 3 EUCTR2017-002187-40-NL Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-GB Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-FR Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-ES Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-BE Belgium;France;Italy;Netherlands;Spain;United Kingdom
NFS-02 injection
Neurophth Therapeutics Inc
2023 Phase 1/Phase 2 NCT05820152 China;United States
NR082 injection
Neurophth Therapeutics Inc
2023 Phase 1/Phase 2 NCT05293626 United States
Wuhan Neurophth Biotechnology Limited Company
2021 Phase 2/Phase 3 NCT04912843 China
Pending
GENSIGHT BIOLOGICS
2016 Phase 3 EUCTR2015-001265-11-IT France;Germany;Italy;United Kingdom;United States
GENSIGHT-BIOLOGICS
2016 Phase 3 EUCTR2015-001266-26-DE France;Germany;Italy;United Kingdom;United States
2016 Phase 3 EUCTR2015-001265-11-DE France;Germany;Italy;United Kingdom;United States
2015 Phase 3 EUCTR2015-001266-26-GB France;Germany;Italy;United Kingdom;United States
2015 Phase 3 EUCTR2015-001265-11-GB France;Germany;Italy;United Kingdom;United States
RAAV2-ND1
Taihe Hospital, Hubei University of Medicine, Shiyan, Hubei Province, China
2021 Phase 0 ChiCTR2000041574 China
RAAV2-ND4
Bin Li
2018 - NCT03428178 China
2011 - NCT01267422 China
Huazhong University of Science and Technology
2017 Phase 2/Phase 3 NCT03153293 China
RAAV2/2-ND4 vector
GENSIGHT BIOLOGICS
2016 Phase 3 EUCTR2015-001266-26-IT France;Germany;Italy;United Kingdom;United States
2016 Phase 3 EUCTR2015-001265-11-IT France;Germany;Italy;United Kingdom;United States
GENSIGHT-BIOLOGICS
2016 Phase 3 EUCTR2015-001266-26-DE France;Germany;Italy;United Kingdom;United States
2016 Phase 3 EUCTR2015-001265-11-DE France;Germany;Italy;United Kingdom;United States
2015 Phase 3 EUCTR2015-001266-26-GB France;Germany;Italy;United Kingdom;United States
2015 Phase 3 EUCTR2015-001265-11-GB France;Germany;Italy;United Kingdom;United States
2013 Phase 1;Phase 2 EUCTR2013-001405-90-FR France
Raxone
SANTHERA PHARMACEUTICALS
- Phase 4 EUCTR2015-004405-16-IT Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain;United Kingdom
Santhera Pharmaceuticals (Switzerland) Limited
2017 Phase 4 EUCTR2015-004405-16-BG Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain;United Kingdom;United States
2016 Phase 4 EUCTR2015-004405-16-PT Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain;United Kingdom;United States
2016 Phase 4 EUCTR2015-004405-16-ES Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain
2016 Phase 4 EUCTR2015-004405-16-DE Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain;United Kingdom;United States
2016 Phase 4 EUCTR2015-004405-16-BE Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain;United Kingdom;United States
2016 Phase 4 EUCTR2015-004405-16-AT Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain;United Kingdom;United States
- Phase 4 EUCTR2015-004405-16-PL Austria;Belgium;Bulgaria;Germany;Italy;Poland;Portugal;Spain;United Kingdom;United States
Recombinant AAV vector serotype 2 containing THE human wild type mitochondrial ND4 gene
GENSIGHT BIOLOGICS
2019 Phase 3 EUCTR2017-002187-40-NL Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-GB Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-FR Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-ES Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 3 EUCTR2017-002187-40-BE Belgium;France;Italy;Netherlands;Spain;United Kingdom
2018 Phase 4 EUCTR2017-002153-11-IT France;Germany;Italy;United Kingdom;United States
2018 Phase 3 EUCTR2017-002153-11-GB France;Germany;Italy;United Kingdom;United States
2016 Phase 3 EUCTR2015-001266-26-IT France;Germany;Italy;United Kingdom;United States
2016 Phase 3 EUCTR2015-001265-11-IT France;Germany;Italy;United Kingdom;United States
GENSIGHT-BIOLOGICS
2016 Phase 3 EUCTR2015-001266-26-DE France;Germany;Italy;United Kingdom;United States
2016 Phase 3 EUCTR2015-001265-11-DE France;Germany;Italy;United Kingdom;United States
2015 Phase 3 EUCTR2015-001266-26-GB France;Germany;Italy;United Kingdom;United States
2015 Phase 3 EUCTR2015-001265-11-GB France;Germany;Italy;United Kingdom;United States
2013 Phase 1;Phase 2 EUCTR2013-001405-90-FR France
Recombinant AAV vector serotype 2 containing THE human wild typemitochondrial ND4 gene
GENSIGHT BIOLOGICS
2018 Phase 3 EUCTR2017-002187-40-IT Belgium;France;Italy;Netherlands;Spain;United Kingdom
Sham injection
Wuhan Neurophth Biotechnology Limited Company
2021 Phase 2/Phase 3 NCT04912843 China
Vitamin B1
Beijing Tongren Hospital, CMU
2018 - ChiCTR-IPR-17013821 China