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 : 23 / 薬物数 : 15 - (DrugBank : 5) / 標的遺伝子数 : 5 - 標的パスウェイ数 : 33
薬物ごとの開発者（Primary Sponsor）、臨床試験情報（抜粋）です。
Béfizal
Hôpital Necker-Enfants Malades
2019 Phase 2/Phase 3 NCT04561466 France;
Cyclosporine
University Hospital, Angers
2011 Phase 2 NCT02176733 France;
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 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;
- Phase 4 EUCTR2017-002153-11-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
2018 Phase 3 NCT03293524 Belgium;France;Italy;Spain;Taiwan;United Kingdom;United States;
Idebenone
Hyogo College of Medicine
2013 Phase 1,2 JPRN-UMIN000017939 Japan;
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;
NR082 injection
Wuhan Neurophth Biotechnology Limited Company
2021 Phase 1/Phase 2 NCT04912843 China;
PENDING
GENSIGHT BIOLOGICS
2016 Phase 3 EUCTR2015-001265-11-IT France;Germany;Italy;United Kingdom;United States;
Pending
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-ND4
Bin Li
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;
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 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;
- Phase 4 EUCTR2017-002153-11-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;
Recombinant Adeno-Associated Viral vector, serotype 2 (rAAV2/2) containing the human wild-type mitochondrial NADH Dehydrogenase 4 gene (ND4)
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;
VA
Hyogo College of Medicine
2013 Phase 1,2 JPRN-UMIN000017939 Japan;
Hôpital Necker-Enfants Malades
2019 Phase 2/Phase 3 NCT04561466 France;
Cyclosporine
University Hospital, Angers
2011 Phase 2 NCT02176733 France;
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 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;
- Phase 4 EUCTR2017-002153-11-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
2018 Phase 3 NCT03293524 Belgium;France;Italy;Spain;Taiwan;United Kingdom;United States;
Idebenone
Hyogo College of Medicine
2013 Phase 1,2 JPRN-UMIN000017939 Japan;
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;
NR082 injection
Wuhan Neurophth Biotechnology Limited Company
2021 Phase 1/Phase 2 NCT04912843 China;
PENDING
GENSIGHT BIOLOGICS
2016 Phase 3 EUCTR2015-001265-11-IT France;Germany;Italy;United Kingdom;United States;
Pending
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-ND4
Bin Li
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;
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 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;
- Phase 4 EUCTR2017-002153-11-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;
Recombinant Adeno-Associated Viral vector, serotype 2 (rAAV2/2) containing the human wild-type mitochondrial NADH Dehydrogenase 4 gene (ND4)
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;
VA
Hyogo College of Medicine
2013 Phase 1,2 JPRN-UMIN000017939 Japan;