Lenadogene nolparvovec ( DrugBank: Lenadogene nolparvovec )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
302レーベル遺伝性視神経症6

302. レーベル遺伝性視神経症


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
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
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size
PhaseCountries
1EUCTR2017-002187-40-NL
(EUCTR)
19/06/201926/09/2018Efficacy and Safety of Bilateral Intravitreal Injection of GS010Efficacy and Safety of Bilateral Intravitreal Injection of GS010: A Randomized, Double-Masked, Placebo-Controlled Trial in Subjects Affected with G11778A ND4 Leber Hereditary Optic Neuropathy for Up to One Year - REFLECT Leber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene;Therapeutic area: Diseases [C] - Eye Diseases [C11]Product Name: Recombinant AAV vector serotype 2 containing the human wild type mitochondrial ND4 gene
Product Code: GS010
INN or Proposed INN: lenadogene nolparvovec
Other descriptive name: Recombinant Adeno-Associated Viral vector, serotype 2 (rAAV2/2) containing the human wild-type mitochondrial NADH Dehydrogenase 4 gene (ND4)
GENSIGHT BIOLOGICSNULLNA Female: yes
Male: yes
90 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;Belgium;Spain;Netherlands;Italy;United Kingdom
2EUCTR2017-002187-40-IT
(EUCTR)
04/12/201829/01/2021Efficacy and Safety of Bilateral Intravitreal Injection of GS010Efficacy and Safety of Bilateral Intravitreal Injection of GS010: A Randomized, Double-Masked, Placebo-Controlled Trial in Subjects Affected with G11778A ND4 Leber Hereditary Optic Neuropathy for Up to One Year - REFLECT Leber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene
MedDRA version: 20.1;Level: LLT;Classification code 10062951;Term: Leber's hereditary optic atrophy neuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Eye Diseases [C11]
Product Name: Recombinant AAV vector serotype 2 containing the human wild typemitochondrial ND4 gene
Product Code: [GS010]
INN or Proposed INN: lenadogene nolparvovec
GENSIGHT BIOLOGICSNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
90Phase 3France;Spain;Belgium;Netherlands;United Kingdom;Italy
3EUCTR2017-002187-40-FR
(EUCTR)
15/06/201816/01/2018Efficacy and Safety of Bilateral Intravitreal Injection of GS010Efficacy and Safety of Bilateral Intravitreal Injection of GS010: A Randomized, Double-Masked, Placebo-Controlled Trial in Subjects Affected with G11778A ND4 Leber Hereditary Optic Neuropathy for Up to One Year - REFLECT Leber Hereditary Optic Neuropathy due to mutations in the mitochondrialNADH Dehydrogenase 4 gene
MedDRA version: 20.1;Level: LLT;Classification code 10062951;Term: Leber's hereditary optic atrophy neuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Eye Diseases [C11]
Product Name: Recombinant AAV vector serotype 2 containing the human wild type mitochondrial ND4 gene
Product Code: GS010
INN or Proposed INN: lenadogene nolparvovec
Other descriptive name: Recombinant Adeno-Associated Viral vector, serotype 2 (rAAV2/2) containing the human wild-type mitochondrial NADH Dehydrogenase 4 gene (ND4)
GENSIGHT BIOLOGICSNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
90Phase 3France;Belgium;Spain;Netherlands;Italy;United Kingdom
4EUCTR2017-002187-40-ES
(EUCTR)
23/05/201802/03/2018Efficacy and Safety of Bilateral Intravitreal Injection of GS010A Randomized, Double-Masked, Placebo-Controlled Trial in Subjects Affected with G11778A ND4 Leber Hereditary Optic Neuropathy for Up to One Year - REFLECT Leber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene
MedDRA version: 20.1;Level: LLT;Classification code 10062951;Term: Leber's hereditary optic atrophy neuropathy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Eye Diseases [C11]
Product Name: Recombinant AAV vector serotype 2 containing the human wild type mitochondrial ND4 gene
Product Code: GS010
INN or Proposed INN: lenadogene nolparvovec
Other descriptive name: Recombinant Adeno-Associated Viral vector, serotype 2 (rAAV2/2) containing the human wild-type mitochondrial NADH Dehydrogenase 4 gene (ND4)
GENSIGHT BIOLOGICSNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
90Phase 3France;Belgium;Spain;Netherlands;Italy;United Kingdom
5EUCTR2017-002187-40-GB
(EUCTR)
30/03/201818/01/2018Efficacy and Safety of Bilateral Intravitreal Injection of GS010Efficacy and Safety of Bilateral Intravitreal Injection of GS010: A Randomized, Double-Masked, Placebo-Controlled Trial in Subjects Affected with G11778A ND4 Leber Hereditary Optic Neuropathy for Up to One Year - REFLECT Leber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene
MedDRA version: 20.1;Level: LLT;Classification code 10062951;Term: Leber's hereditary optic atrophy neuropathy;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Eye Diseases [C11]
Product Name: Recombinant AAV vector serotype 2 containing the human wild type mitochondrial ND4 gene
Product Code: GS010
INN or Proposed INN: lenadogene nolparvovec
Other descriptive name: Recombinant Adeno-Associated Viral vector, serotype 2 (rAAV2/2) containing the human wild-type mitochondrial NADH Dehydrogenase 4 gene (ND4)
GENSIGHT BIOLOGICSNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
90 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;Belgium;Spain;Netherlands;Italy;United Kingdom
6EUCTR2017-002187-40-BE
(EUCTR)
15/03/201816/01/2018Efficacy and Safety of Bilateral Intravitreal Injection of GS010Efficacy and Safety of Bilateral Intravitreal Injection of GS010: A Randomized, Double-Masked, Placebo-Controlled Trial in Subjects Affected with G11778A ND4 Leber Hereditary Optic Neuropathy for Up to One Year - REFLECT Leber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene;Therapeutic area: Diseases [C] - Eye Diseases [C11]Product Name: Recombinant AAV vector serotype 2 containing the human wild type mitochondrial ND4 gene
Product Code: GS010
INN or Proposed INN: lenadogene nolparvovec
Other descriptive name: Recombinant Adeno-Associated Viral vector, serotype 2 (rAAV2/2) containing the human wild-type mitochondrial NADH Dehydrogenase 4 gene (ND4)
GENSIGHT BIOLOGICSNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
90 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;Spain;Belgium;Netherlands;Italy;United Kingdom