DDrare: Database of Drug Development for Rare Diseases

302. レーベル遺伝性視神経症
［臨床試験数：20，薬物数：14（DrugBank：4），標的遺伝子数：5，標的パスウェイ数：32］

Searched query = "Leber hereditary optic neuropathy"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Drug = Béfizal; Cyclosporine; GS010; Genetic: GS010; Idebenone; Lenadogene nolparvovec; NADH; PENDING; Pending; RAAV2-ND4; RAAV2/2-ND4 VECTOR; Recombinant AAV vector serotype 2 containing the human wild type mitochondrial ND4 gene; Recombinant Adeno-Associated Viral vector, serotype 2 (rAAV2/2) containing the human wild-type mitochondrial NADH Dehydrogenase 4 gene (ND4); VA

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT03293524 (ClinicalTrials.gov)	March 12, 2018	19/9/2017	Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year	Efficacy 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	Leber Hereditary Optic Neuropathy	Genetic: GS010;Drug: Placebo	GenSight Biologics	NULL	Active, not recruiting	15 Years	N/A	All	90	Phase 3	United States;Belgium;France;Italy;Spain;Taiwan;United Kingdom

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NCT03293524
(ClinicalTrials.gov)

March 12, 2018

19/9/2017

Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year

Efficacy 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

Leber Hereditary Optic Neuropathy

Genetic: GS010;Drug: Placebo

GenSight Biologics

NULL

Active, not recruiting

15 Years

N/A

All

Phase 3

United States;Belgium;France;Italy;Spain;Taiwan;United Kingdom