302. Leber hereditary optic neuropathy
20 clinical trials,   14 drugs   (DrugBank: 4 drugs),   5 drug target genes,   32 drug target pathways

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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
5 trials found
No.TrialIDDate_
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PhaseCountries
1EUCTR2015-001265-11-IT
(EUCTR)
18/03/201604/11/2020A Phase III gene therapy clinical trial in LHON Subjects Affected for 6 Months or LessSperimentazione clinica pilota, randomizzata, in doppio cieco, controllata con simulazione per valutare l'efficacia di un'unica iniezione intravitreale di GS010 (rAAV2/2-ND4) in soggetti affetti, per un periodo di 6 mesi o inferiore, da neuropatia ottica ereditaria di Leber a causa della mutazione G11778A nel gene mitocondriale NADH deidrogenasi 4 - RESCUE 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: PENDING
Other descriptive name: RAAV2/2-ND4 VECTOR
GENSIGHT BIOLOGICSNULLNot RecruitingFemale: yes
Male: yes
40Phase 3United States;France;Germany;United Kingdom;Italy
2EUCTR2015-001266-26-IT
(EUCTR)
18/03/201610/05/2019A Phase III gene therapy clinical trial in LHON Subjects Affected for more 7 monthsSperimentazione clinica pilota, randomizzata, in doppio cieco, controllata con simulazione per valutare l'efficacia di un'unica iniezione intravitreale di GS010 (rAAV2/2-ND4) in soggetti affetti, per un periodo maggiore di 6 mesi e fino a 12 mesi da neuropatia ottica ereditaria di Leber a causa della mutazione G11778A nel gene mitocondriale NADH deidrogenasi 4 - REVERSE 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
Other descriptive name: RAAV2/2-ND4 VECTOR
GENSIGHT BIOLOGICSNULLNot RecruitingFemale: yes
Male: yes
40Phase 3France;United States;Germany;United Kingdom;Italy
3EUCTR2015-001266-26-GB
(EUCTR)
08/10/201515/06/2016A Phase III gene therapy clinical trial in LHON Subjects Affectedfor more 7 monthsA Randomized, Double-Masked, Sham-Controlled, Pivotal Clinical Trial to Evaluate the Efficacy of a Single Intravitreal Injection of GS010 (rAAV2/2-ND4) in Subjects Affected for more than 6 Months and to 12 months by Leber Hereditary Optic Neuropathy Due to the G11778A Mutation in the Mitochondrial NADH Dehydrogenase 4 Gene 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: pending
Other descriptive name: RAAV2/2-ND4 VECTOR
GENSIGHT-BIOLOGICSNULLNot RecruitingFemale: yes
Male: yes
40Phase 3United States;France;Germany;Italy;United Kingdom
4EUCTR2015-001265-11-GB
(EUCTR)
08/10/201520/05/2016A Phase III gene therapy clinical trial in LHON Subjects Affectedfor 6 Months or LessA Randomized, Double-Masked, Sham-Controlled, Pivotal ClinicalTrial to Evaluate the Efficacy of a Single Intravitreal Injection of GS010 (rAAV2/2-ND4) in Subjects Affected for 6 Months or Less by Leber Hereditary Optic Neuropathy Due to the G11778A Mutation in the Mitochondrial NADH Dehydrogenase 4 Gene 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: pending
Other descriptive name: RAAV2/2-ND4 VECTOR
GENSIGHT-BIOLOGICSNULLNot RecruitingFemale: yes
Male: yes
40Phase 3France;United States;Germany;Italy;United Kingdom
5EUCTR2013-001405-90-FR
(EUCTR)
26/12/201318/02/2014A Phase I/IIa gene therapy clinical trial in LHON patientsA phase I/IIa, non randomized, escalating dose, open-label study to evaluate safety and efficacy of GS010 (rAAV2/2-ND4) in patients suffering from Leber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene Leber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene
MedDRA version: 16.1;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
Other descriptive name: RAAV2/2-ND4 VECTOR
GENSIGHT-BIOLOGICSNULLNot RecruitingFemale: yes
Male: yes
22Phase 1;Phase 2France