Gs010    (DrugBank: -)

1 disease
IDDisease name (Link within this page)Number of trials
302Leber hereditary optic neuropathy15

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.
15 / 20 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
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]GENSIGHT BIOLOGICSNULLNA Female: yes
Male: yes
90Phase 3France;Belgium;Spain;Netherlands;Italy;United Kingdom
2EUCTR2017-002187-40-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
3EUCTR2017-002187-40-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
4EUCTR2017-002187-40-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]
GENSIGHT BIOLOGICSNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
90Phase 3France;Belgium;Spain;Netherlands;Italy;United Kingdom
5EUCTR2017-002187-40-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]GENSIGHT BIOLOGICSNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
90Phase 3France;Spain;Belgium;Netherlands;Italy;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2017-002153-11-GB
(EUCTR)
13/03/201814/02/2018Long-term Follow-up of ND4 LHON Subjects Treated With GS010Long-term Follow-up of ND4 LHON Subjects Treated With GS010 OcularGene Therapy in the RESCUE or REVERSE Phase III Clinical Trials 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
GENSIGHT BIOLOGICSNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
76Phase 3France;United States;Germany;Italy;United Kingdom
7NCT03293524
(ClinicalTrials.gov)
March 12, 201819/9/2017Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 YearEfficacy 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 YearLeber Hereditary Optic NeuropathyGenetic: GS010;Drug: PlaceboGenSight BiologicsNULLActive, not recruiting15 YearsN/AAll90Phase 3United States;Belgium;France;Italy;Spain;Taiwan;United Kingdom
8EUCTR2015-001265-11-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
9EUCTR2015-001266-26-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
10EUCTR2015-001266-26-DE
(EUCTR)
21/01/201611/08/2015A Phase III gene therapy clinical trial in LHON Subjects Affected for more than 7months or moreA 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 mounths 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]GENSIGHT-BIOLOGICSNULLNot Recruiting Female: yes
Male: yes
40Phase 3France;United States;Germany;Italy;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2015-001265-11-DE
(EUCTR)
21/01/201611/08/2015A Phase III gene therapy clinical trial in LHON Subjects Affected for 6 Months or Less A 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 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]GENSIGHT-BIOLOGICSNULLNot Recruiting Female: yes
Male: yes
40Phase 3France;United States;Germany;Italy;United Kingdom
12EUCTR2015-001266-26-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
13EUCTR2015-001265-11-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
14EUCTR2013-001405-90-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
15EUCTR2017-002153-11-IT
(EUCTR)
12/03/2018Long-term Follow-up of ND4 LHON Subjects Treated With GS010 Ocular Gene Therapy in the RESCUE or REVERSE Phase III Clinical TrialsLong-term Follow-up of ND4 LHON Subjects Treated With GS010 Ocular Gene Therapy in the RESCUE or REVERSE Phase III Clinical Trials - RESCUE and REVERSE Long-term Follow-up 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
GENSIGHT BIOLOGICSNULLNAFemale: yes
Male: yes
76Phase 4France;United States;Germany;United Kingdom;Italy