DDrare: Database of Drug Development for Rare Diseases

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	15

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	EUCTR2017-002187-40-NL (EUCTR)	19/06/2019	26/09/2018	Efficacy and Safety of Bilateral Intravitreal Injection of GS010	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 - REFLECT	Leber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene;Therapeutic area: Diseases [C] - Eye Diseases [C11]		GENSIGHT BIOLOGICS	NULL	NA			Female: yes Male: yes	90	Phase 3	France;Belgium;Spain;Netherlands;Italy;United Kingdom
2	EUCTR2017-002187-40-FR (EUCTR)	15/06/2018	16/01/2018	Efficacy and Safety of Bilateral Intravitreal Injection of GS010	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 - 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 BIOLOGICS	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	90	Phase 3	France;Belgium;Spain;Netherlands;Italy;United Kingdom
3	EUCTR2017-002187-40-ES (EUCTR)	23/05/2018	02/03/2018	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 - 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 BIOLOGICS	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	90	Phase 3	France;Belgium;Spain;Netherlands;Italy;United Kingdom
4	EUCTR2017-002187-40-GB (EUCTR)	30/03/2018	18/01/2018	Efficacy and Safety of Bilateral Intravitreal Injection of GS010	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 - 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 BIOLOGICS	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	90	Phase 3	France;Belgium;Spain;Netherlands;Italy;United Kingdom
5	EUCTR2017-002187-40-BE (EUCTR)	15/03/2018	16/01/2018	Efficacy and Safety of Bilateral Intravitreal Injection of GS010	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 - REFLECT	Leber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene;Therapeutic area: Diseases [C] - Eye Diseases [C11]		GENSIGHT BIOLOGICS	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	90	Phase 3	France;Spain;Belgium;Netherlands;Italy;United Kingdom
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
6	EUCTR2017-002153-11-GB (EUCTR)	13/03/2018	14/02/2018	Long-term Follow-up of ND4 LHON Subjects Treated With GS010	Long-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 BIOLOGICS	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	76	Phase 3	France;United States;Germany;Italy;United Kingdom
7	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
8	EUCTR2015-001265-11-IT (EUCTR)	18/03/2016	04/11/2020	A Phase III gene therapy clinical trial in LHON Subjects Affected for 6 Months or Less	Sperimentazione 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 BIOLOGICS	NULL	Not Recruiting			Female: yes Male: yes	40	Phase 3	United States;France;Germany;United Kingdom;Italy
9	EUCTR2015-001266-26-IT (EUCTR)	18/03/2016	10/05/2019	A Phase III gene therapy clinical trial in LHON Subjects Affected for more 7 months	Sperimentazione 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 BIOLOGICS	NULL	Not Recruiting			Female: yes Male: yes	40	Phase 3	France;United States;Germany;United Kingdom;Italy
10	EUCTR2015-001266-26-DE (EUCTR)	21/01/2016	11/08/2015	A Phase III gene therapy clinical trial in LHON Subjects Affected for more than 7months or more	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 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-BIOLOGICS	NULL	Not Recruiting			Female: yes Male: yes	40	Phase 3	France;United States;Germany;Italy;United Kingdom
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
11	EUCTR2015-001265-11-DE (EUCTR)	21/01/2016	11/08/2015	A 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-BIOLOGICS	NULL	Not Recruiting			Female: yes Male: yes	40	Phase 3	France;United States;Germany;Italy;United Kingdom
12	EUCTR2015-001265-11-GB (EUCTR)	08/10/2015	20/05/2016	A Phase III gene therapy clinical trial in LHON Subjects Affectedfor 6 Months or Less	A 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-BIOLOGICS	NULL	Not Recruiting			Female: yes Male: yes	40	Phase 3	France;United States;Germany;Italy;United Kingdom
13	EUCTR2015-001266-26-GB (EUCTR)	08/10/2015	15/06/2016	A Phase III gene therapy clinical trial in LHON Subjects Affectedfor more 7 months	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 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-BIOLOGICS	NULL	Not Recruiting			Female: yes Male: yes	40	Phase 3	United States;France;Germany;Italy;United Kingdom
14	EUCTR2013-001405-90-FR (EUCTR)	26/12/2013	18/02/2014	A Phase I/IIa gene therapy clinical trial in LHON patients	A 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-BIOLOGICS	NULL	Not Recruiting			Female: yes Male: yes	22	Phase 1;Phase 2	France
15	EUCTR2017-002153-11-IT (EUCTR)		12/03/2018	Long-term Follow-up of ND4 LHON Subjects Treated With GS010 Ocular Gene Therapy in the RESCUE or REVERSE Phase III Clinical Trials	Long-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 BIOLOGICS	NULL	NA			Female: yes Male: yes	76	Phase 4	France;United States;Germany;United Kingdom;Italy

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

EUCTR2017-002187-40-NL
(EUCTR)

19/06/2019

26/09/2018

Efficacy and Safety of Bilateral Intravitreal Injection of GS010

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 - REFLECT

Leber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene;Therapeutic area: Diseases [C] - Eye Diseases [C11]

GENSIGHT BIOLOGICS

NULL

Female: yes
Male: yes

Phase 3

France;Belgium;Spain;Netherlands;Italy;United Kingdom

EUCTR2017-002187-40-FR
(EUCTR)

15/06/2018

16/01/2018

Efficacy and Safety of Bilateral Intravitreal Injection of GS010

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 BIOLOGICS

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 3

France;Belgium;Spain;Netherlands;Italy;United Kingdom

EUCTR2017-002187-40-ES
(EUCTR)

23/05/2018

02/03/2018

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 - 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 BIOLOGICS

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 3

France;Belgium;Spain;Netherlands;Italy;United Kingdom

EUCTR2017-002187-40-GB
(EUCTR)

30/03/2018

18/01/2018

Efficacy and Safety of Bilateral Intravitreal Injection of GS010

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 BIOLOGICS

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 3

France;Belgium;Spain;Netherlands;Italy;United Kingdom

EUCTR2017-002187-40-BE
(EUCTR)

15/03/2018

16/01/2018

Efficacy and Safety of Bilateral Intravitreal Injection of GS010

Leber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene;Therapeutic area: Diseases [C] - Eye Diseases [C11]

GENSIGHT BIOLOGICS

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 3

France;Spain;Belgium;Netherlands;Italy;United Kingdom

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

EUCTR2017-002153-11-GB
(EUCTR)

13/03/2018

14/02/2018

Long-term Follow-up of ND4 LHON Subjects Treated With GS010

Long-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 BIOLOGICS

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 3

France;United States;Germany;Italy;United Kingdom

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

EUCTR2015-001265-11-IT
(EUCTR)

18/03/2016

04/11/2020

A Phase III gene therapy clinical trial in LHON Subjects Affected for 6 Months or Less

Sperimentazione 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 BIOLOGICS

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

United States;France;Germany;United Kingdom;Italy

EUCTR2015-001266-26-IT
(EUCTR)

18/03/2016

10/05/2019

A Phase III gene therapy clinical trial in LHON Subjects Affected for more 7 months

Sperimentazione 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 BIOLOGICS

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

France;United States;Germany;United Kingdom;Italy

EUCTR2015-001266-26-DE
(EUCTR)

21/01/2016

11/08/2015

A Phase III gene therapy clinical trial in LHON Subjects Affected for more than 7months or more

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 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-BIOLOGICS

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

France;United States;Germany;Italy;United Kingdom

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

EUCTR2015-001265-11-DE
(EUCTR)

21/01/2016

11/08/2015

A 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-BIOLOGICS

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

France;United States;Germany;Italy;United Kingdom

EUCTR2015-001265-11-GB
(EUCTR)

08/10/2015

20/05/2016

A Phase III gene therapy clinical trial in LHON Subjects Affectedfor 6 Months or Less

A 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-BIOLOGICS

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

France;United States;Germany;Italy;United Kingdom

EUCTR2015-001266-26-GB
(EUCTR)

08/10/2015

15/06/2016

A Phase III gene therapy clinical trial in LHON Subjects Affectedfor more 7 months

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 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-BIOLOGICS

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

United States;France;Germany;Italy;United Kingdom

EUCTR2013-001405-90-FR
(EUCTR)

26/12/2013

18/02/2014

A Phase I/IIa gene therapy clinical trial in LHON patients

A 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-BIOLOGICS

NULL

Not Recruiting

Female: yes
Male: yes

Phase 1;Phase 2

France

EUCTR2017-002153-11-IT
(EUCTR)

12/03/2018

Long-term Follow-up of ND4 LHON Subjects Treated With GS010 Ocular Gene Therapy in the RESCUE or REVERSE Phase III Clinical Trials

Long-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 BIOLOGICS

NULL

Female: yes
Male: yes

Phase 4

France;United States;Germany;United Kingdom;Italy