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	2

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-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
2	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

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-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

Phase 3

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

EUCTR2017-002187-40-ES
(EUCTR)

23/05/2018

02/03/2018