DDrare: Database of Drug Development for Rare Diseases

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	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
2	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
3	JPRN-UMIN000017939	2013/10/01	20/06/2015	Multicenter study of therapeutic effects of Idebenone in patients with Leber hereditary optic neuropathy	Multicenter study of therapeutic effects of Idebenone in patients with Leber hereditary optic neuropathy - Clinical trial of Idebenone in patients with LHON	Leber hereditary optic neuropathy	1. Clinical trial medicine: Idebenone 900mg/day 2. Objectives: 50 patients with LHON 3. Exclusion criteria: a) A smoker b) A patient with abnormality of hepatic function c) A patient who present seizures, delirium and hallucination d) Pregnancy or Lactation e) A patient who is associated with agranulocytosis f) A patient with chronic renal failure g) A patient with anaphylactic shock against Idebenone 4. Duration of drug administration: 6 months 5. Examinations schedules: Both subjective and objective examinations are performed as following schedules; a) At the base line: Visual acuity (VA), Critical flicker frequency (CFF), Visual field (VF: Humphry 30-2), central retinal thickness (CRT), functional MRI (f-MRI), searching the mitochondrial mutation b) 8 weeks: VA, CFF, VF, CRT, f-MRI c) 16 weeks: VA, CFF, VF, CRT, f-MRI d) 24 weeks: VA, CFF, VF, CRT, f-MRI e) 32 weeks: VA, CFF, VF, CRT, f-MRI f) 40 weeks: VA, CFF, VF, CRT, f-MRI g) 48 weeks: VA, CFF, VF, CRT, f-MRI	Hyogo College of Medicine	Kitasato UniversityJikei University School of MedicineTokyo Medical University	Complete: follow-up complete	10years-old	80years-old	Male and Female	50	Phase 1;Phase 2	Japan

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

EUCTR2015-001266-26-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

EUCTR2015-001265-11-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

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

JPRN-UMIN000017939

2013/10/01

20/06/2015

Multicenter study of therapeutic effects of Idebenone in patients with Leber hereditary optic neuropathy

Multicenter study of therapeutic effects of Idebenone in patients with Leber hereditary optic neuropathy - Clinical trial of Idebenone in patients with LHON

Leber hereditary optic neuropathy

1. Clinical trial medicine: Idebenone 900mg/day
2. Objectives: 50 patients with LHON
3. Exclusion criteria:
a) A smoker
b) A patient with abnormality of hepatic function
c) A patient who present seizures, delirium and hallucination
d) Pregnancy or Lactation
e) A patient who is associated with agranulocytosis
f) A patient with chronic renal failure
g) A patient with anaphylactic shock against Idebenone
4. Duration of drug administration: 6 months
5. Examinations schedules: Both subjective and objective examinations are performed as following schedules;
a) At the base line: Visual acuity (VA), Critical flicker frequency (CFF), Visual field (VF: Humphry 30-2), central retinal thickness (CRT), functional MRI (f-MRI), searching the mitochondrial mutation
b) 8 weeks: VA, CFF, VF, CRT, f-MRI
c) 16 weeks: VA, CFF, VF, CRT, f-MRI
d) 24 weeks: VA, CFF, VF, CRT, f-MRI
e) 32 weeks: VA, CFF, VF, CRT, f-MRI
f) 40 weeks: VA, CFF, VF, CRT, f-MRI
g) 48 weeks: VA, CFF, VF, CRT, f-MRI

Hyogo College of Medicine

Kitasato UniversityJikei University School of MedicineTokyo Medical University

Complete: follow-up complete

10years-old

80years-old

Male and Female

Phase 1;Phase 2

Japan