302. レーベル遺伝性視神経症 Leber hereditary optic neuropathy Clinical trials / Disease details


臨床試験数 : 22 薬物数 : 16 - (DrugBank : 4) / 標的遺伝子数 : 5 - 標的パスウェイ数 : 33

  
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1NCT04912843
(ClinicalTrials.gov)
June 18, 202113/5/2021Gene Therapy Clinical Trial for the Treatment Of Leber's HereDitary Optic Neuropathy Associated With ND4 MutationsA Phase 1/2/3, Single-arm, Multi-center, Two-stage Clinical Trial to Evaluate the Safety and Efficacy of Gene Therapy for Leber Hereditary Optic Neuropathy (LHON) Associated With ND4 MutationLeber Hereditary Optic Neuropathy (LHON)Drug: NR082 injectionWuhan Neurophth Biotechnology Limited CompanyNULLRecruiting12 Years75 YearsAll55Phase 1/Phase 2China
2EUCTR2017-002187-40-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]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 BIOLOGICSNULLNA Female: yes
Male: yes
90 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;Belgium;Spain;Netherlands;Italy;United Kingdom
3NCT04561466
(ClinicalTrials.gov)
March 26, 201914/3/2019Trial of Befizal® 200 mg for the Treatment of Leber Hereditary Optic NeuropathyStudy of Efficacy of Befizal® 200 mg for the Treatment of Leber Hereditary Optic NeuropathySafety Issues;Efficacy, SelfDrug: BéfizalHôpital Necker-Enfants MaladesEuropean Georges Pompidou Hospital;CLAIROPRecruiting18 YearsN/AAll14Phase 2/Phase 3France
4EUCTR2017-002187-40-IT
(EUCTR)
04/12/201829/01/2021Efficacy 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]
Product Name: Recombinant AAV vector serotype 2 containing the human wild typemitochondrial ND4 gene
Product Code: [GS010]
INN or Proposed INN: lenadogene nolparvovec
GENSIGHT BIOLOGICSNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
90Phase 3France;Spain;Belgium;Netherlands;United Kingdom;Italy
5EUCTR2017-002187-40-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
6EUCTR2017-002187-40-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
7EUCTR2017-002187-40-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]
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 finished Female: yes
Male: yes
90 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited Kingdom;Italy;Netherlands;Spain;Belgium;France
8EUCTR2017-002187-40-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]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 finished Female: yes
Male: yes
90 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;Spain;Belgium;Netherlands;Italy;United Kingdom
9EUCTR2017-002153-11-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
10NCT03293524
(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
11NCT03153293
(ClinicalTrials.gov)
December 27, 201710/5/2017A Single Intravitreal Injection of rAAV2-ND4 for the Treatment of Leber's Hereditary Optic NeuropathyA Single Intravitreal Injection of rAAV2-ND4 for the Treatment of Leber's Hereditary Optic NeuropathyLeber Hereditary Optic NeuropathyDrug: rAAV2-ND4Huazhong University of Science and TechnologyShiyan Taihe HospitalActive, not recruiting10 Years65 YearsAll159Phase 2/Phase 3China
12EUCTR2015-001266-26-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
13EUCTR2015-001265-11-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
14EUCTR2015-001265-11-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]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 Recruiting Female: yes
Male: yes
40 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;United States;Germany;Italy;United Kingdom
15EUCTR2015-001266-26-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]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 Recruiting Female: yes
Male: yes
40 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;United States;Germany;Italy;United Kingdom
16EUCTR2015-001265-11-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
17EUCTR2015-001266-26-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
18EUCTR2013-001405-90-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
19JPRN-UMIN000017939
2013/10/0120/06/2015Multicenter study of therapeutic effects of Idebenone in patients with Leber hereditary optic neuropathyMulticenter study of therapeutic effects of Idebenone in patients with Leber hereditary optic neuropathy - Clinical trial of Idebenone in patients with LHON Leber hereditary optic neuropathy1. 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 MedicineKitasato UniversityJikei University School of MedicineTokyo Medical UniversityComplete: follow-up complete10years-old80years-oldMale and Female50Phase 1,2Japan
20NCT02176733
(ClinicalTrials.gov)
July 201125/6/2014Trial of Cyclosporine in the Acute Phase of Leber Hereditary Optic NeuropathyLeber Hereditary Optic NeuropathyDrug: cyclosporineUniversity Hospital, AngersNULLRecruiting18 YearsN/ABoth12Phase 2France
21NCT01267422
(ClinicalTrials.gov)
April 201127/12/2010Safety and Efficacy Study of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy (LHON)Safety and Efficacy Study of a Single Intravitreal Injection of rAAV2-ND4 Treatment of Leber Hereditary Optic NeuropathyLeber Hereditary Optic NeuropathyDrug: rAAV2-ND4Bin LiHuazhong University of Science and TechnologyCompleted8 Years60 YearsAll9N/AChina
22EUCTR2017-002153-11-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