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 : 23 / 薬物数 : 15 - (DrugBank : 5) / 標的遺伝子数 : 5 - 標的パスウェイ数 : 33
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 |
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1 | NCT05293626 (ClinicalTrials.gov) | January 2023 | 9/12/2021 | Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND4 Mutations | A Phase 1/2, Single-Arm, Open-Label, Dose-Finding Clinical Trial to Evaluate the Safety and Efficacy of Gene Therapy for Leber's Hereditary Optic Neuropathy (LHON) Associated With ND4 Mutation | Leber Hereditary Optic Neuropathy (LHON) | Drug: NR082 Injection;Device: Injection needle | Neurophth Therapeutics Inc | NULL | Not yet recruiting | 18 Years | 75 Years | All | 18 | Phase 1/Phase 2 | NULL |
2 | 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] | 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 | NA | Female: yes Male: yes | 90 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;Belgium;Spain;Netherlands;Italy;United Kingdom | ||
3 | NCT04561466 (ClinicalTrials.gov) | March 26, 2019 | 14/3/2019 | Trial of Befizal® 200 mg for the Treatment of Leber Hereditary Optic Neuropathy | Study of Efficacy of Befizal® 200 mg for the Treatment of Leber Hereditary Optic Neuropathy | Safety Issues;Efficacy, Self | Drug: Béfizal | Hôpital Necker-Enfants Malades | European Georges Pompidou Hospital;CLAIROP | Recruiting | 18 Years | N/A | All | 14 | Phase 2/Phase 3 | France |
4 | NCT03672968 (ClinicalTrials.gov) | February 22, 2019 | 13/9/2018 | EAP_GS010_single Patient | EAP Single Patient: Safety of Bilateral Intravitreal Injection of GS010 in a Single Subject Affected With G11778A ND4 Leber Hereditary Optic Neuropathy | Leber Hereditary Optic Neuropathy (Optic, Atrophy, Hereditary, Leber) | Genetic: GS010 | GenSight Biologics | NULL | Available | N/A | N/A | All | NULL | ||
5 | EUCTR2017-002187-40-IT (EUCTR) | 04/12/2018 | 29/01/2021 | 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] | Product Name: Recombinant AAV vector serotype 2 containing the human wild typemitochondrial ND4 gene Product Code: [GS010] INN or Proposed INN: lenadogene nolparvovec | GENSIGHT BIOLOGICS | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 90 | Phase 3 | France;Spain;Belgium;Netherlands;United Kingdom;Italy | ||
6 | EUCTR2017-002153-11-IT (EUCTR) | 12/07/2018 | 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 | Not Recruiting | Female: yes Male: yes | 76 | Phase 4 | United States;France;Germany;United Kingdom;Italy | ||
7 | 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 | ||
8 | 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 | ||
9 | 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] | 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 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;Belgium;Spain;Netherlands;Italy;United Kingdom | ||
10 | 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] | 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 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;Spain;Belgium;Netherlands;Italy;United Kingdom | ||
11 | 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 | ||
12 | NCT03153293 (ClinicalTrials.gov) | December 27, 2017 | 10/5/2017 | A Single Intravitreal Injection of rAAV2-ND4 for the Treatment of Leber's Hereditary Optic Neuropathy | A Single Intravitreal Injection of rAAV2-ND4 for the Treatment of Leber's Hereditary Optic Neuropathy | Leber Hereditary Optic Neuropathy | Drug: rAAV2-ND4 | Huazhong University of Science and Technology | Shiyan Taihe Hospital | Active, not recruiting | 10 Years | 65 Years | All | 159 | Phase 2/Phase 3 | China |
13 | 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 | ||
14 | 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 | ||
15 | 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] | 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 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;United States;Germany;Italy;United Kingdom | ||
16 | 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] | 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 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;United States;Germany;Italy;United Kingdom | ||
17 | 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 | ||
18 | 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 | ||
19 | NCT02064569 (ClinicalTrials.gov) | February 13, 2014 | 13/2/2014 | Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients | An Open Label Dose Escalation Clinical Trial to Evaluate the Safety and the Tolerability of GS010 (rAAV2/2-ND4) in Patients With Leber Hereditary Optic Neuropathy Due to Mutations in the Mitochondrial NADH Dehydrogenase 4 Gene | Leber Hereditary Optic Neuropathy | Genetic: GS010 | GenSight Biologics | NULL | Completed | 18 Years | N/A | All | 19 | Phase 1/Phase 2 | France |
20 | 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 | ||
21 | 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 1,2 | Japan |
22 | NCT02176733 (ClinicalTrials.gov) | July 2011 | 25/6/2014 | Trial of Cyclosporine in the Acute Phase of Leber Hereditary Optic Neuropathy | Leber Hereditary Optic Neuropathy | Drug: cyclosporine | University Hospital, Angers | NULL | Recruiting | 18 Years | N/A | Both | 12 | Phase 2 | France | |
23 | NCT01267422 (ClinicalTrials.gov) | April 2011 | 27/12/2010 | Safety 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 Neuropathy | Leber Hereditary Optic Neuropathy | Drug: rAAV2-ND4 | Bin Li | Huazhong University of Science and Technology | Completed | 8 Years | 60 Years | All | 9 | N/A | China |