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
Showing 1 to 10 of 22 diseases
| 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 | NCT04912843 (ClinicalTrials.gov) | June 18, 2021 | 13/5/2021 | Gene Therapy Clinical Trial for the Treatment Of Leber's HereDitary Optic Neuropathy Associated With ND4 Mutations Gene Therapy Clinical Trial for the Treatment Of Leber's HereDitary Optic Neuropathy Associated With ... | A 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 Mutation A Phase 1/2/3, Single-arm, Multi-center, Two-stage Clinical Trial to Evaluate the Safety and Efficac ... | Leber Hereditary Optic Neuropathy (LHON) | Drug: NR082 injection | Wuhan Neurophth Biotechnology Limited Company | NULL | Recruiting | 12 Years | 75 Years | All | 55 | Phase 1/Phase 2 | China |
| 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 Efficacy and Safety of Bilateral Intravitreal Injection of GS010: A Randomized, Double-Masked, Place ... | Leber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene;Therapeutic area: Diseases [C] - Eye Diseases [C11] Leber Hereditary Optic Neuropathydue to mutations in the mitochondrial NADH Dehydrogenase 4 gene;Th ... | 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) Product Name: Recombinant AAV vector serotype 2 containing the human wild type mitochondrial ND4 gen ... | 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 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (P ... | 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 | 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 Efficacy and Safety of Bilateral Intravitreal Injection of GS010: A Randomized, Double-Masked, Place ... | 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] Leber Hereditary Optic Neuropathydue to mutations in the mitochondrial NADH Dehydrogenase 4 gene Med ... | Product Name: Recombinant AAV vector serotype 2 containing the human wild typemitochondrial ND4 gene Product Code: [GS010] INN or Proposed INN: lenadogene nolparvovec Product Name: Recombinant AAV vector serotype 2 containing the human wild typemitochondrial ND4 gene ... | GENSIGHT BIOLOGICS | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 90 | Phase 3 | France;Spain;Belgium;Netherlands;United Kingdom;Italy | ||
| 5 | 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 Efficacy and Safety of Bilateral Intravitreal Injection of GS010: A Randomized, Double-Masked, Place ... | 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] Leber Hereditary Optic Neuropathydue to mutations in the mitochondrialNADH Dehydrogenase 4 gene MedD ... | 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) Product Name: Recombinant AAV vector serotype 2 containing the human wild type mitochondrial ND4 gen ... | GENSIGHT BIOLOGICS | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 90 | Phase 3 | France;Belgium;Spain;Netherlands;Italy;United Kingdom | ||
| 6 | 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 A Randomized, Double-Masked, Placebo-Controlled Trial in Subjects Affected with G11778A ND4 Leber He ... | 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] Leber Hereditary Optic Neuropathydue to mutations in the mitochondrial NADH Dehydrogenase 4 gene Med ... | 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) Product Name: Recombinant AAV vector serotype 2 containing the human wild type mitochondrial ND4 gen ... | GENSIGHT BIOLOGICS | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 90 | Phase 3 | France;Belgium;Spain;Netherlands;Italy;United Kingdom | ||
| 7 | 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 Efficacy and Safety of Bilateral Intravitreal Injection of GS010: A Randomized, Double-Masked, Place ... | 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] Leber Hereditary Optic Neuropathydue to mutations in the mitochondrial NADH Dehydrogenase 4 gene Me ... | 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) Product Name: Recombinant AAV vector serotype 2 containing the human wild type mitochondrial ND4 gen ... | 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 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (P ... | United Kingdom;Italy;Netherlands;Spain;Belgium;France | ||
| 8 | 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 Efficacy and Safety of Bilateral Intravitreal Injection of GS010: A Randomized, Double-Masked, Place ... | Leber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene;Therapeutic area: Diseases [C] - Eye Diseases [C11] Leber Hereditary Optic Neuropathydue to mutations in the mitochondrial NADH Dehydrogenase 4 gene;Th ... | 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) Product Name: Recombinant AAV vector serotype 2 containing the human wild type mitochondrial ND4 gen ... | 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 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (P ... | France;Spain;Belgium;Netherlands;Italy;United Kingdom | ||
| 9 | 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 Long-term Follow-up of ND4 LHON Subjects Treated With GS010 OcularGene Therapy in the RESCUE or REVE ... | 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] Leber Hereditary Optic Neuropathydue to mutations in the mitochondrial NADH Dehydrogenase 4 gene Med ... | Product Name: Recombinant AAV vector serotype 2 containing the human wild type mitochondrial ND4 gene Product Code: GS010 Product Name: Recombinant AAV vector serotype 2 containing the human wild type mitochondrial ND4 gen ... | GENSIGHT BIOLOGICS | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 76 | Phase 3 | France;United States;Germany;Italy;United Kingdom | ||
| 10 | 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 & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation ... | 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 Efficacy and Safety of Bilateral Intravitreal Injection of GS010: A Randomized, Double-Masked, Place ... | 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 |