RAAV2/2-ND4 VECTOR
( DrugBank: - / KEGG DRUG: - )
1 disease
| 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 | 7 |
302. レーベル遺伝性視神経症
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
Showing 1 to 7 of 7 trials
| 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 | EUCTR2015-001265-11-IT (EUCTR) | 18/03/201620160318 | 04/11/202020201104 | 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 Sperimentazione clinica pilota, randomizzata, in doppio cieco, controllata con simulazione per valut ... | 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 Neuropathy due 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: PENDING Other descriptive name: RAAV2/2-ND4 VECTOR Product Name: Recombinant AAV vector serotype 2 containing the human wild type mitochondrial ND4 gen ... | GENSIGHT BIOLOGICS | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 3 | United States;France;Germany;United Kingdom;Italy | ||
| 2 | EUCTR2015-001266-26-IT (EUCTR) | 18/03/201620160318 | 10/05/201920190510 | 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 Sperimentazione clinica pilota, randomizzata, in doppio cieco, controllata con simulazione per valut ... | 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 Neuropathy due 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 Other descriptive name: RAAV2/2-ND4 VECTOR Product Name: Recombinant AAV vector serotype 2 containing the human wild type mitochondrial ND4 gen ... | GENSIGHT BIOLOGICS | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 3 | France;United States;Germany;United Kingdom;Italy | ||
| 3 | EUCTR2015-001265-11-DE (EUCTR) | 21/01/201620160121 | 11/08/201520150811 | 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 A Randomized, Double-Masked, Sham-Controlled, Pivotal Clinical Trial to Evaluate the Efficacy of a S ... | Leber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene;Therapeutic area: Diseases [C] - Eye Diseases [C11] Leber Hereditary Optic Neuropathy due 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: pending Other descriptive name: RAAV2/2-ND4 VECTOR Product Name: Recombinant AAV vector serotype 2 containing the human wild type mitochondrial ND4 gen ... | GENSIGHT-BIOLOGICS | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 3 | France;United States;Germany;Italy;United Kingdom | ||
| 4 | EUCTR2015-001266-26-DE (EUCTR) | 21/01/201620160121 | 11/08/201520150811 | 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 A Randomized, Double-Masked, Sham-Controlled, Pivotal Clinical Trial to Evaluate the Efficacy of a S ... | Leber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene;Therapeutic area: Diseases [C] - Eye Diseases [C11] Leber Hereditary Optic Neuropathy due 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: pending Other descriptive name: RAAV2/2-ND4 VECTOR Product Name: Recombinant AAV vector serotype 2 containing the human wild type mitochondrial ND4 gen ... | GENSIGHT-BIOLOGICS | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 3 | France;United States;Germany;Italy;United Kingdom | ||
| 5 | EUCTR2015-001265-11-GB (EUCTR) | 08/10/201520151008 | 20/05/201620160520 | 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 A Randomized, Double-Masked, Sham-Controlled, Pivotal ClinicalTrial to Evaluate the Efficacy of a Si ... | Leber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene;Therapeutic area: Diseases [C] - Eye Diseases [C11] Leber Hereditary Optic Neuropathy due 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: pending Other descriptive name: RAAV2/2-ND4 VECTOR Product Name: Recombinant AAV vector serotype 2 containing the human wild type mitochondrial ND4 gen ... | GENSIGHT-BIOLOGICS | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 3 | France;United States;Germany;Italy;United Kingdom | ||
| 6 | EUCTR2015-001266-26-GB (EUCTR) | 08/10/201520151008 | 15/06/201620160615 | 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 A Randomized, Double-Masked, Sham-Controlled, Pivotal Clinical Trial to Evaluate the Efficacy of a S ... | Leber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene;Therapeutic area: Diseases [C] - Eye Diseases [C11] Leber Hereditary Optic Neuropathy due 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: pending Other descriptive name: RAAV2/2-ND4 VECTOR Product Name: Recombinant AAV vector serotype 2 containing the human wild type mitochondrial ND4 gen ... | GENSIGHT-BIOLOGICS | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 3 | United States;France;Germany;Italy;United Kingdom | ||
| 7 | EUCTR2013-001405-90-FR (EUCTR) | 26/12/201320131226 | 18/02/201420140218 | 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 A phase I/IIa, non randomized, escalating dose, open-label study to evaluate safety and efficacy of ... | 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] Leber Hereditary Optic Neuropathy due 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 Other descriptive name: RAAV2/2-ND4 VECTOR Product Name: Recombinant AAV vector serotype 2 containing the human wild type mitochondrial ND4 gen ... | GENSIGHT-BIOLOGICS | NULL | Not Recruiting | Female: yes Male: yes | 22 | Phase 1;Phase 2 | France |