19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details
臨床試験数 : 899 / 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182
| 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 | EUCTR2008-005223-28-DE (EUCTR) | 21/01/2010 | 11/08/2009 | A Study of Eliglustat Tartrate (Genz-112638) in Patients with Gaucher Disease Who Have Reached Thereapeutic Goals with Enzyme Replacement Therapy | A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have reached therapeutic goals with enzyme replacement therapy - ENCORE | Gaucher Disease type I MedDRA version: 18.0;Level: PT;Classification code 10018048;Term: Gaucher's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: Eliglustat Other descriptive name: not available Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: Eliglustat Other descriptive name: not available Trade Name: Cerezyme Product Name: Cerezyme Product Code: imiglucerase INN or Proposed INN: IMIGLUCERASE Other descriptive name: Recombinant human derived macrophage-targeted ß-Glucocerebrosidase Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: Eliglustat Other descriptive name: not available | Genzyme Europe B.V. | NULL | Not Recruiting | Female: yes Male: yes | 150 | Phase 3 | United States;Spain;Turkey;Russian Federation;United Kingdom;Italy;Egypt;France;Czech Republic;Canada;Argentina;Brazil;Australia;Netherlands;Germany | ||
| 2 | EUCTR2008-005223-28-GB (EUCTR) | 16/11/2009 | 06/07/2009 | A Study of Eliglustat Tartrate (Genz-112638) in Patients with Gaucher Disease Who Have Reached Thereapeutic Goals with Enzyme Replacement Therapy | A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have Reached Therapeutic Goals with Enzyme Replacement Therapy - ENCORE | Gaucher Disease type I MedDRA version: 14.1;Level: PT;Classification code 10018048;Term: Gaucher's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: Eliglustat Other descriptive name: not available Trade Name: Cerezyme Product Name: Cerezyme Product Code: imiglucerase INN or Proposed INN: IMIGLUCERASE Other descriptive name: Recombinant human derived macrophage-targeted ß-Glucocerebrosidase | Genzyme Europe B.V. | NULL | Not Recruiting | Female: yes Male: yes | 150 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Spain;Turkey;Russian Federation;Italy;United Kingdom;Egypt;France;Czech Republic;Canada;Argentina;Brazil;Australia;Netherlands;Germany | ||
| 3 | EUCTR2008-005223-28-NL (EUCTR) | 04/11/2009 | 01/07/2009 | A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have Reached Therapeutic Goals with Enzyme Replacement Therapy - ENCORE | A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have Reached Therapeutic Goals with Enzyme Replacement Therapy - ENCORE | Gaucher Disease type I MedDRA version: 9.1;Level: PT;Classification code 10018048;Term: Gaucher's disease | Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: Eliglustat Other descriptive name: not available Trade Name: Cerezyme Product Name: Cerezyme Product Code: Imiglucerase INN or Proposed INN: IMIGLUCERASE Other descriptive name: Recombinant human derived macrophage-targeted ß-Glucocerebrosidase | Genzyme Europe B.V. | NULL | Not Recruiting | Female: yes Male: yes | 186 | Phase 3 | France;Czech Republic;Spain;Germany;Netherlands;Italy;United Kingdom | ||
| 4 | EUCTR2008-005223-28-FR (EUCTR) | 22/10/2009 | 05/08/2009 | A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have been Stabilized with Cerezyme - ENCORE | A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have been Stabilized with Cerezyme - ENCORE | Gaucher Disease type I MedDRA version: 9.1;Level: PT;Classification code 10018048;Term: Gaucher's disease | Product Name: Genz-112638 Product Code: Genz-112638 Trade Name: Cerezyme Product Name: Cerezyme Product Code: imiglucerase INN or Proposed INN: IMIGLUCERASE Other descriptive name: Recombinant human derived macrophage-targeted ß-Glucocerebrosidase | Genzyme Europe B.V. | NULL | Not Recruiting | Female: yes Male: yes | 96 | Phase 3 | France;Czech Republic;Spain;Germany;Netherlands;Italy;United Kingdom | ||
| 5 | EUCTR2008-005223-28-CZ (EUCTR) | 23/09/2009 | 21/08/2009 | A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have Reached Therapeutic goals with Enzyme Replacement Therapy - ENCORE | A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have Reached Therapeutic goals with Enzyme Replacement Therapy - ENCORE | Gaucher Disease type I MedDRA version: 9.1;Level: PT;Classification code 10018048;Term: Gaucher's disease | Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: Eliglustat tartrate Other descriptive name: not available Trade Name: Cerezyme Product Name: Cerezyme Product Code: Imiglucerase INN or Proposed INN: IMIGLUCERASE Other descriptive name: recombinant human derived macrophage-targeted ß-glucocerebrosidase | Genzyme Europe B.V. | NULL | Not Recruiting | Female: yes Male: yes | 186 | Phase 3 | Germany;United Kingdom;Czech Republic;Netherlands;France;Spain;Italy |