256. 筋型糖原病 Muscle glycogenosis Clinical trials / Disease details
臨床試験数 : 180 / 薬物数 : 133 - (DrugBank : 29) / 標的遺伝子数 : 25 - 標的パスウェイ数 : 105
| 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 | EUCTR2013-001768-48-AT (EUCTR) | 21/02/2016 | 12/01/2016 | A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease | A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease | Pompe disease MedDRA version: 18.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: BMN 701 Product Code: BMN 701 INN or Proposed INN: reveglucosidase alpha Other descriptive name: GILT-rhGAA Product Name: BMN 701 Product Code: BMN 701 INN or Proposed INN: reveglucosidase alpha Other descriptive name: GILT-rhGAA | BioMarin Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 70 | Phase 3 | Portugal;France;United States;Belgium;Austria;Netherlands;Germany;Japan;Italy;United Kingdom;Switzerland | ||
| 2 | EUCTR2013-001768-48-DE (EUCTR) | 23/09/2014 | 22/05/2014 | A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease | A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease | Pompe disease MedDRA version: 18.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: BMN 701 Product Code: BMN 701 INN or Proposed INN: reveglucosidase alpha Other descriptive name: GILT-rhGAA Product Name: BMN 701 Product Code: BMN 701 INN or Proposed INN: reveglucosidase alpha Other descriptive name: GILT-rhGAA | BioMarin Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | Portugal;France;United States;Argentina;Belgium;Austria;Netherlands;Colombia;Germany;Japan;Italy;United Kingdom | ||
| 3 | EUCTR2013-001768-48-BE (EUCTR) | 08/09/2014 | 12/05/2014 | A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease | A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease | Pompe disease MedDRA version: 18.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: BMN 701 Product Code: BMN 701 INN or Proposed INN: reveglucosidase alpha Other descriptive name: GILT-rhGAA Product Name: BMN 701 Product Code: BMN 701 INN or Proposed INN: reveglucosidase alpha Other descriptive name: GILT-rhGAA | BioMarin Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | Portugal;France;United States;Argentina;Belgium;Austria;Netherlands;Germany;Colombia;Japan;Italy;United Kingdom |