256. Muscle glycogenosis Clinical trials / Disease details
Clinical trials : 180 / Drugs : 153 - (DrugBank : 30) / Drug target genes : 31 - Drug target pathways : 134
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 | EUCTR2013-001768-48-GB (EUCTR) | 27/11/2013 | 22/10/2013 | 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 alfa Other descriptive name: GILT-rhGAA | BioMarin Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 70 | Phase 3 | Portugal;France;United States;Belgium;Austria;Denmark;Netherlands;Germany;Colombia;Japan;Italy;United Kingdom |