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 |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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-PT (EUCTR) | 30/01/2015 | 02/07/2014 | 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: GILT-rhGAA Other descriptive name: GILT-rhGAA Product Name: BMN 701 Product Code: BMN 701 INN or Proposed INN: GILT-rhGAA Other descriptive name: GILT-rhGAA | BioMarin Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | France;United States;Portugal;Argentina;Belgium;Austria;Netherlands;Germany;Colombia;Japan;Italy;United Kingdom | ||
| 3 | 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 | ||
| 4 | EUCTR2013-001768-48-IT (EUCTR) | 22/09/2014 | 01/07/2014 | 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: 17.0;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: GILT-rhGAA 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;United Kingdom;Italy | ||
| 5 | 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 | ||
| 6 | 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 | ||
| 7 | EUCTR2011-001805-28-DE (EUCTR) | 14/02/2013 | 28/06/2012 | A long term study of the safety of BMN 701 in patients with POMPE disease | A Long-Term Study for Extended BMN 701Treatment of Patients with Pompe Disease who have Participated in a BMN 701 Study | 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 Other descriptive name: GILT-rhGAA | BioMarin Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 21 | Phase 2 | United States;Australia;Germany;New Zealand;United Kingdom | ||
| 8 | EUCTR2010-023561-22-DE (EUCTR) | 04/07/2012 | 07/02/2012 | A Study of the Safety and Tolerability of BMN 701 in Patients with Late-onset Pompe Disease | A Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant human GAA) in Patients with Late-onset Pompe Disease - A Phase 1/2 Study of BMN 701 in Patients with Late-onset Pompe Disease | Pompe disease MedDRA version: 16.0;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 Other descriptive name: GILT-rhGAA | BioMarin Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 1/2 | France;United States;Netherlands;Germany;United Kingdom | ||
| 9 | EUCTR2011-001805-28-GB (EUCTR) | 03/07/2012 | 26/04/2012 | A Long-Term Study for Extended BMN 701Treatment of Patients with Pompe Disease who have Participated in a BMN 701 Study | A Long-Term Study for Extended BMN 701Treatment of Patients with Pompe Disease who have Participated in a BMN 701 Study | 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 Other descriptive name: GILT-rhGAA | BioMarin Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 2 | United States;France;Australia;Germany;United Kingdom | ||
| 10 | EUCTR2010-023561-22-GB (EUCTR) | 15/08/2011 | 11/04/2011 | A Study of the Safety and Tolerability of BMN 701 in Patients with Late-onset Pompe Disease | A Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant human GAA) in Patients with Late-onset Pompe Disease - A Phase 1/2 Study of BMN 701 in Patients with Late-onset Pompe Disease | Pompe disease MedDRA version: 13.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: BMN 701 Product Code: BMN 701 Other descriptive name: GILT-rhGAA | BioMarin Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 28 | Phase 1;Phase 2 | United States;Germany;United Kingdom |