256. 筋型糖原病
［臨床試験数：161，薬物数：114（DrugBank：23），標的遺伝子数：26，標的パスウェイ数：106］
Searched query = "Muscle glycogenosis", "Muscular glycogenosis", "Muscle glycogen storage disease", "Muscular glycogen storage disease", "Glycogen storage disease type 0", "GSD0", "Glycogen synthase deficiency", "Glycogen storage disease type II", "GSDII", "Pompe disease", "Alpha-1,4-glucosidase acid deficiency", "Glycogen storage disease type III", "GSDIII", "Cori disease", "Glycogen debranching enzyme deficiency", "Glycogen storage disease type IV", "GSDIV", "Andersen disease", "Glycogen-branching enzyme deficiency", "GBED", "Glycogen storage disease type V", "GSDV", "McArdle disease", "Muscle phosphorylase deficiency", "Muscular phosphorylase deficiency", "Glycogen storage disease type VII", "GSDVII", "Tarui disease", "Phosphofructokinase deficiency", "PFK deficiency", "Glycogen storage disease type IX", "GSDIX", "Phosphorylase kinase deficiency", "Phosphoglycerate kinase deficiency", "PGK deficiency", "Glycogen storage disease type X", "GSDX", "Phosphoglycerate mutase deficiency", "Glycogen storage diseass type XI", "GSDXI", "Kanno disease", "Lactate dehydrogenase deficiency", "Glycogen storage diseass type XII", "GSDXII", "Aldolase A deficiency", "Glycogen storage diseass type XIII", "GSDXIII", "Beta-enolase deficiency", "Glycogen storage diseass type XIV", "GSDXIV", "Phosphoglucomutase deficiency", "Glycogen storage diseass type XV", "GSDXV", "Glycogenin 1 deficiency"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| 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 | ||
| 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 |
| 6 | NCT01924845 (ClinicalTrials.gov) | April 2014 | 13/8/2013 | BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study) | 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 | Late-onset Pompe Disease | Drug: BMN 701 | BioMarin Pharmaceutical | NULL | Terminated | 18 Years | N/A | All | 24 | Phase 3 | United States;Belgium;France;Germany;Italy;Netherlands;Portugal;United Kingdom |
| 7 | 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 | ||
| 8 | 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 | ||
| 9 | 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;Phase 2 | France;United States;Netherlands;Germany;United Kingdom | ||
| 10 | 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 | ||
| 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 |
| 11 | NCT01435772 (ClinicalTrials.gov) | August 15, 2011 | 8/9/2011 | Extension Study for Patients Who Have Participated in a BMN 701 Study | A Long-Term Study for Extended BMN 701 Treatment of Patients With Pompe Disease Who Have Participated in a BMN 701 Study | Pompe Disease | Biological: BMN 701 | BioMarin Pharmaceutical | NULL | Terminated | 13 Years | N/A | All | 21 | Phase 2 | United States;Australia;France;Germany;New Zealand;United Kingdom |
| 12 | 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 | ||
| 13 | NCT01230801 (ClinicalTrials.gov) | January 17, 2011 | 27/10/2010 | Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 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 | Pompe Disease | Biological: BMN 701 | BioMarin Pharmaceutical | NULL | Completed | 13 Years | N/A | All | 22 | Phase 1;Phase 2 | United States;Australia;France;Germany;United Kingdom |