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

  
10 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2013-001768-48-AT
(EUCTR)
21/02/201612/01/2016A 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 DiseaseA 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 IncNULLNot RecruitingFemale: yes
Male: yes
70Phase 3Portugal;France;United States;Belgium;Austria;Netherlands;Germany;Japan;Italy;United Kingdom;Switzerland
2EUCTR2013-001768-48-PT
(EUCTR)
30/01/201502/07/2014A 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 DiseaseA 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 IncNULLNot RecruitingFemale: yes
Male: yes
50Phase 3France;United States;Portugal;Argentina;Belgium;Austria;Netherlands;Germany;Colombia;Japan;Italy;United Kingdom
3EUCTR2013-001768-48-DE
(EUCTR)
23/09/201422/05/2014A 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 DiseaseA 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 IncNULLNot RecruitingFemale: yes
Male: yes
50Phase 3Portugal;France;United States;Argentina;Belgium;Austria;Netherlands;Colombia;Germany;Japan;Italy;United Kingdom
4EUCTR2013-001768-48-IT
(EUCTR)
22/09/201401/07/2014A 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 DiseaseA 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 IncNULLNot RecruitingFemale: yes
Male: yes
50Phase 3Portugal;France;United States;Argentina;Belgium;Austria;Netherlands;Germany;Colombia;Japan;United Kingdom;Italy
5EUCTR2013-001768-48-BE
(EUCTR)
08/09/201412/05/2014A 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 DiseaseA 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 IncNULLNot RecruitingFemale: yes
Male: yes
50Phase 3Portugal;France;United States;Argentina;Belgium;Austria;Netherlands;Germany;Colombia;Japan;Italy;United Kingdom
6EUCTR2013-001768-48-GB
(EUCTR)
27/11/201322/10/2013A 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 DiseaseA 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 IncNULLNot RecruitingFemale: yes
Male: yes
70Phase 3Portugal;France;United States;Belgium;Austria;Denmark;Netherlands;Germany;Colombia;Japan;Italy;United Kingdom
7EUCTR2011-001805-28-DE
(EUCTR)
14/02/201328/06/2012A long term study of the safety of BMN 701 in patients with POMPE diseaseA 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 IncNULLNot RecruitingFemale: yes
Male: yes
21Phase 2United States;Australia;Germany;New Zealand;United Kingdom
8EUCTR2010-023561-22-DE
(EUCTR)
04/07/201207/02/2012A Study of the Safety and Tolerability of BMN 701 in Patients with Late-onset Pompe DiseaseA 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 IncNULLNot RecruitingFemale: yes
Male: yes
30Phase 1/2France;United States;Netherlands;Germany;United Kingdom
9EUCTR2011-001805-28-GB
(EUCTR)
03/07/201226/04/2012A Long-Term Study for Extended BMN 701Treatment of Patients with Pompe Disease who have Participated in a BMN 701 StudyA 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 IncNULLNot RecruitingFemale: yes
Male: yes
30Phase 2United States;France;Australia;Germany;United Kingdom
10EUCTR2010-023561-22-GB
(EUCTR)
15/08/201111/04/2011A Study of the Safety and Tolerability of BMN 701 in Patients with Late-onset Pompe DiseaseA 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 IncNULLNot RecruitingFemale: yes
Male: yes
28Phase 1;Phase 2United States;Germany;United Kingdom