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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
14 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2016-000942-77-PT
(EUCTR)
24/07/201812/04/2018Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe DiseaseA phase 3 randomized, multicenter, multinational, double-blinded study comparing the efficacy and safety of repeated biweekly infusions of neoGAA (GZ402666) and alglucosidase alfa in treatment-naïve patients with late onset Pompe disease Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: neoGAA
Product Code: GZ402666
INN or Proposed INN: neoGAA
Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA)
Trade Name: MYOZYME®
Product Name: Myozyme®
INN or Proposed INN: ALGLUCOSIDASE ALFA
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
96Phase 3United States;Portugal;Taiwan;Spain;Russian Federation;Colombia;Switzerland;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Czech Republic;Mexico;Canada;Argentina;Poland;Brazil;Belgium;Bulgaria;Germany;Japan;Sweden
2EUCTR2016-003475-21-GB
(EUCTR)
17/05/201703/01/2017A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated with Alglucosidase AlfaAn Open-label Ascending Dose Cohort Study to Assess the Safety, Pharmacokinetics, and Preliminary Efficacy of Avalglucosidase Alfa (NeoGAA, GZ402666) in Patients with Infantile-onset Pompe Disease Treated with Alglucosidase Alfa Who Demonstrate Clinical Decline or Sub-optimal Clinical Response Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.1;Level: PT;Classification code 10053185;Term: Glycogen storage disease type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Avalglucosidase Alfa (NeoGAA)
Product Code: GZ402666
INN or Proposed INN: Avalglucosidase Alfa (NeoGAA)
Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan Avalglucosidase Alfa (NeoGAA)
Trade Name: MYOZYME®
Product Name: Myozyme®
INN or Proposed INN: ALGLUCOSIDASE ALFA
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
34Phase 2United States;France;Taiwan;Germany;Japan;United Kingdom
3EUCTR2016-000942-77-NL
(EUCTR)
21/03/201715/06/2016Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe DiseaseA phase 3 randomized, multicenter, multinational, double-blinded study comparing the efficacy and safety of repeated biweekly infusions of neoGAA (GZ402666) and alglucosidase alfa in treatment-naïve patients with late onset Pompe disease Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: neoGAA
Product Code: GZ402666
INN or Proposed INN: NEO-GAA
Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA)
Trade Name: MYOZYME®
Product Name: Myozyme®
INN or Proposed INN: ALGLUCOSIDASE ALFA
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
96Phase 3Portugal;United States;Taiwan;Spain;Russian Federation;Colombia;Switzerland;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Bulgaria;Germany;Japan;Sweden
4EUCTR2016-000942-77-DE
(EUCTR)
18/11/201627/06/2016Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe DiseaseA Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Avalglucosidase Alfa (neoGAA, GZ402666) and Alglucosidase Alfa in Treatment naïve Patients with Late-onset Pompe Disease Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: neoGAA or avalglucosidase alfa
Product Code: GZ402666
Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA)
Trade Name: MYOZYME®
Product Name: Myozyme®
INN or Proposed INN: ALGLUCOSIDASE ALFA
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
100Phase 3United States;Portugal;Taiwan;Spain;Russian Federation;Colombia;Switzerland;Italy;France;Denmark;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Bulgaria;Germany;Japan;Sweden
5EUCTR2016-000942-77-ES
(EUCTR)
20/10/201622/06/2016Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe DiseaseA Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of NeoGAA (GZ402666) and Alglucosidase Alfa in Treatmentnaïve Patients with Late-onset Pompe Disease Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 19.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: neoGAA
Product Code: GZ402666
INN or Proposed INN: NeoGAA
Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA)
Trade Name: MYOZYME®
Product Name: Myozyme®
INN or Proposed INN: ALGLUCOSIDASE ALFA
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
90Phase 3United States;Taiwan;Spain;Turkey;Austria;Russian Federation;Colombia;United Kingdom;Switzerland;Italy;France;Czech Republic;Mexico;Canada;Argentina;Belgium;Brazil;Denmark;Netherlands;Germany;Japan;Sweden;Korea, Republic of
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2016-000942-77-BE
(EUCTR)
14/10/201601/07/2016Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe DiseaseA Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of NeoGAA (GZ402666) and Alglucosidase Alfa in Treatmentnaïve Patients with Late-onset Pompe Disease - COMET Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: neoGAA
Product Code: GZ402666
Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA)
Trade Name: MYOZYME®
Product Name: Myozyme®
INN or Proposed INN: ALGLUCOSIDASE ALFA
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
100Phase 3United States;Portugal;Taiwan;Spain;Russian Federation;Colombia;Switzerland;Italy;France;Denmark;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Germany;Japan;Sweden
7EUCTR2016-000942-77-CZ
(EUCTR)
05/10/201627/06/2016Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe DiseaseA phase 3 randomized, multicenter, multinational, double-blinded study comparing the efficacy and safety of repeated biweekly infusions of neoGAA (GZ402666) and alglucosidase alfa in treatment-naïve patients with late onset Pompe disease Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: neoGAA
Product Code: GZ402666
Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA)
Trade Name: MYOZYME®
Product Name: Myozyme®
INN or Proposed INN: ALGLUCOSIDASE ALFA
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
96Phase 3Portugal;United States;Taiwan;Spain;Russian Federation;Colombia;Switzerland;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Bulgaria;Germany;Japan;Sweden
8EUCTR2013-003321-28-DK
(EUCTR)
14/11/201418/09/2014Avalglucosidase Alfa Extension StudyAn open-label, multicenter, multinational extension study of the long-term safety and pharmacokinetics of repeated biweekly infusions of Avalglucosidase Alfa (NeoGAA, GZ402666) in patients with Pompe disease Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.1;Level: PT;Classification code 10053185;Term: Glycogen storage disease type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Avalglucosidase Alfa
Product Code: GZ402666
Other descriptive name: RECOMBINANT HUMAN ?-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
21Phase 2United States;France;Belgium;Denmark;Netherlands;Germany;United Kingdom;Italy
9EUCTR2013-003321-28-DE
(EUCTR)
20/10/201425/09/2014Avalglucosidase Alfa Extension StudyAn open-label, multicenter, multinational extension study of the long-term safety and pharmacokinetics of repeated biweekly infusions of Avalglucosidase Alfa (NeoGAA, GZ402666) in patients with Pompe disease Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.1;Level: PT;Classification code 10053185;Term: Glycogen storage disease type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Avalglucosidase Alfa
Product Code: GZ402666
Other descriptive name: RECOMBINANT HUMAN ?-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
21Phase 2United States;France;Belgium;Denmark;Netherlands;Germany;United Kingdom;Italy
10EUCTR2013-003321-28-NL
(EUCTR)
25/09/201416/07/2014NeoGAA Extension StudyAn open-label, multicenter, multinational extension study of the long-term safety and pharmacokinetics of repeated biweekly infusions of neoGAA in patients with Pompe disease Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.1;Level: PT;Classification code 10053185;Term: Glycogen storage disease type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: neoGAA
Product Code: GZ402666
INN or Proposed INN: GZ402666
Other descriptive name: RECOMBINANT HUMAN ?-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
21Phase 2France;United States;Belgium;Denmark;Germany;Netherlands;Italy;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2013-003321-28-BE
(EUCTR)
12/05/201417/02/2014Avalglucosidase Alfa Extension StudyAn Open-Label, Multicenter, Multinational Extension Study Of The Long-Term Safety And Pharmacokinetics Of Repeated Biweekly Infusions Of Avalglucosidase Alfa (NeoGAA, GZ402666) In Patients With Pompe Disease Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.1;Level: PT;Classification code 10053185;Term: Glycogen storage disease type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Avalglucosidase Alfa
Product Code: GZ402666
Other descriptive name: RECOMBINANT HUMAN ?-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
21Phase 2France;United States;Belgium;Denmark;Netherlands;Germany;Italy;United Kingdom
12EUCTR2013-003321-28-FR
(EUCTR)
19/03/201417/06/2015NeoGAA Extension StudyAn open-label, multicenter, multinational extension study of the long-term safety and pharmacokinetics of repeated biweekly infusions of neoGAA in patients with Pompe disease Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 18.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: neoGAA
Product Code: GZ402666
Other descriptive name: RECOMBINANT HUMAN ?-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
18United States;France;Belgium;Denmark;Netherlands;Germany;Italy;United Kingdom
13EUCTR2016-003475-21-FR
(EUCTR)
02/06/2017A Study to Assess Safety and Efficacy of NeoGAA Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated with Alglucosidase AlfaAn Open-label Ascending Dose Cohort Study to Assess the Safety, Pharmacokinetics, and Preliminary Efficacy of NeoGAA (GZ402666) in Patients with Infantile-onset Pompe Disease Treated with Alglucosidase Alfa Who Demonstrate Clinical Decline or Sub-optimal Clinical Response Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 20.0;Level: PT;Classification code 10053185;Term: Glycogen storage disease type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: neoGAA
Product Code: GZ402666
INN or Proposed INN: -
Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA)
Trade Name: MYOZYME®
Product Name: Myozyme®
INN or Proposed INN: ALGLUCOSIDASE ALFA
Genzyme CorporationNULLNAFemale: yes
Male: yes
34Phase 2United States;France;Taiwan;Germany;Japan;United Kingdom
14EUCTR2016-000942-77-PL
(EUCTR)
23/11/2017Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe DiseaseA phase 3 randomized, multicenter, multinational, double-blinded study comparing the efficacy and safety of repeated biweekly infusions of Avalglucosidase Alfa (neoGAA ,GZ402666) and alglucosidase alfa in treatment-naïve patients with late onset Pompe disease Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: neoGAA or avalglucosidase alfa
Product Code: GZ402666
Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA)
Trade Name: MYOZYME®
Product Name: Myozyme®
INN or Proposed INN: ALGLUCOSIDASE ALFA
Genzyme CorporationNULLNAFemale: yes
Male: yes
100Phase 3United States;Portugal;Taiwan;Spain;Russian Federation;Colombia;Switzerland;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Argentina;Belgium;Poland;Bulgaria;Germany;Japan;Sweden