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"
6 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT00567073
(ClinicalTrials.gov)
November 20111/12/2007Pompe Pregnancy Sub-RegistryA Sub-Registry to Observe the Effect of Myozyme (Alglucosidase Alfa) Treatment on Pregnancy and Infant Growth in Women With Pompe DiseaseGlycogen Storage Disease Type II (GSD-II);Pompe Disease (Late-onset);Glycogenesis 2 Acid Maltase DeficiencyBiological: alglucosidase alpha;Other: No TreatmentGenzyme, a Sanofi CompanyNULLRecruitingN/AN/AFemale20United States;Italy
2EUCTR2006-003644-31-DE
(EUCTR)
03/08/200711/07/2007An Open-Label Extension Study of Patients with Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704An Open-Label Extension Study of Patients with Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704 Glycogen Storage Disease type II (Pompe's disease)
MedDRA version: 9.1;Level: LLT;Classification code 10036143;Term: Pompe's disease
Trade Name: Myozyme
Product Name: Myozyme
Product Code: rhGAA, acid alpha glucosidase
INN or Proposed INN: Alglucosidase alfa
Other descriptive name: alglucosidase alpha
Genzyme Europe B.V.NULLNot RecruitingFemale: yes
Male: yes
82Germany;Netherlands;France
3EUCTR2006-003644-31-NL
(EUCTR)
01/08/200704/06/2007An Open-Label Extension Study of Patients with Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704An Open-Label Extension Study of Patients with Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704 Glycogen Storage Disease type II (Pompe's disease)
MedDRA version: 9.1;Level: LLT;Classification code 10036143;Term: Pompe's disease
Trade Name: Myozyme
Product Name: Myozyme
Product Code: rhGAA, acid alpha glucosidase
INN or Proposed INN: Alglucosidase alfa
Other descriptive name: alglucosidase alpha
Genzyme Europe BVNULLNot RecruitingFemale: yes
Male: yes
82Germany;Netherlands;France
4EUCTR2005-002759-42-DE
(EUCTR)
18/07/200724/07/2009A Randomized, Double-Blind, Multicenter, Multinational, Placebo-Controlled Study of the Safety, Efficacy, and Pharmacokinetics of Myozyme, Recombinant Human Acid alpha-Glucosidase (rhGAA), Treatment in Patients with Late-Onset Pompe Disease - LOTSA Randomized, Double-Blind, Multicenter, Multinational, Placebo-Controlled Study of the Safety, Efficacy, and Pharmacokinetics of Myozyme, Recombinant Human Acid alpha-Glucosidase (rhGAA), Treatment in Patients with Late-Onset Pompe Disease - LOTS Glycogen Storage Disease type II (Pompe's disease)
MedDRA version: 9.1;Level: LLT;Classification code 10036143;Term: Pompe's disease
Trade Name: Myozyme
Product Name: Myozyme
INN or Proposed INN: Alglucosidase alfa
Other descriptive name: alglucosidase alpha
Genzyme Europe BVNULLNot RecruitingFemale: yes
Male: yes
90Germany
5EUCTR2006-003644-31-FR
(EUCTR)
18/01/200711/12/2006An Open-Label Extension Study of Patients with Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704An Open-Label Extension Study of Patients with Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704 Glycogen Storage Disease type II (Pompe's disease)Trade Name: Myozyme
Product Name: Myozyme
Product Code: rhGAA, acid alpha glucosidase
INN or Proposed INN: Alglucosidase alfa
Other descriptive name: alglucosidase alpha
Genzyme Europe BVNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
90Germany;Netherlands;France
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2005-002829-31-BE
(EUCTR)
09/02/2006Full title of the trial : A Single Centre, Open-Label, Bridging Study of the Safety, Pharmacokinetics and Efficacy of Recombinant Human Acid Alpha-Glucosidase (rhGAA) Treatment in Patients with Late-Onset Pompe Disease (Glycogen Storage Disease Type II) - mini LOTSFull title of the trial : A Single Centre, Open-Label, Bridging Study of the Safety, Pharmacokinetics and Efficacy of Recombinant Human Acid Alpha-Glucosidase (rhGAA) Treatment in Patients with Late-Onset Pompe Disease (Glycogen Storage Disease Type II) - mini LOTS Glycogen Storage Disease type II (Pompe´s disease)Product Name: Myozyme
Product Code: rhGAA, acid alpha glucosidase
INN or Proposed INN: Alglucosidase alfa
Other descriptive name: alglucosidase alpha
Genzyme Europe BVNULLNot RecruitingFemale: yes
Male: yes
5Phase 2Belgium;United Kingdom