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"
4 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT02525172August 201510/8/2015Immune Modulation Therapy for Pompe DiseaseImmune Modulation Therapy for ERT-naïve or ERT-treated Pompe Disease PatientsPompe DiseaseDrug: Rituximab;Drug: intravenous immune globulin;Drug: Bortezomib;Drug: MethotrexateNational Taiwan University HospitalNULLRecruitingN/AN/ABoth8Phase 4Taiwan
2NCT00701129October 200917/6/2008An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe DiseaseAn Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme®-Naive, CRIM(-) Patients With Infantile-onset Pompe DiseasePompe Disease;Glycogen Storage Disease Type IIBiological: Alglucosidase Alfa;Drug: Methotrexate;Drug: RituximabGenzyme, a Sanofi CompanyNULLCompletedN/AN/AAll4Phase 4United States;Israel
3EUCTR2015-000583-34-Outside-EU/EEA15/04/2015Immune Tolerance Induction StudyAn Exploratory Study of the Safety and Efficacy of Immune Tolerance Induction (ITI) in Patients with Pompe Disease Who Have Previously Received Myozyme Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 17.1Level: LLTClassification code 10036143Term: Pompe's diseaseSystem Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Alglucosidase alfa
Product Code: Myozyme®
INN or Proposed INN: ALGLUCOSIDASE ALFA
Product Name: Cyclophosphamide
INN or Proposed INN: CYCLOPHOSPHAMIDE MONOHYDRATE
Other descriptive name: CYCLOPHOSPHAMIDE MONOHYDRATE
Product Name: Rituximab
INN or Proposed INN: RITUXIMAB
Product Name: Methotrexate
INN or Proposed INN: METHOTREXATE SODIUM
Other descriptive name: METHOTREXATE SODIUM
Genzyme Corporation IncNULLNAFemale: yes
Male: yes
9United States;Israel
4EUCTR2015-000584-14-Outside-EU/EEA15/04/2015An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe DiseaseAn Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naïve CRIM(-) Patients with Infantile-Onset Pompe Disease Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 17.1Level: LLTClassification code 10036143Term: Pompe's diseaseSystem Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Myozyme®
INN or Proposed INN: ALGLUCOSIDASE ALFA
Product Name: Rituximab
INN or Proposed INN: RITUXIMAB
Product Name: Methotrexate
INN or Proposed INN: METHOTREXATE SODIUM
Other descriptive name: METHOTREXATE SODIUM
Genzyme Corporation IncNULLNAFemale: yes
Male: yes
4United States