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 | EUCTR2005-002759-42-DE (EUCTR) | 18/07/2007 | 24/07/2009 | A 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 | A 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 BV | NULL | Not Recruiting | Female: yes Male: yes | 90 | Germany | |||
| 2 | EUCTR2005-002829-31-GB (EUCTR) | 31/08/2005 | 07/07/2005 | Full 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 | Full 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) | Genzyme Europe BV | NULL | Not Recruiting | Female: yes Male: yes | 5 | Phase 2 | Belgium;United Kingdom | |||
| 3 | EUCTR2004-002168-59-IT (EUCTR) | 14/10/2004 | 03/01/2005 | An Open-Label, Multicenter, Multinational, Study of the Safety, Efficacy,Pharmacokinetics, and Pharmacodynamics of Recombinant Human Acid alpha-Glucosidase (rhGAA) Treatment in Patients > 6 and <= 36 Months Old with Infantile-OnsetPompe Disease (Glycogen Storage Disease Type II) | An Open-Label, Multicenter, Multinational, Study of the Safety, Efficacy,Pharmacokinetics, and Pharmacodynamics of Recombinant Human Acid alpha-Glucosidase (rhGAA) Treatment in Patients > 6 and <= 36 Months Old with Infantile-OnsetPompe Disease (Glycogen Storage Disease Type II) | Treatment for Type II Glycogenosis MedDRA version: 6.1;Level: PT;Classification code 10053185 | Product Name: Recombinant human acid alfa-glucosidase (rhGAA) Product Code: NA INN or Proposed INN: alglucosidase alfa Product Name: Recombinant human acid alfa-glucosidase (rhGAA) Product Code: NA INN or Proposed INN: alglucosidase alfa | GENZYME | NULL | Not Recruiting | Female: yes Male: yes | 20 | Italy | |||
| 4 | NCT00051935 (ClinicalTrials.gov) | January 2003 | 17/1/2003 | A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II | Open-Label, Pilot Study of the Safety, Pharmacokinetics and Pharmacodynamics of Recombinant Human Acid Alpha-Glucosidase (rhGAA) as Enzyme Replacement Therapy in Siblings With Glycogen Storage Disease Type II (GSD-II). | Glycogen Storage Disease Type II;Pompe Disease;Acid Maltase Deficiency Disease;Glycogenosis 2 | Drug: Alglucosidase alfa | Genzyme, a Sanofi Company | NULL | Completed | N/A | N/A | Both | 2 | Phase 2 | United States |
| 5 | NCT00025896 (ClinicalTrials.gov) | May 2001 | 31/10/2001 | Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease | A Prospective Multinational, Multicenter, Clinical Trial of the Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase (rhGAA) in Cross-Reacting Immunologic Material-Positive Patients With Classical Infantile Pompe Disease | Pompe Disease;Glycogen Storage Disease Type II;Acid Maltase Deficiency Disease;Glycogenosis 2 | Drug: recombinant human acid alpha-glucosidase (rhGAA) | Genzyme, a Sanofi Company | NULL | Completed | N/A | N/A | Both | 8 | Phase 2 | United States |
| 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 | EUCTR2005-002829-31-BE (EUCTR) | 09/02/2006 | Full 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 | Full 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 BV | NULL | Not Recruiting | Female: yes Male: yes | 5 | Phase 2 | Belgium;United Kingdom |