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 | NCT04327973 (ClinicalTrials.gov) | September 1, 2021 | 27/3/2020 | Expanded Access for ATB200/AT2221 for the Treatment of IOPD | Expanded Use Of Recombinant Human Acid Alpha-Glucosidase/N-butyl-deoxynojirimycin (ATB200/AT2221) For Patients With Infantile-Onset Pompe Disease | Pompe Disease Infantile-Onset | Biological: ATB200;Drug: AT2221 | Amicus Therapeutics | NULL | Available | N/A | 17 Years | All | United States;Italy;Taiwan | ||
| 2 | EUCTR2011-002154-32-GB (EUCTR) | 07/12/2011 | 07/10/2011 | Drug-drug interaction study between AT2220 and ERT | AN OPEN-LABEL MULTI-CENTER, INTERNATIONAL STUDY TO INVESTIGATE DRUG-DRUG INTERACTIONS BETWEEN AT2220 AND ALGLUCOSIDASE ALFA IN PATIENTS WITH POMPE DISEASE | Pompe Disease MedDRA version: 14.1;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: Duvoglustat hydrochloride Product Code: AT2220 INN or Proposed INN: Duvoglustat hydrochloride Other descriptive name: 1-deoxynojirimycin hydrochloride Product Name: Duvoglustat hydrochloride Product Code: AT2220 INN or Proposed INN: Duvoglustat hydrochloride Other descriptive name: 1-deoxynojirimycin hydrochloride | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 22 | United States;France;Canada;United Kingdom | |||
| 3 | NCT01380743 (ClinicalTrials.gov) | October 31, 2011 | 23/6/2011 | Drug-drug Interaction Study | An Open-Label, Multi-Center, International Study to Investigate Drug-Drug Interactions Between AT2220 and Alglucosidase Alfa in Patients With Pompe Disease | Pompe Disease | Drug: duvoglustat;Drug: rhGAA | Amicus Therapeutics | NULL | Completed | 18 Years | 65 Years | All | 25 | Phase 2 | United States;Canada;France;United Kingdom |
| 4 | EUCTR2008-002302-18-DE (EUCTR) | 16/02/2009 | 26/09/2008 | An open-label, multicenter, study to evaluate the safety, tolerability, pharmacodynamics, and pharmacokinetics of three dosing regimens of oral AT2220 in patients with Pompe disease - | Pompe Disease MedDRA version: 9.1;Level: LLT;Classification code 10036143;Term: Pompe's disease | Product Code: AT2220 Other descriptive name: 1-deoxynojirimycin hydrochloride Product Code: AT2220 Other descriptive name: 1-deoxynojirimycin hydrochloride | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 18 | United Kingdom;Germany | ||||
| 5 | EUCTR2008-002302-18-GB (EUCTR) | 18/12/2008 | 07/01/2009 | An open-label, multicenter, study to evaluate the safety, tolerability, pharmacodynamics, and pharmacokinetics of three dosing regimens of oral AT2220 in patients with Pompe disease - | Pompe Disease MedDRA version: 9.1;Level: LLT;Classification code 10036143;Term: Pompe's disease | Product Code: AT2220 Other descriptive name: 1-deoxynojirimycin hydrochloride Product Code: AT2220 Other descriptive name: 1-deoxynojirimycin hydrochloride | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 18 | Germany;United Kingdom | ||||
| 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 | NCT00688597 (ClinicalTrials.gov) | December 8, 2008 | 30/5/2008 | Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease | An Open-Label, Multicenter Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of Three Dosing Regimens of Oral AT2220 in Patients With Pompe Disease | Pompe Disease | Drug: Duvoglustat | Amicus Therapeutics | NULL | Terminated | 18 Years | 74 Years | All | 3 | Phase 2 | United States;Australia;Canada;France;Germany;Netherlands;United Kingdom |
| 7 | EUCTR2013-002257-30-GB (EUCTR) | 21/10/2013 | A dose finding study with intravenous administration of duvoglustat hydrochloride (AT2220) and Myozyme in Pompe patients | AN OPEN-LABEL SAFETY AND DOSE-FINDING STUDY OF INTRAVENOUS DUVOGLUSTAT CO-ADMINISTERED WITH RECOMBINANT HUMAN ACID a-GLUCOSIDASE IN SUBJECTS WITH POMPE DISEASE | Pompe disease MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 24 | Phase 2 | United States;Netherlands;United Kingdom |