256. 筋型糖原病 Muscle glycogenosis Clinical trials / Disease details
臨床試験数 : 180 / 薬物数 : 153 - (DrugBank : 30) / 標的遺伝子数 : 31 - 標的パスウェイ数 : 134
| 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) | August 15, 2019 | 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 | 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 | ||||
| 4 | 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 |