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 | JPRN-JapicCTI-194887 | 17/9/2019 | 26/07/2019 | ATB200/AT2221phase3 double blind randomized study | A phase 3 double-blind randomized study to assess the efficacy and safety of intravenous ATB200 co-administered with oral AT2221 in adult subjects with late- onset pompe disease compared with alglucosidase alfa/placebo | Pompe disease | Intervention name : Recombinant human acid alfa-glucosidase (ATB200) INN of the intervention : Recombinant human acid alfa-glucosidase Dosage And administration of the intervention : 20 mg/kg IV infusion over a 4-hour duration 1 hour after AT2221(Miglustat) administration every 2 weeks Intervention name : Miglustat (AT2221) INN of the intervention : Miglustat Dosage And administration of the intervention : Subjects weighing >= 50 kg, 260mg (4 oral capsules of AT2221) 1hour prior to ATB200 infusion every 2 weeks. Subjects weighing >= 40 kg to < 50 kg, 195 mg (3 oral capsules) 1 hour prior to ATB200 infusion every 2 weeks. Control intervention name : Alglucosidase Alfa (Genetical Recombination) INN of the control intervention : Alglucosidase Alfa (Genetical Recombination) Dosage And administration of the control intervention : 20 mg/kg IV infusion over a 4-hour duration 1 hour after placebo administration every 2 weeks Control intervention name : Plecebo INN of the control intervention : - Dosage And administration of the control intervention : - | Amicus Therapeutics, Inc./CMIC Co., Ltd. | NULL | complete | 18 | BOTH | 110 | Phase 3 | Japan, Asia except Japan, North America, South America, Europe, Oceania | |
| 2 | 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 |