256. 筋型糖原病 Muscle glycogenosis Clinical trials / Disease details
臨床試験数 : 180 / 薬物数 : 133 - (DrugBank : 29) / 標的遺伝子数 : 25 - 標的パスウェイ数 : 105
| 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 | EUCTR2020-004686-39-IT (EUCTR) | 27/04/2021 | 07/06/2021 | Clinical Study for IOPD Participants Less Than or Equal to 6 Months of age to Evaluate Efficacy and Safety of enzyme replacement therapy (ERT) WithAvalglucosidase Alfa(Baby-COMET) | An Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of avalglucosidase alfa inTreatment-naïve Pediatric Participants Less than or Equal to 6 Months of Age with Infantile-Onset Pompe Disease (IOPD) - Baby-COMET | Glycogen storage disease type II MedDRA version: 20.1;Level: PT;Classification code 10053185;Term: Glycogen storage disease type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Avalglucosidase Alfa Product Code: [GZ402666] INN or Proposed INN: avalglucosidasi alfa Other descriptive name: RECOMBINANT HUMAN ALFA-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN | SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 3 | France;United States;Taiwan;Belgium;Netherlands;Germany;United Kingdom;China;Italy |