19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details
臨床試験数 : 854 / 薬物数 : 716 - (DrugBank : 105) / 標的遺伝子数 : 70 - 標的パスウェイ数 : 191
| 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 | EUCTR2021-002320-20-IT (EUCTR) | 14/01/2022 | 22/10/2021 | A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry disease | A randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglustat compared with usual standard of care on left ventricular mass index in participants with Fabry disease and left ventricular hypertrophy - . | Fabry's disease MedDRA version: 24.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Fabrazyme Product Name: . Product Code: [.] INN or Proposed INN: AGALSIDASI BETA Trade Name: Galafold Product Name: . Product Code: [.] INN or Proposed INN: Migalastat Trade Name: Replagal Product Name: . Product Code: [.] INN or Proposed INN: AGALSIDASI ALFA Product Name: Venglustat Product Code: [SAR402671, GZ402671 o GZ/SAR402671] INN or Proposed INN: venglustat malato Other descriptive name: GZ/SAR402671 | SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Canada;Poland;Denmark;Norway;Germany;Netherlands;China;Japan;Korea, Republic of | ||
| 2 | EUCTR2018-000368-27-IT (EUCTR) | 22/10/2018 | 04/11/2020 | ND | ND - ND | Fabry disease MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Digestive System Diseases [C06] | Trade Name: FABRAZYME - 35 MG POLVERE PER CONCENTRATO PER SOLUZIONE PER INFUSIONE ENDOVENOSA 1 FLACONCINO Product Name: fabrazyme Product Code: [A16AB04] INN or Proposed INN: AGALSIDASI BETA | FONDAZIONE POLICLINICO UNIVERSITARIO AGOSTINO GEMELLI IRCCS UNIVERSITA' CATTOLICA DEL SACRO CUORE | NULL | Not Recruiting | Female: yes Male: yes | 14 | Phase 4 | Italy |