19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details
臨床試験数 : 899 / 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182
| 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 | EUCTR2021-002550-82-NL (EUCTR) | 22/12/2022 | 30/07/2022 | Effectiveness of ambroxol for treating children and adults with Gaucher disease type 3 | Effectiveness of ambroxol in children and adults with Gaucher disease 3: n-of-1 series - ATTACK-GD3 | Gaucher disease type 3 MedDRA version: 20.0;Level: HLGT;Classification code 10027424;Term: Metabolic and nutritional disorders congenital;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Ambroxol hydrochloride INN or Proposed INN: AMBROXOL Hydrochloride Other descriptive name: Mucoangin Product Name: Ambroxol hydrochloride INN or Proposed INN: AMBROXOL HYDROCHLORIDE Other descriptive name: AMBROXOL HYDROCHLORIDE PH. EUR. | Amsterdam UMC | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 4 | Phase 3 | Netherlands |