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 | EUCTR2015-003105-41-GB (EUCTR) | 13/10/2015 | 08/09/2015 | Treatment for Nonsense Mutation Mucopolysaccharidosis Type I | CNS Unmet Medical Need in Mucopolysaccharidosis: A Phase 2 Safety and Pharmacokinetics Study of Ataluren (COMPASS) - COMPASS | Nonsense mutation Mucopolysaccharidosis Type I MedDRA version: 18.1;Level: PT;Classification code 10056886;Term: Mucopolysaccharidosis I;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN | PTC Therapeutics Inc. | NULL | Not Recruiting | Female: yes Male: yes | 5 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United Kingdom | ||
| 2 | EUCTR2014-002596-28-GB (EUCTR) | 28/01/2015 | 12/06/2015 | Treatment for Nonsense Mutation Mucopolysaccharidosis Type I | A Phase 2 Study of Ataluren (PTC124) as Treatment for Nonsense Mutation Mucopolysaccharidosis Type I | Nonsense mutation Mucopolysaccharidosis Type I MedDRA version: 20.1;Level: PT;Classification code 10056886;Term: Mucopolysaccharidosis I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN | PTC Therapeutics Inc. | NULL | Not Recruiting | Female: yes Male: yes | 18 | Phase 2 | Germany;United Kingdom |