19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 854 / Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 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 | EUCTR2009-017349-77-IT (EUCTR) | 15/03/2010 | 26/04/2010 | A Phase I/II clinical trial of hematopoietic stem cell gene therapy for the treatment of Metachromatic Leukodystrophy - TIGET-MLD | A Phase I/II clinical trial of hematopoietic stem cell gene therapy for the treatment of Metachromatic Leukodystrophy - TIGET-MLD | metachromatic leukodystrophy MedDRA version: 9.1;Level: PT;Classification code 10024381 | Product Name: autologous CD34+ cells transduced with a lentiviral vector encoding the ARSA cDNA Trade Name: BUSILVEX INN or Proposed INN: Busulfan | FONDAZIONE CENTRO S. RAFFAELE DEL MONTE TABOR | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Phase 1/2 | Italy |