Autologous cd34+ cells transduced with a lentiviral vector encoding the arsa cdna (DrugBank: -)
1 diseaseID | Disease name (Link within this page) | Number of trials |
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19 | Lysosomal storage disease | 1 |
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 |
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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;Phase 2 | Italy |