19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 899 / Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 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 | EUCTR2016-002328-10-NL (EUCTR) | 29/10/2018 | 07/11/2018 | Gene Therapy in patients with Mucopolysaccharidosis disease | A Phase I/II Open Label, Dose Escalation, Safety Study in Subjects withMucopolysaccharidosis type VI (MPS VI) Using Adeno-Associated ViralVector 8 to Deliver the human ARSB gene to Liver. | The clinical trial will be conducted on patients withMucopolysaccharidosis Type VI. MPS VI is characterized by growthretardation, corneal clouding, cardiac valve disease, organomegaly, skeletal dysplasia, without central nervous system involvement;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: AAV2/8.TBG.hARSB Product Code: not applicable INN or Proposed INN: NA Other descriptive name: ADENO-ASSOCIATED VIRAL (AAV) SEROTYPE 8 (AAV2/8) VECTOR WITH LIVER-SPECIFIC THYROXINE-BINDING GLOBULIN (TBG) PROMOTER, DRIVING THE EXPRESSION OF THE HUMAN ARSB GENE | FONDAZIONE TELETHON | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 10 | Phase 1 | Turkey;Netherlands;Italy |