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 | NCT03725670 (ClinicalTrials.gov) | October 30, 2018 | 25/9/2018 | Lentiviral Gene Therapy for MLD | Gene Therapy for Metachromatic Leukodystrophy (MLD) Using a Self-inactivating Lentiviral Vector (TYF-ARSA) | Metachromatic Leukodystrophy (MLD) | Biological: Lentivirus-mediated delivery of ARSA to the CNS. | Shenzhen Geno-Immune Medical Institute | NULL | Recruiting | 1 Month | N/A | All | 10 | N/A | China |