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 | NCT05682144 (ClinicalTrials.gov) | January 15, 2023 | 12/12/2022 | ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I | A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Adult Patients With Mucopolysaccharidosis Type I Hurler-Scheie and Scheie | Mucopolysaccharidosis IH/S;Mucopolysaccharidosis IS | Biological: Autologous Plasmablasts (B cells) | Immusoft Corporation | NULL | Not yet recruiting | 18 Years | N/A | All | 2 | Phase 1 | United States |