19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details
臨床試験数 : 899 / 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 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 |
| 2 | NCT04284254 (ClinicalTrials.gov) | December 2022 | 11/11/2019 | MT2018-18: Sleeping Beauty Transposon-Engineered Plasmablasts for Hurler Syndrome Post Allo HSCT | Sleeping Beauty Transposon-Engineered Plasmablasts for Expression and Delivery of Alpha-L-iduronidase in Patients With Hurler Syndrome That Have Previously Undergone Allogeneic Transplantation | Mucopolysaccharidosis Type IH (MPS IH, Hurler Syndrome);Mucopolysaccharidosis Type IH;MPS IH, Hurler Syndrome | Drug: Autologous Plasmablasts | Masonic Cancer Center, University of Minnesota | NULL | Withdrawn | 3 Years | 8 Years | All | 0 | Phase 1/Phase 2 | NULL |