Table 2.
Clinical Trials for MPS VII
| Study Identification | Type | Phase | Drug | Clinical Trials |
|---|---|---|---|---|
| UX003-CL201 | Interventional | Phase I/II | UX003 (Vestronidase alfa) | NCT01856218 |
| UX003-CL301 | Interventional | Phase III | UX003 (Vestronidase alfa) | NCT02230566 |
| UX003-CL202 | Interventional | Phase III | UX003 (Vestronidase alfa) | NCT02432144 |
| UX003-CL203 | Interventional | Phase II | UX003 (Vestronidase alfa) | NCT02418455 |
| UX003-CL401 | Observational prospective | NA | NA | |
| 13-606 | Interventional | NA | UX003 (Vestronidase alfa) | NCT02097251 |
| BSLY-06-2018 | Observational prospective | NA | NA | NCT02298699 |
| UX003-EAP | Interventional | UX003 (Vestronidase alfa) | NCT03775174 | |
| U54NS065768 | Observational prospective | NA | NA | NCT01870375 |
| 20-31520 | Interventional | Phase I | Aldurazyme (laronidase), Elaprase (idursulfase), Vimizim (elosulfase alfa), Naglazyme (galsulfase), Mepsevii (vestronidase alfa-vjbk), Lumizyme (alglucosidase alfa), Kanuma (sebelipase alfa) | NCT04532047 |
| MT2013-31 | Interventional | Phase II | Biological: Stem Cell Transplantation Drug: IMD Preparative Regimen (Anti-thymocyte Globulin (ATG), Fludarabine, Busulfan) |
NCT02171104 |
| R01HD073292 | Observational prospective | NA | NA | NCT05368038 |
| MT2008-02 | Interventional | Phase II | Procedure: Stem Cell Transplantation Drugs: Cyclophosphamide, Campath-1H Drug: Busulfan |
NCT00668564 |
| 2009LS088/MT2009-19 | Interventional | Phase II | Procedure: Allogeneic stem cell transplantation Drugs: Campath-1H, Cyclophosphamide, Busulfan, Cyclosporine A, Mycophenolate Mofetil |
NCT01043640 |