172. 低ホスファターゼ症 Hypophosphatasia Clinical trials / Disease details
臨床試験数 : 34 / 薬物数 : 17 - (DrugBank : 4) / 標的遺伝子数 : 3 - 標的パスウェイ数 : 6
| 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 | JPRN-UMIN000003828 | 2010/07/01 | 01/07/2010 | Allogeneic Bone Marrow and Mesenchymal Stem Cell Transplantation for patients with severe Hypophosphatasia | Allogeneic Bone Marrow and Mesenchymal Stem Cell Transplantation for patients with severe Hypophosphatasia - Allogeneic Bone Marrow and Mesenchymal Stem Cell Transplantation for patients with severe Hypophosphatasia | Hypophosphatasia | 1. BM harvest 1)Agreement about Harvesting BM 2)Harvest volume; 100-120ml 3)Anesthetic protocol; discuss by donor 2.BMT and MSCT First, we perform allogeneic BMT, and then allogeneic MSCT from same BM donor. Only MSCT is repeatedly performed if the patient deteriorates symptoms after BMT and MSCT. 1)BMT -Timing of BMT as soon as possible after diagnosis -Conditioning Buslfan 0.9-1mg/kg/dose every 6 hours, 4days Cyclophosphamide 50mg/kg/dose, 4days Antithymocyte globulin 1.25mg/kg/dose, 4days -Prophylaxis of GVHD Methotrexate 10-15mg/m2/dose, 4days Taclolimus, 0.02-0.04mg/kg/day, about 6 months 2)MSCT -Cultured-Expansion of MSCs from BM -Preservation of MSCs -Timing of MSCT About 14 to 21 days After BMT -Administration route and volume More than 106/kg is intravenously injected. -Administration of immunosuppressant drugs -Taclolimus 0.02-0.04mg/kg/day, about 6 months 3. Examination period From July 1, 2010 to March 31, 2013 | Shimane University School of Medicine | National Institute of Advanced Industrial Science and Technology | Complete: follow-up complete | Not applicable | 6months-old | Male and Female | 5 | Phase 2,3 | Japan |