172. 低ホスファターゼ症 [臨床試験数:32,薬物数:18(DrugBank:4),標的遺伝子数:3,標的パスウェイ数:6

Searched query = "Hypophosphatasia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

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1 trial found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1JPRN-UMIN000003828
2010/07/0101/07/2010Allogeneic Bone Marrow and Mesenchymal Stem Cell Transplantation for patients with severe Hypophosphatasia Hypophosphatasia1. 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 MedicineNational Institute of Advanced Industrial Science and TechnologyComplete: follow-up completeNot applicable6months-oldMale and Female5Phase 2,3Japan