DDrare: Database of Drug Development for Rare Diseases

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.

Drug = ASFOTASE ALFA; Alkaline Phosphatase; Asfotas Alfa; Asfotase Alfa; Asfotase Alfa (ALXN1215); Asfotase alfa; BPS804; Biochemical analysis of different bone markers.; Cyclophosphamide; ENB-0040; Fully human IgG2 lambda monoclonal antibody; Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein; Human recombinant tissue non-specific alkaline phosphatase fusion protein; Methotrexate; Other: High resolution peripheral quantitative computed tomography (HRpQCT); Other: Microindentation; Retrospective Case Only; Strensiq

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		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;Phase 3	Japan

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

JPRN-UMIN000003828

2010/07/01

01/07/2010

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

Phase 2;Phase 3

Japan