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	EUCTR2010-024013-31-DE (EUCTR)	30/06/2011	07/02/2011	A clinical study to evaluate the safety and preliminary efficacy of BPS804 in adults with hypophosphatasia	An open-label, intra-patient dose-escalation study to evaluate the safety and tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of multiple infusions of BPS804 in adults with hypophosphatasia	Hypophosphatasia (HPP) is a rare genetic metabolic disorder which results in impaired skeletal mineralization, and which is caused by the absence of or by deficient enzymatic activity of the tissue-nonspecific alkaline phosphatase (TNSALP) MedDRA version: 14.1;Level: PT;Classification code 10049933;Term: Hypophosphatasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: BPS804 Product Code: BPS804 Other descriptive name: fully human IgG2 lambda monoclonal antibody	Novartis Pharma Services AG	NULL	Not Recruiting			Female: yes Male: yes	9		Germany

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EUCTR2010-024013-31-DE
(EUCTR)

30/06/2011

07/02/2011

A clinical study to evaluate the safety and preliminary efficacy of BPS804 in adults with hypophosphatasia

An open-label, intra-patient dose-escalation study to evaluate the safety and tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of multiple infusions of BPS804 in adults with hypophosphatasia

Hypophosphatasia (HPP) is a rare genetic metabolic disorder which results in impaired skeletal mineralization, and which is caused by the absence of or by deficient enzymatic activity of the tissue-nonspecific alkaline phosphatase (TNSALP)
MedDRA version: 14.1;Level: PT;Classification code 10049933;Term: Hypophosphatasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Product Name: BPS804
Product Code: BPS804
Other descriptive name: fully human IgG2 lambda monoclonal antibody

Novartis Pharma Services AG

NULL

Not Recruiting

Female: yes
Male: yes

Germany