Extrait

Primary Objectives •To evaluate safety and tolerability of BPS804 when administered as multiple, dose escalating i.v. infusions in adults with hypophosphatasia (HPP) •To determine the pharmacodynamic (PD) effect and preliminary efficacy of BPS804 when administered as multiple, dose escalating i.v. infusions on the serum bone formation marker bone-specific alkaline phosphatase (BSAP) and serum tissue non-specific alkaline phosphatase (TNSALP)

Critère d'inclusion

• 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)

Liens

• ictrp
• euctr