Clinical Trial: Dose Escalation Study to Evaluate the Safety and Tolerability of Multiple Infusions of BPS804 in Adults With Hypophosphatasia (HPP)

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: 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 <

Brief Summary:

The purpose of the study is to determine tolerability, PK/PD and preliminary efficacy of BPS804 in adult patients with HPP treated with multiple escalating doses of BPS804.

This study will allow a comparison of several doses of the study drug within the first two weeks after administration and after a longer assessment period for the highest dose level to enable selection of dose ranges to be tested in subsequent studies in the HPP indication.


Detailed Summary:
Sponsor: Novartis Pharmaceuticals

Current Primary Outcome:

  • The number (percent) of patients experiencing adverse events or serious adverse events [ Time Frame: 141 days following initial investigational product administration ]
  • Change from baseline in primary serological bone biomarkers [ Time Frame: 141 days following initial investigational product administration ]


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Characterization of the pharmacokinetic profile of BPS804: area under the plasma concentration-time curve (AUC) [ Time Frame: 1, 29 and 141 days following initial investigational product administration ]
  • Characterization of the pharmacokinetic profile of BPS804: observed maximum plasma concentration following drug administration (Cmax) [ Time Frame: 1, 15 and 29 days following initial investigational product administration ]
  • Characterization of the pharmacokinetic profile of BPS804: time to reach the maximum concentration (Tmax) [ Time Frame: 1, 15 and 29 days following initial investigational product administration ]
  • Change from baseline in secondary biomarkers [ Time Frame: 141 days following initial investigational product administration ]
  • The number (percent) of patients developing anti-BPS804 antibodies [ Time Frame: 141 days following initial investigational product administration ]


Original Secondary Outcome: Same as current

Information By: Novartis

Dates:
Date Received: July 29, 2011
Date Started: July 2011
Date Completion:
Last Updated: March 18, 2013
Last Verified: March 2013