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