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.
This study was conducted and previously posted by Novartis. The record was transferred to Ultragenyx in February 2021.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
8
Mereo BioPharma 3 Ltd Investigative Site
Würzburg, Germany
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
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
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