This study is designed to provide information on the safety, tolerability, pharmacokinetics (PK) and bone biomarker response following multiple BPS804 administration in multiple dosing regimens. This information will permit a comparison of the possible risks and benefits of different dosing regimens of the study drug to enable optimal doses and dose intervals to be tested in subsequent studies.
This study was conducted and previously posted by Novartis. The record was transferred to Ultragenyx in February 2021.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
44
Novartis Investigative Site
Anaheim, California, United States
Novartis Investigative Site
Miami, Florida, United States
Novartis Investigative Site
Miami, Florida, United States
Novartis Investigative Site
Berlin, New Jersey, United States
Change from baseline to month 9 in bone mineral density at the lumbar spine for the individual BPS804 groups and pooled placebo arms.
Time frame: 9 months
The number (percent) of subjects experiencing adverse events or serious adverse events
Time frame: 9 months
Change from baseline during 9 months of serological bone biomarkers for the individual BPS804 groups and pooled placebo arms.
Time frame: 9 months
Characterization of the PK profile of BPS804: area under the plasma concentration-time curve (AUC)
Time frame: 260 days
Characterization of the PK profile of BPS804: time to reach the maximum Characterization of the PK profile of BPS804: maximum plasma concentration (Cmax)
Time frame: 260 days
Characterization of the PK profile: time to reach the maximum concentration (Tmax)
Time frame: 260 days
Characterization of the PK profile of BPS804: half-life (T1/2)
Time frame: 260 days
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Novartis Investigative Site
Duncansville, Pennsylvania, United States