This is a phase 1, first-in-human, multi-center, open-label, single dose cohort study to evaluate the safety and tolerability, pharmacokinetics (PK), exploratory pharmacodynamics (PD), and biomarkers of target engagement of CSL889 following single intravenous (IV) doses in subjects with sickle cell disease (SCD). The study involves sequential dose escalation of cohorts with between-group assessments of key safety and PK variables.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
28
Administered as an IV infusion
University of Illinois Hospital and Health Science Systems
Chicago, Illinois, United States
The Johns Hopkins Hospital
Baltimore, Maryland, United States
Percentage of subjects with treatment-emergent adverse events (TEAEs) by Cohort
Time frame: Up to 32 days after start of CSL889 infusion
Percentage of subjects with TEAEs by severity by Cohort
Time frame: Up to 32 days after start of CSL889 infusion
Percentage of subjects with TEAEs by causality by Cohort
Time frame: Up to 32 days after start of CSL889 infusion
Maximum observed serum concentration (Cmax) of CSL889 by Cohort
Time frame: Up to 32 days after CSL889 infusion
Area under CSL889 serum concentration-time curve (AUC) from time 0 to time t (AUC0-t) by Cohort
Time frame: Up to 32 days after CSL889 infusion
Maximum observed serum concentration (Cmax) of CSL889 by Cohort AUC extrapolated to infinity (AUC0-inf) by CSL889 dose level
Time frame: Up to 32 days after CSL889 infusion
Time of Cmax (tmax) of CSL889 by Cohort
Time frame: Up to 32 days after CSL889 infusion
Terminal half-life (t1/2) of CSL889 by Cohort
Time frame: Up to 32 days after CSL889 infusion
Clearance (CL) of CSL889 by Cohort
Time frame: Up to 32 days after CSL889 infusion
Volume of distribution (Vz) of CSL889 by Cohort
Time frame: Up to 32 days after CSL889 infusion
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University of Minnesota
Minneapolis, Minnesota, United States
Jacobi Medical Center
The Bronx, New York, United States
Brody School of Medicine at East Carolina University
Greenville, North Carolina, United States
Ohio State University
Columbus, Ohio, United States
UPMC Hillman Cancer Center
Pittsburgh, Pennsylvania, United States
Medical University of South Carolina
Charleston, South Carolina, United States
Amsterdam UMC Academic Medical Center
Amsterdam, Netherlands
Erasmus University Medical Center
Rotterdam, Netherlands
...and 5 more locations
Percentage of subjects with detectable antibodies to CSL889 by Cohort
Time frame: Up to 32 days after CSL889 infusion