This is an open-label, multicenter, within-participant dose-escalation study examining up to 3 dose levels of DISC-3405 and will assess the safety, tolerability, PK, and PD of DISC 3405 in participants with sickle cell disease.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
24
DISC-3405 is administered subcutaneously.
University of Alabama at Birmingham
Birmingham, Alabama, United States
RECRUITINGEmory University
Atlanta, Georgia, United States
RECRUITINGSafety and tolerability of DISC-3405 administration in participants with SCD
Proportion of participants with treatment-emergent adverse events (TEAEs), changes in vital signs, changes in physical examinations, changes in electrocardiograms (ECGs), and changes in clinical laboratory results
Time frame: Up to 36 weeks
Pharmacodynamic (PD) properties of DISC-3405 in participants with SCD by change from baseline for hemoglobin (Hgb)
Time frame: Up to 36 weeks
Pharmacodynamic (PD) properties of DISC-3405 in participants with SCD by change from baseline for hematocrit (HCT)
Time frame: Up to 36 weeks
Pharmacodynamic (PD) properties of DISC-3405 in participants with SCD by change from baseline for reticulocyte count
Time frame: Up to 36 weeks
Pharmacodynamic (PD) properties of DISC-3405 in participants with SCD by change from baseline for Red Blood Cell Count (RBC)
Time frame: Up to 36 weeks
Pharmacodynamic (PD) properties of DISC-3405 in participants with SCD by change from baseline for Lactate Dehydrogenase (LDH)
Time frame: Up to 36 weeks
Pharmacodynamic (PD) properties of DISC-3405 in participants with SCD by change from baseline for bilirubin (direct and indirect)
Time frame: Up to 36 weeks
Area under the plasma concentration versus time curve (AUCt,) following the first dose
Time frame: Up to 36 weeks
Area under the plasma concentration versus time curve extrapolated to infinity (AUC∞) following the first dose
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Augusta University
Augusta, Georgia, United States
RECRUITINGUniversity of Illinois Hospital and Health Sciences System
Chicago, Illinois, United States
RECRUITINGInnovative Hematology - Indiana Hemophilia & Thrombosis Center
Indianapolis, Indiana, United States
RECRUITINGMount Sinai Hospital
New York, New York, United States
RECRUITINGDuke University Medical Center
Durham, North Carolina, United States
RECRUITINGBrody School of Medicine at East Carolina University
Greenville, North Carolina, United States
RECRUITINGCincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
RECRUITINGUniversity of Pittsburgh Medical Center (UPMC)
Pittsburgh, Pennsylvania, United States
RECRUITINGTime frame: Up to 36 weeks
Maximum plasma concentration (Cmax) following the first dose
Time frame: Up to 36 weeks
Time to maximum plasma concentration after drug administration (Tmax)
Time frame: Up to 36 weeks
Elimination half-life (T1/2el) following the first dose
Time frame: Up to 36 weeks
Apparent clearance (CL/F) following the first dose
Time frame: Up to 36 weeks
Pre-dose trough concentration (Ctrough) after repeating doses
Time frame: Up to 36 weeks
Apparent volume of distribution corrected for bioavailability (Vd/F)
Time frame: Up to 36 weeks