The purpose of the current Phase 1/2a single dose, dose-escalating study is to evaluate the acute safety, pharmacokinetics (PK) and pharmacodynamics (PD) properties of MOD-5014 in adult subjects with moderate/severe congenital hemophilia A or B. This will be a single-dose, open label, dose-escalating study. Each dose cohort will be concluded by a safety review, following which escalation to the next dose cohort will be approved.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
27
Long Acting Factor VIIa
City of Hope
Duarte, California, United States
Children's Hospital Los Angeles
Los Angeles, California, United States
Orthopaedic Hemophilia Treatment Center
Los Angeles, California, United States
Composite safety and tolerability parameters as measured by adverse events, electrocardiograms (ECG), laboratory results, vital signs and injection site reactions
To assess the acute safety and tolerability of single intravenous (IV) administration of escalating MOD-5014 doses in hemophilic subjects with and without inhibitors.
Time frame: 30 days
Pharmacokinetic Profile
To evaluate the pharmacokinetic profile (MOD-5014 serum levels; Area Under the Curve (AUC); Cmax; Tmax; T1/2) of single IV administration of escalating MOD-5014 doses in hemophilic subjects with and without inhibitors.
Time frame: 14 days
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University of California Davis Medical Center
Sacramento, California, United States
Rush University Medical Center
Chicago, Illinois, United States
Bleeding & Clotting Disorders Institute
Peoria, Illinois, United States
Indiana Hemophilia & Thrombosis Center
Indianapolis, Indiana, United States
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States
UPMC Presbyterian Shadyside
Pittsburgh, Pennsylvania, United States
UT Southwestern Medical Center
Dallas, Texas, United States
...and 1 more locations