The purpose of the trial is to evaluate the PK, bioavailability, PD, efficacy and safety of MarzAA for on demand treatment and control of bleeding episodes in adult subjects with inherited bleeding disorders.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
19
Single intravenous dose and ascending doses of subcutaneous injection of MarzAA, followed by a fixed dose of MarzAA for the treatment of bleeding episodes
UC Davis Medical Center
Sacramento, California, United States
Comparative MarzAA activity by dose level/stage and confirm the Phase 2 dose
Comparative pharmacokinetics by dose level/stage based on examination of AUX for each of the dose groups in each cohort.
Time frame: Dosing period for each stage in a cohort will be approximately 5 to 11 days
Bleeding episode treatment success
Proportion of bleeding events treated with MarzAA achieving hemostatic efficacy based on a four-point scale according to the Investigator's assessment
Time frame: 24 hours after the first administration of study drug
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University of California -San Francisco
San Francisco, California, United States
University of Colorado Hemophilia and Thrombosis Center
Aurora, Colorado, United States
Rush University
Chicago, Illinois, United States
Children's Hospital of Michigan
Detroit, Michigan, United States
Michigan State University Center for Bleeding Disorders & Clotting Disorders
East Lansing, Michigan, United States
East Carolina University
Greenville, North Carolina, United States
Mazumdar Shaw Medical Centre
Bengaluru, India
St. John's Medical College Hospital
Bengaluru, India
Amrita Institute of Medical Sciences and Research Centre
Kochi, India
...and 10 more locations