A Phase 1 Safety, Pharmacokinetics And Pharmacodynamics Study Of PF-05280602, A Recombinant Factor VIIa Variant (813d), In Adult Subjects With Hemophilia A Or B

Status:	Completed
Conditions:	Anemia, Hematology
Therapuetic Areas:	Hematology
Healthy:	No
Age Range:	18 - 64
Updated:	4/21/2016
Start Date:	December 2011
End Date:	February 2015

An Ascending Single Dose Study To Evaluate The Safety, Tolerability And Pharmacokinetics/Pharmacodynamics Of Pf-05280602, A Recombinant Factor Viia Variant (813d), In Adult Hemophilia A And B Subjects With Or Without Inhibitors

This study hypothesizes that the study drug, PF-05280602 (at the selected doses) will be
safe to administer to subjects with severe Hemophilia A or B with or without inhibitors and
will demonstrate evidence of hemostatic activity. This is supported by the preclinical
findings in hemophilic animal models.

Inclusion Criteria:

- Male subjects 18 to <65 years old with severe hemophilia A or B with or without
inhibitors to FVIII or FIX.

- Subjects is willing and able to comply with the mandatory washout periods prior to
screening and prior to dosing and through 48 hours post dosing. At screening this
includes a washout of FIX for 96 hours and FVIII for 72 houts. At dosing this
includes a washout of FIX for 96 hours and FVIII and other hemostatic agents for 72
hours through 48 hours post dosing.

- Subjects must agree and commit to using a a highly effective method of birth control
from the time of screening through four weeks after study drug administration.

Exclusion Criteria:

- Presence of a bleeding disorder in addition to hemophilia A or B.

- Regular, concomitant therapy with immunomodulating drugs (eg, intravenous
immunoglobulin, and routine systemic corticosteroids).

- History of coronary artery disease, thrombolic disease or diagnosis of prothrombic
disorder.

We found this trial at

sites

Cincinnati Children´s Hospital Medical Center

Cincinnati, Ohio 45229

from

Cincinnati, OH