Phase 1 Safety, Pharmacokinetics And Pharmacodynamics Study Of Recombinant Factor VIIa Variant (813d) In Adult Subjects With Hemophilia

C

Catalyst Biosciences

Status and phase

Completed

Phase 1

Conditions

Hemophilia A

Treatments

Biological: PF-05280602

Study type

Interventional

Funder types

Industry

Identifiers

NCT01439971

B3051001

2011-002170-23 (EudraCT Number)

Details and patient eligibility

About

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.

Enrollment

29 patients

Sex

Male

Ages

18 to 64 years old

Volunteers

No Healthy Volunteers

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.

Trial design

Primary purpose

Basic Science

Allocation

Non-Randomized

Interventional model

Sequential Assignment

Masking

None (Open label)

29 participants in 5 patient groups

1

Experimental group

Treatment:

Biological: PF-05280602

2

Experimental group

Treatment:

Biological: PF-05280602

3

Experimental group

Treatment:

Biological: PF-05280602

4

Experimental group

Treatment:

Biological: PF-05280602

5

Experimental group

Treatment:

Biological: PF-05280602

Trial contacts and locations

23

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