Study of the Pharmacokinetics and Safety of Recombinant Factor VIIa Fusion Protein (rVIIa-FP, CSL689) in Patients With Congenital Factor VII Deficiency

CSL Behring

Status and phase: Completed; Phase 1

Conditions: Congenital Coagulation Factor VII Deficiency

Treatments: Biological: CSL689; Biological: Eptacog alfa (activated) or pdFVII

Study type: Interventional

Funder types: Industry

Identifiers

NCT02470871

CSL689_1002

2014-002982-32 (EudraCT Number)

Details and patient eligibility

About

The purpose of this study is to investigate the pharmacokinetics (PK) and safety of rVIIa-FP (CSL689) in a total of 10 to 16 male or female adults with inherited coagulation factor VII (FVII) deficiency. Subjects will receive a single dose of their routine FVII replacement product (ie, either recombinant activated coagulation FVII [rFVIIa, eptacog alfa (activated)] or plasma-derived FVII [pdFVII]) as a comparator, and will then be randomly assigned to a single low dose or a single high dose of the study product CSL689 (8 subjects per CSL689 dose level). Serial blood samples for PK analysis will be taken up to 24 hours after the eptacog alfa (activated) or pdFVII injection, and up to 48 hours after the CSL689 injection. Subject safety will be routinely monitored throughout the study.

Enrollment: 9 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Proven congenital FVII deficiency.
• Age ≥ 18 years.
• FVII level < 2% of normal levels.
• Minimum of 50 previous exposure days to pdFVII (including prothrombin complex concentrates [PCCs]) or rFVIIa.

Exclusion criteria

• History of, or risk factors for, thromboembolic events, including known deep vein thrombosis.
• Inhibitor to FVII or rFVIIa, current or historic.
• Known or suspected hypersensitivity to hamster protein, to CSL689, or to any excipient of CSL689.
• Known or suspected allergy to rFVIIa or hamster protein.
• Major surgery within 1 month before screening.
• Advanced atherosclerotic disease (ie, known history of ischemic heart disease, or ischemic stroke).
• Human immunodeficiency virus (HIV)-positive subjects with cluster of differentiation 4 (CD4)+ lymphocyte count of < 200/µL at screening.
• Use of an investigational agent within 30 days before the study.
• Use of concomitant therapy not permitted during the study (ie, other platelet inhibitors, desmopressin, fibrinolysis inhibitors, except if used as local treatment [eg, for oral bleeds])

Trial design

Primary purpose: Other

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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9 participants in 2 patient groups

Low-dose CSL689

Experimental group

Description:

Single dose of subject's routine FVII replacement therapy (either eptacog alfa \[activated\] \[ie, comparator drug 1\] or pdFVII \[ie, comparator drug 2\]), followed by a single dose of CSL689 at the low dose

Treatment:

Biological: Eptacog alfa (activated) or pdFVII

Biological: CSL689

High-dose CSL689

Experimental group

Description:

Single dose of subject's routine FVII replacement therapy (either eptacog alfa \[activated\] \[ie, comparator drug 1\] or pdFVII \[ie, comparator drug 2\]), followed by a single dose of CSL689 at the high dose.

Treatment:

Biological: Eptacog alfa (activated) or pdFVII

Biological: CSL689