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Completed

NCT Number: NCT02470871

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

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.

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Key information

Conditions

Congenital Coagulation Factor VII Deficiency

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Site Reference 5280023, Njmegen, Netherlands

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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])

Treatment and study plan

Eptacog alfa (activated) or pdFVII

Biological

Comparator Drug 1: Recombinant activated FVII (rFVIIa). Subjects with eptacog alfa (activated) as their routine FVII replacement therapy will receive a single dose of eptacog alfa (activated) in the study.

Comparator Drug 2: Plasma-derived FVII (pdFVII). Subjects with pdFVII as their routine FVII replacement therapy will receive a single injection of pdFVII in the study.

CSL689

Biological

Experimental Drug: Recombinant fusion protein, linking activated FVII with albumin (rVIIa-FP). Subjects will receive a single dose of CSL689 at either a low dose (Arm 1) or a high dose (Arm 2)

Primary outcomes

  1. Terminal half-life of plasma FVIIa activity

    Time frame: Up to 48 hours after CSL689 injection

  2. Maximum observed plasma FVIIa activity

    Time frame: Before injection and at up to 9 time points until 48 hours after injection

  3. Area under the curve (AUC0-t)

    Time frame: Before injection and at up to 9 time points until 48 hours after injection

    Area under plasma FVIIa activity versus time curve from time 0 to last sample with quantifiable activity

Secondary outcomes

  1. Total clearance

    Time frame: Before injection and at up to 9 time points until 48 hours after injection

    Total clearance of plasma FVIIa activity

  2. Volume of distribution of the terminal phase

    Time frame: Before injection and at up to 9 time points until 48 hours after injection

  3. AUC(0-inf)

    Time frame: Before injection and at up to 9 time points until 48 hours after injection

    Area under plasma FVIIa activity versus time curve from time 0 extrapolated to infinity

  4. Incremental recovery

    Time frame: Before injection and at up to 9 time points until 48 hours after injection

    Incremental recovery of plasma FVIIa activity

  5. Time of occurrence of maximum observed plasma FVIIa activity

    Time frame: Before injection and at up to 9 time points until 48 hours after injection

  6. Number of subjects with antibodies against Chinese hamster ovary protein and FVII

    Time frame: Up to 30 days after CSL689 injection

  7. Number of subjects with inhibitors against FVII

    Time frame: Up to 30 days after CSL689 injection

Sponsors and collaborators

Lead sponsor

CSL Behring

Industry

Registry information

Official study title

Multi-center, Randomized, Open-label, Parallel-Arm, Single-dose, Pharmacokinetic Study of rVIIa-FP (CSL689) in Subjects With Congenital Factor VII Deficiency

Important dates

Study start
2015
Primary completion
2016
Study completion
2016
First posted
Jun 12, 2015
Registry last updated
Apr 26, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.