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NCT Number: NCT01184820

Trial to Evaluate the Pharmacokinetics and Safety Profile of BAY94-9027 Following Single and Multiple Dose Administration

The purpose of this study is to describe the pharmacokinetics (PK) of BAY94-9027(the test drug). Pharmacokinetics means that we will measure how well the study drug corrects the factor VIII levels in your blood and how long it takes for the levels to fall back to your baseline level. The study is also designed to determine if the pharmacokinetics of BAY94-9027 change following repeat dosing over 8 weeks, determine if BAY94-9027 is safe, tolerable, and effective for the treatment of severe hemophilia A and define the appropriate dose of BAY94-9027. Two doses of BAY94-9027 will be studied.

The first 8 subjects enrolled in the study (cohort 1) will receive a low dose (25 IU/kg) and will be treated 2 days a week for 8 weeks (total of 16 doses). The second 8 subjects (cohort 2) will receive a higher dose and will be treated 1 day a week for 8 weeks (total 8 doses). All subjects will receive a single dose of rFVIII (Bayer Kogenate FS) to determine the PK by measuring blood levels for 2 days before they start the study drug BAY94-9027. Factor VIII blood levels for BAY94-9027 will be measured for 7 days after the first and last dose to see describe the PK. Safety & tolerability assessment include vital signs, coagulation and hematological parameter, clinical chemistry, measurement of FVIII inhibitor and polyethylene glycol (PEG) antibodies will be done during the course of the study.

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Key information

Conditions

Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Age range

18 year–65 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 1

Primary location

Davis, California, United States

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Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Male subjects with severe hemophilia A (documented plasma baseline Factor VIII level <1 %)
  • >/= 18 but </= 65 years of age
  • Previously treated with Factor VIII concentrate(s) for a minimum of 150 exposure days (as supported by the subject's medical history)
  • Immunocompetent with a CD4+ lymphocyte count > 400/mm³
  • Signed informed consent from subject

Exclusion criteria

  • Documented history of inhibitor to Factor VIII with a titer >/= 0.6 BU (Biological Unit), by the Nijmegen modified assay. However, subjects with a maximum historical titer of </= 1.0 BU with the classical Bethesda assay on a single measurement but with at least 3 subsequent successive negative results (< 0.6 BU) thereafter are eligible.
  • Unable to stop Factor VIII treatment to complete a minimum 72 hour washout
  • Current evidence of inhibitor to Factor VIII with a titer >/= 0.6 BU, measured at the time of screening
  • Abnormal renal function (serum creatinine > 1.5 times the upper limit of the normal range)
  • Total bilirubin > 1.5 times the upper limit of the normal range
  • Active hepatic disease (alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels > 2 times the upper limit of the normal range)
  • Any concomitant coagulation disorder other than hemophilia A (including lupus anticoagulant)
  • Platelet count < 100,000/mm³
  • Within the last 3 months prior to study entry or during the study will be treated with an immunomodulating drug other than anti-retroviral chemotherapy (e.g., a interferon, steroids, rituximab, etc)
  • Any subject who requires major surgery during study period. Minor procedures may be approved if discussed in advance with the medical expert.

Treatment and study plan

BAY94-9027 + Recombinant Factor VIII (Kogenate FS, BAY14-2222)

Biological

Single dose of Kogenate FS and 16 doses of BAY94-9027 given 2 times a week for 8 weeks. Both drugs to be given intravenously.

BAY94-9027 + Recombinant Factor VIII (Kogenate FS, BAY14-2222))

Biological

Single dose of Kogenate FS and 9 doses of BAY94-9027 given once a week for 8 weeks. Both drugs to be given intravenously.

Primary outcomes

  1. Safety as assessed by measuring immunogenicity

    Time frame: Up to 8 weeks

    Antibodies to FVIII, polyethylene glycol (PEG) and BAY94-9027

  2. Adverse events collection

    Time frame: Up to 8 weeks

  3. Area under the plasma concentration vs time curve from time 0 to the last data point (AUC0-tlast)

    Time frame: Up to 8 weeks

  4. Area under the plasma concentration vs time curve from zero to infinity after single (first) dose (AUC0-inf)

    Time frame: Up to 8 weeks

  5. Maximum drug concentration in plasma (Cmax)

    Time frame: Up to 8 weeks

  6. Half-life associated with the terminal slope (t1/2)

    Time frame: Up to 8 weeks

  7. Time to reach maximum drug concentration in plasma after single (first) dose (Tmax)

    Time frame: Up to 8 weeks

  8. Mean residence time (MRT)

    Time frame: Up to 8 weeks

  9. Total body clearance (CL)

    Time frame: Up to 8 weeks

    Total body clearance of drug from plasma (volume/time) or (volume/time/body weight) or ((volume/time)*(1.73/body surface area)) calculated after intravenous administration

  10. Apparent volume of distribution at steady state (Vss)

    Time frame: Up to 8 weeks

    Based on the chromogenic, one-stage and PEG capture assays

  11. Incremental recovery of FVIII

    Time frame: Up to 8 weeks

    Recovery was assessed using two different assays (chromogenic and one-stage assay)

Sponsors and collaborators

Lead sponsor

Bayer

Industry

Registry information

Official study title

An Open-label Phase 1 Trial to Evaluate the Pharmacokinetics and Safety Profile of BAY94-9027 Following Single and Multiple Dose Administration in Two Cohorts of Previously Treated Male Subjects With Severe Hemophilia A

Important dates

Study start
2010
Primary completion
2011
Study completion
2011
First posted
Aug 19, 2010
Registry last updated
Sep 7, 2018

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.

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