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Hemophilia APhase 1NCT01184820

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

A Phase 1 study testing BAY94-9027 + Recombinant Factor VIII (Kogenate FS, BAY14-2222) and BAY94-9027 + Recombinant Factor VIII (Kogenate FS, BAY14-2222)) in people with Hemophilia A, run by Bayer at 4 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

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. Read more about this study

Who is it for?
Ages 18 to 65 years, men only
Conditions the study is for
Hemophilia A
What is being tested?
BAY94-9027 + Recombinant Factor VIII (Kogenate FS, BAY14-2222) and BAY94-9027 + Recombinant Factor VIII (Kogenate FS, BAY14-2222))
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
14 participants
Where
4 sitesUnited States
Study dates
13 Oct 2010to 10 Oct 2011
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 65 years
  • Men only
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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)
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  • 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.

Trial locations and contacts

4 sites in 1 country.

United States4 sites

  • Study site

    Boston, Massachusetts, 02115-6195

  • Study site

    Davis, California, 95616

  • Study site

    Minneapolis, Minnesota, 55455

  • Study site

    Syracuse, New York, 13210

Other trials in United States

About this study

Official 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

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.

Conditions
Hemophilia A
Being tested
BAY94-9027 + Recombinant Factor VIII (Kogenate FS, BAY14-2222), BAY94-9027 + Recombinant Factor VIII (Kogenate FS, BAY14-2222))
Keywords
Pharmacokinetics, Safety
Medical subject headings
Hemophilia A

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • 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.

Study groups in NCT01184820
Study groupRoleWhat they receive
Arm 1ExperimentalNot described
Receives: Biological: BAY94-9027 + Recombinant Factor VIII (Kogenate FS, BAY14-2222)
Arm 2ExperimentalNot described
Receives: Biological: BAY94-9027 + Recombinant Factor VIII (Kogenate FS, BAY14-2222))

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Safety as assessed by measuring immunogenicity

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

    Measured: Up to 8 weeks

  2. Adverse events collection

    Measured: Up to 8 weeks

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

    Measured: Up to 8 weeks

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

    Measured: Up to 8 weeks

  5. Maximum drug concentration in plasma (Cmax)

    Measured: Up to 8 weeks

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

    Measured: Up to 8 weeks

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

    Measured: Up to 8 weeks

  8. Mean residence time (MRT)

    Measured: Up to 8 weeks

  9. Total body clearance (CL)

    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

    Measured: Up to 8 weeks

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

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

    Measured: Up to 8 weeks

  11. Incremental recovery of FVIII

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

    Measured: Up to 8 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted19 Aug 2010 (estimated)
  2. Study start13 Oct 2010
  3. Primary completion10 Oct 2011
  4. Study completion10 Oct 2011

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Basic science
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
14 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01184820
Sponsor's protocol ID
13401
Status last verified
Sep 2018
First submitted
13 Jul 2010
Last updated
7 Sep 2018
Results posted
No
Data monitoring committee
No

Publications

  • Coyle TE, Reding MT, Lin JC, Michaels LA, Shah A, Powell J. Phase I study of BAY 94-9027, a PEGylated B-domain-deleted recombinant factor VIII with an extended half-life, in subjects with hemophilia A. J Thromb Haemost. 2014 Apr;12(4):488-96. doi: 10.1111/jth.12506.

    PubMed 24843882

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing BAY94-9027 + Recombinant Factor VIII (Kogenate FS, BAY14-2222) and BAY94-9027 + Recombinant Factor VIII (Kogenate FS, BAY14-2222)) in people with Hemophilia A, run by Bayer at 4 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 7 Sep 2018. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Safety as assessed by measuring immunogenicity (time frame: Up to 8 weeks). The study lists 11 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01184820, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Sep 2018; AzTrials last refreshed its copy on 8 Nov 2026.

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