NCT00629837

Brief Summary

The primary objective of this study is to determine the pharmacokinetic profile after single administration of two doses of BAY 79-4980 (high and low: 35 IU FVIII/Kg reconstituted in 22 mg and 13 mg of liposomes/Kg, respectively) compared to rFVIII-FS (35 IU/Kg reconstituted in 2.5 mL WFI/1000 IU) in PTPs aged 12 to 60 years with severe hemophilia A.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
27

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Sep 2005

Shorter than P25 for phase_1

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

September 1, 2005

Completed
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2006

Completed
2 years until next milestone

First Submitted

Initial submission to the registry

February 27, 2008

Completed
8 days until next milestone

First Posted

Study publicly available on registry

March 6, 2008

Completed
Last Updated

November 18, 2014

Status Verified

November 1, 2014

First QC Date

February 27, 2008

Last Update Submit

November 17, 2014

Conditions

Hemophilia A

Keywords

FVIIILong actingpk

Outcome Measures

Primary Outcomes (1)

  • To determine the pk profile after single administration of two doses of BAY 79-4980 (high and low: 35 IU FVIII/Kg reconstituted in 22 mg and 13 mg of liposomes/kg, respectively) compared to rFVIII-FS (35 IU/Kg reconstituted in 2.5 mL WFI/1000 IU) in PTPs

    6 weeks

Secondary Outcomes (5)

  • To determine the infusion tolerability of both BAY 79-4980 doses, by evaluation of vital signs and adverse events

    6 weeks

  • To determine the safety of both BAY 79-4980 doses by measuring the effects on laboratory parameters - especially the lipid profile and adverse events

    6 weeks

  • To determine the pk characteristics of liposomes - esp body clearance by measuring the major liposome component 1-palmitoyl-2-oleoyl-sn-glycerol-3-phosphocholine (POPC) and the non-biological liposome component, MPEG 2000 DSPE, as surrogate marker

    6 weeks

  • To determine the activity of rFVIII over time (as determined by thrombin generation assay and the rotation thromboelastography [RoTEG] assay) for both doses of BAY 79-4980 compared to rFVIII-FS

    6 weeks

  • Additional analyses of the number and timing of spontaneous bleeds after each study treatment will be assessed

    6 weeks

Study Arms (4)

Arm 1

EXPERIMENTAL
Biological: Recomb. Factor VIII (Kogenate FS Liposome, BAY79-4980)

Arm 2

EXPERIMENTAL
Biological: Recomb. Factor VIII (Kogenate FS Liposome, BAY79-4980)

Arm 3

ACTIVE COMPARATOR
Biological: Recombinant Factor VIII (Kogenate FS, BAY14-2222)

Arm 4

ACTIVE COMPARATOR
Biological: Recombinant Factor VIII (Kogenate FS, BAY14-2222)

Interventions

Recomb. Factor VIII (Kogenate FS Liposome, BAY79-4980)BIOLOGICAL

Low dose of BAY 79-4980 \[13mg of liposomes/kg\] then cross over to rFVIII-FS (35 IU/kg reconstituted in 2.5 mL WFI / 1000 IU).

Arm 1
Recombinant Factor VIII (Kogenate FS, BAY14-2222)BIOLOGICAL

rFVIII-FS (35 IU/kg reconstituted in 2.5mL WFI /1000 IU) then cross over to low dose of BAY 79-4980 \[13mg of liposomes/kg\]

Arm 3

Eligibility Criteria

Age12 Years - 60 Years
Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Males aged 12 to 60 years
  • Hemophilia A with plasma FVIII level less than 1% (severe hemophilia)
  • No history of FVIII inhibitor antibody formation and no current evidence of inhibitor antibody measured using the Nijmegen modified Bethesda assay (\< 0.6 Nijmegen Bethesda Units \[N.B.U.\]/mL)
  • No signs or symptoms of an acute bleeding episode on the day of infusion
  • Four or more days without treatment with FVIII prior to the day of infusion
  • Subject (or the subject's legal representative) must provide written informed consent and authorization of use and disclosure of Protected Health Information (PHI)
  • Subjects must have been previously treated with FVIII concentrate for a total of at least 200 exposure days, including 20 exposure days in the previous 12 months. Previous treatment can have been with any type of rFVIII or plasma-derived FVIII concentrate

You may not qualify if:

  • Individuals with abnormal renal function (serum creatinine concentrations greater than 1.3 mg/dL) or active hepatic disease (persistent aspartate aminotransferase \[AST\] or alanine aminotransferase \[ALT\] increases to greater than five times the upper limit of normal).
  • Individuals with anemia, as defined by hemoglobin level less than 12 g/dL
  • Any individual with a past history of severe reaction(s) to FVIII products
  • Any individual on interferon treatment or who has received interferon within the previous 3 months
  • Any individual with thrombocytopenia (platelets greater than or equal to 100,000 cells/mm3) or known hematologic/bleeding problems other than hemophilia A
  • Any individual who is receiving or has received other experimental drugs within 3 months prior to study entry
  • Any individual with known dislipidemic disease or actively taking cholesterol lowering drugs for the treatment of hypercholesterolemia or hyperlipidemia (e.g., statins, cholesterol absorption inhibitors, bile acid sequestrants, nicotinic acid or fibrates) or individuals taking anaesthetic drugs
  • Any individual who requires pre-medication for FVIII infusions (e.g., antihistamines)
  • Any individual with high blood pressure (defined as diastolic blood pressure great than or equal to 100 mm/Hg)
  • Any patient who cannot forego FVIII treatment for at least 4 days prior to study entry or between study infusions due to a need for more frequent prophylactic treatment because of a pre-existing medical condition
  • Any patient with known allergy or severe reactions to liposomes or PEG
  • Individuals with any other known disease affecting hemostasis besides hemophilia A
  • Any patient who is not suitable for participation in this trial for any reason, according to the Investigator

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Unknown Facility

Davis, California, 95616, United States

Location

Unknown Facility

Orange, California, 92868-3974, United States

Location

MeSH Terms

Conditions

Hemophilia A

Interventions

Factor VIIIF8 protein, humanBAY 14-2222

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Blood Coagulation FactorsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsProtein PrecursorsBiological Factors

Study Officials

  • Bayer Study Director

    Bayer

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
TRIPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
CROSSOVER
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 27, 2008

First Posted

March 6, 2008

Study Start

September 1, 2005

Study Completion

March 1, 2006

Last Updated

November 18, 2014

Record last verified: 2014-11

Locations

US(2)