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Recombinant Von Willebrand Factor / Recombinant Factor VIII Complex (rVWF:rFVIII): A Phase 1 Study Evaluating the Pharmacokinetics (PK), Safety, and Tolerability in Type 3 Von Willebrand Disease (VWD)


Phase 1
18 Years
60 Years
Not Enrolling
Both
Von Willebrand Disease

Thank you

Trial Information

Recombinant Von Willebrand Factor / Recombinant Factor VIII Complex (rVWF:rFVIII): A Phase 1 Study Evaluating the Pharmacokinetics (PK), Safety, and Tolerability in Type 3 Von Willebrand Disease (VWD)


Inclusion Criteria:



- Subject has voluntarily given written informed consent (before conduct of any
study-related procedures)

- The subject has hereditary type 3 VWD (<= 3 IU/dL VWF:Ag)or severe type 1 or type 2A
VWD (VWF:RCo <= 10% and FVIII:C <20%)

- The subject has a medical history of at least 25 exposure days to VWF/FVIII
coagulation factor concentrates

- The subject has a Karnofsky score >= 70%

- The subject is between 18 to 60 years of age (on the day of signing the informed
consent)

- NOT APPLICABLE IN ITALY: Female subjects of child-bearing potential must have a
negative pregnancy test and agree to practice contraception using a method of proven
reliability from the day of screening until the study completion visit

- APPLICABLE ONLY IN ITALY: Female subjects of child-bearing potential must have a
negative pregnancy test and agree to practice non-hormonal-based contraception using
a method of proven reliability (IUD acceptable) from the day of screening until 96
hours after the last investigational drug infusion

- NOT APPLICABLE IN ITALY: The subject must agree not to be on any therapy
(hormone-based contraception acceptable) interfering with coagulation factor
pharmacokinetics until 96 hours after the last investigational drug infusion

- APPLICABLE ONLY IN ITALY: The subject must agree not to be on any therapy interfering
with coagulation factor pharmacokinetics until 96 hours after the last
investigational drug infusion

Exclusion Criteria:

- The subject has been diagnosed with a hereditary or acquired coagulation disorder
other than VWD (including qualitative and quantitative platelet disorders and/or an
international normalized ratio (INR) > 1.4)

- The subject has been diagnosed with an ADAMTS13 deficiency with less than 10%
ADAMTS13 activity

- The subject has a history or presence of VWF inhibitor

- The subject has a history or presence of FVIII inhibitor with a titer >= 0.4 BU (by
Nijmegen assay) or >= 0.6 BU (by Bethesda assay)

- The subject has a known hypersensitivity to mouse or hamster proteins

- The subject has a medical history of immunological disorders, excluding seasonal
allergic rhinitis/conjunctivitis, food allergies or animal allergies

- The subject has a medical history of a thromboembolic event

- The subject is HIV positive with an absolute CD4 count < 200/mm3

- The subject has been diagnosed with cardiovascular disease (New York Heart
Association (NYHA) classes 1-4)

- The subject has been diagnosed with insulin-dependent diabetes mellitus

- The subject has an acute illness (e.g. influenza, flu-like syndrome, allergic
rhinitis/conjunctivitis)

- The subject has been diagnosed with liver disease, as evidenced by, but not limited
to, any of the following: serum ALT three times the upper limit of normal,
hypoalbuminemia, portal vein hypertension (e.g. presence of otherwise unexplained
splenomegaly, history of esophageal varices)

- The subject has been diagnosed with renal disease, with a serum creatinine level >= 2
mg/dL

- In the judgment of the investigator, the subject has another clinically significant
concomitant disease (e.g. uncontrolled hypertension, diabetes type II) that may pose
additional risks for the subject

- The subject has been treated with an immunomodulatory drug, excluding topical
treatment (e.g. ointments, nasal sprays) within 30 days before enrollment

- The subject has been treated with drugs known to induce thrombotic thrombocytopenic
purpura (TTP) (e.g. Adenosine diphosphate (ADP) receptor inhibitors (Clopidogrel,
Ticlopidine)) within 60 days before enrollment

- The subject is receiving or anticipates receiving another investigational and/or
interventional drug within 30 days before enrollment

- The subject is a lactating female

- The subject has a history of drug or alcohol abuse within the last 5 years

- The subject has a progressive fatal disease and/or life expectancy of less than 3
months

- The subject is identified by the investigator as being unable or unwilling to
cooperate with study procedures

- The subject suffers from a mental condition rendering him/her unable to understand
the nature, scope and possible consequences of the study and/or evidence of an
uncooperative attitude

- Subject is in prison or compulsory detention by regulatory and/or juridical order

Type of Study:

Interventional

Study Design:

Allocation: Randomized, Endpoint Classification: Safety Study, Intervention Model: Crossover Assignment, Masking: Single Blind (Subject)

Outcome Measure:

To demonstrate the immediate tolerability and safety after single-dose injections of rVWF:rFVIII at various doses

Outcome Time Frame:

Up to 30 days after the last investigational product infusion

Safety Issue:

Yes

Principal Investigator

Tobias Suiter, MD

Investigator Role:

Study Director

Investigator Affiliation:

Baxter Healthcare Corporation

Authority:

United States: Food and Drug Administration

Study ID:

070701

NCT ID:

NCT00816660

Start Date:

December 2008

Completion Date:

October 2010

Related Keywords:

  • Von Willebrand Disease
  • Hemophilia A
  • Von Willebrand Diseases
  • Von Willebrand Disease, Type 3

Name

Location

Rush University Medical CenterChicago, Illinois  60612-3824
Rochester General HospitalRochester, New York  14621
Brown Cancer CenterLouisville, Kentucky  40202
University of TexasHouston, Texas  77225
Comprehensive Center for Bleeding DisordersMilwaukee, Wisconsin  53201
Emory University School of Medicine, Dept. of PediatricsAtlanta, Georgia  30092
Indiana Hemophilia and Thrombosis CenterIndianapolis, Indiana  46260
University of Kentucky Hemophilia Treatment CenterLexington, Kentucky  40536-0284
Brigham & Women´s Hospital, Hematology DivisionBoston, Massachusetts  02115
Hemophilia Center of Western PAPittsburgh, Pennsylvania  15213-4306