Pharmacokinetic, Safety and Tolerability Study of Recombinant Von Willebrand Factor / Recombinant Factor VIII Complex in Type 3 Von Willebrand Disease

April 29, 2021 updated by: Baxalta now part of Shire

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)

The objectives of this study are to evaluate the immediate tolerability and safety of rVWF:rFVIII in subjects with Type 3 Von Willebrand Disease after administration of various dosages of VWF:RCo.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
32

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Austria
      • Vienna, Austria, 1090
        • General Hospital Vienna (Allgemeines Krankenhaus der Stadt Wien), University Department for Internal Medicine I
  • Canada
    • Nova Scotia
      • Halifax, Nova Scotia, Canada, B3H 2YP
        • Q.E.II Health Sciences Centre
  • Germany
      • Berlin, Germany, 10249
        • Vivantes Klinikum Im Friedrichshain
      • Hannover, Germany, 30625
        • Hannover Medical School - Clinic for Haematology, Haemostaseology, Oncology and Stem Cell Transplantation
      • Münster, Germany, 48143
        • Institut für Thrombophilie und Hämostaseologie
  • Italy
      • Firenze, Italy, 50134
        • Azienda Ospedaliero-universitaria "Careggi"
      • Genova, Italy, 16147
        • Giannia Gaslini Children´s Hospital
      • Milan, Italy, 20122
        • Ospedale Maggiore di Milano, Centro Emofilia e Trombosi "Angelo Bianchi Bonomi"
      • Naples, Italy, 80144
        • Ospedale San Giovanni Bosco, Centro Emofilia Divisione di Ematologia
      • Padua, Italy, 35128
        • University of Padua Medical School
      • Vicenza, Italy, 80144
        • Ospedale di Vicenza - U.L.S.S.N.6
  • United Kingdom
      • Birmingham, United Kingdom, B15 2TT
        • West Midlands Region Adult Haemophilia Centre, Queen Elizabeth Hospital
      • London, United Kingdom, W12 0NN
        • Imperial College School of Medicine, Hammersmith Hospital
      • Manchester, United Kingdom, M13 9WL
        • Central Manchester Healthcare NHS Trust, Manchester Haemophilia Comprehensive Care Centre
      • Truro, United Kingdom, TR1 3LJ
        • Royal Cornwall Hospital
  • United States
    • Georgia
      • Atlanta, Georgia, United States, 30092
        • Emory University School of Medicine, Dept. of Pediatrics
    • Illinois
      • Chicago, Illinois, United States, 60612
        • Rush University Medical Center
    • Indiana
      • Indianapolis, Indiana, United States, 46260
        • Indiana Hemophilia and Thrombosis Center
    • Kentucky
      • Lexington, Kentucky, United States, 40536-0284
        • University of Kentucky Hemophilia Treatment Center
      • Louisville, Kentucky, United States, 40202
        • Brown Cancer Center
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Brigham & Women´s Hospital, Hematology Division
    • New York
      • Rochester, New York, United States, 14621
        • Rochester General Hospital
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15213-4306
        • Hemophilia Center of Western PA
    • Texas
      • Houston, Texas, United States, 77030
        • University of Texas
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53225-3548
        • Comprehensive Center for Bleeding Disorders

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years to 60 years (Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Other
  • Allocation: Randomized
  • Interventional Model: Crossover Assignment
  • Masking: Single

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: 1
Biological: Recombinant von Willebrand factor : recombinant FVIII (rVWF:rFVIII)
Single dose, dose escalation, various cohorts
Active Comparator: 2
Biological: Recombinant von Willebrand factor : recombinant FVIII (rVWF:rFVIII)
Single dose, dose escalation, various cohorts
Biological: Marketed plasma-derived VWF/FVIII concentrate
Cross-over: recombinant FVIII (rVWF:rFVIII) and marketed plasma-derived VWF/FVIII concentrate

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
To demonstrate the immediate tolerability and safety after single-dose injections of rVWF:rFVIII at various doses
Time Frame: Up to 30 days after the last investigational product infusion
Up to 30 days after the last investigational product infusion

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Baxalta now part of Shire

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
December 1, 2008

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
August 31, 2010

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
August 31, 2010

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
January 2, 2009

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 2, 2009

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
January 5, 2009

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
May 3, 2021

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 29, 2021

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 070701

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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Drug Interventions

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

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