Von Willebrand DiseasePhase 1NCT00816660
Pharmacokinetic, Safety and Tolerability Study of Recombinant Von Willebrand Factor / Recombinant Factor VIII Complex in Type 3 Von Willebrand Disease
A Phase 1 study testing Recombinant von Willebrand factor : recombinant FVIII (rVWF:rFVIII) and Marketed plasma-derived VWF/FVIII concentrate in people with Von Willebrand Disease, run by Baxalta now part of Shire at 25 sites in 6 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Von Willebrand Disease
- What is being tested?
- Recombinant von Willebrand factor : recombinant FVIII (rVWF:rFVIII) and Marketed plasma-derived VWF/FVIII concentrate
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 32 participants
- Where
- 25 sitesin 6 countries
- Study dates
- 1 Dec 2008to 31 Aug 2010
- 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 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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
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- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
25 sites in 6 countries.
25 sites in 6 countries
United States10 sites
Emory University School of Medicine, Dept. of Pediatrics
Atlanta, Georgia, 30092
Brigham & Women´s Hospital, Hematology Division
Boston, Massachusetts, 02115
Rush University Medical Center
Chicago, Illinois, 60612
University of Texas
Houston, Texas, 77030
Indiana Hemophilia and Thrombosis Center
Indianapolis, Indiana, 46260
University of Kentucky Hemophilia Treatment Center
Lexington, Kentucky, 40536-0284
Brown Cancer Center
Louisville, Kentucky, 40202
Comprehensive Center for Bleeding Disorders
Milwaukee, Wisconsin, 53225-3548
Hemophilia Center of Western PA
Pittsburgh, Pennsylvania, 15213-4306
Rochester General Hospital
Rochester, New York, 14621
Italy6 sites
Azienda Ospedaliero-universitaria "Careggi"
Florence, 50134
Giannia Gaslini Children´s Hospital
Genova, 16147
Ospedale Maggiore di Milano, Centro Emofilia e Trombosi "Angelo Bianchi Bonomi"
Milan, 20122
Ospedale San Giovanni Bosco, Centro Emofilia Divisione di Ematologia
Naples, 80144
University of Padua Medical School
Padua, 35128
Ospedale di Vicenza - U.L.S.S.N.6
Vicenza, 80144
United Kingdom4 sites
West Midlands Region Adult Haemophilia Centre, Queen Elizabeth Hospital
Birmingham, B15 2TT
Imperial College School of Medicine, Hammersmith Hospital
London, W12 0NN
Central Manchester Healthcare NHS Trust, Manchester Haemophilia Comprehensive Care Centre
Manchester, M13 9WL
Royal Cornwall Hospital
Truro, TR1 3LJ
Germany3 sites
Vivantes Klinikum im Friedrichshain
Berlin, 10249
Hannover Medical School - Clinic for Haematology, Haemostaseology, Oncology and Stem Cell Transplantation
Hanover, 30625
Institut für Thrombophilie und Hämostaseologie
Münster, 48143
Austria1 site
General Hospital Vienna (Allgemeines Krankenhaus der Stadt Wien), University Department for Internal Medicine I
Vienna, 1090
About this study
Official title: 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.
- Conditions
- Von Willebrand Disease
- Being tested
- Recombinant von Willebrand factor : recombinant FVIII (rVWF:rFVIII), Marketed plasma-derived VWF/FVIII concentrate
- Medical subject headings
- von Willebrand Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Recombinant von Willebrand factor : recombinant FVIII (rVWF:rFVIII)Biological
Single dose, dose escalation, various cohorts
Marketed plasma-derived VWF/FVIII concentrateBiological
Cross-over: recombinant FVIII (rVWF:rFVIII) and marketed plasma-derived VWF/FVIII concentrate
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Not described Receives: Biological: Recombinant von Willebrand factor : recombinant FVIII (rVWF:rFVIII) |
| 2 | Active comparator | Not described Receives: Biological: Recombinant von Willebrand factor : recombinant FVIII (rVWF:rFVIII); Biological: Marketed plasma-derived VWF/FVIII concentrate |
What is being measured
The primary outcome is the main question the study is designed to answer.
To demonstrate the immediate tolerability and safety after single-dose injections of rVWF:rFVIII at various doses
Measured: Up to 30 days after the last investigational product infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jan 2009 (estimated)
- Study start1 Dec 2008
- Primary completion31 Aug 2010
- Study completion31 Aug 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Von Willebrand DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Other
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 32 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baxalta now part of Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00816660
- Sponsor's protocol ID
- 070701
- Status last verified
- Apr 2021
- First submitted
- 2 Jan 2009
- Last updated
- 3 May 2021
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Mannucci PM, Kempton C, Millar C, Romond E, Shapiro A, Birschmann I, Ragni MV, Gill JC, Yee TT, Klamroth R, Wong WY, Chapman M, Engl W, Turecek PL, Suiter TM, Ewenstein BM; rVWF Ad Hoc Study Group. Pharmacokinetics and safety of a novel recombinant human von Willebrand factor manufactured with a plasma-free method: a prospective clinical trial. Blood. 2013 Aug 1;122(5):648-57. doi: 10.1182/blood-2013-01-479527. Epub 2013 Jun 18.
PubMed 23777763
Study at a glance
From the registry record, last updated by the sponsor on 3 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT00816660
- Acronym / sponsor ID
- 070701
- Conditions
- Von Willebrand Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 32 participants (actual)
- Lead sponsor
- Baxalta now part of ShireIndustry (pharma, biotech, devices)
- Study start
- 1 Dec 2008 (actual)
- Primary completion
- 31 Aug 2010 (actual)
- Study completion
- 31 Aug 2010 (actual)
- First posted
- 5 Jan 2009 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Biological: Recombinant von Willebrand factor : recombinant FVIII (rVWF:rFVIII) |
| 2 | Active comparator | Biological: Recombinant von Willebrand factor : recombinant FVIII (rVWF:rFVIII), Biological: Marketed plasma-derived VWF/FVIII concentrate |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (9 listed)
- 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)
Key exclusion criteria (23 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Recombinant von Willebrand factor : recombinant FVIII (rVWF:rFVIII)Single dose, dose escalation, various cohorts | Biological | 1, 2 |
| Marketed plasma-derived VWF/FVIII concentrateCross-over: recombinant FVIII (rVWF:rFVIII) and marketed plasma-derived VWF/FVIII concentrate | Biological | 2 |
Primary outcome
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 May 2021.
- Derived from this study: PubMed 23777763Mannucci PM, Kempton C, Millar C, Romond E, Shapiro A, Birschmann I, Ragni MV, Gill JC, Yee TT, Klamroth R, Wong WY, Chapman M, Engl W, Turecek PL, Suiter TM, Ewenstein BM; rVWF Ad Hoc Study Group. Pharmacokinetics and safety of a novel recombinant human von Willebrand factor manufactured with a plasma-free method: a prospective clinical trial. Blood. 2013 Aug 1;122(5):648-57. doi: 10.1182/blood-2013-01-479527. Epub 2013 Jun 18.
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Von Willebrand Diseases
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
Takeda
25 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Recombinant von Willebrand factor : recombinant FVIII (rVWF:rFVIII) and Marketed plasma-derived VWF/FVIII concentrate in people with Von Willebrand Disease, run by Baxalta now part of Shire at 25 sites in 6 countries.
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 9 inclusion and 23 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 3 May 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 25 sites in United States, Italy, United Kingdom, Germany, Austria and 1 more country. 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?
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). 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 NCT00816660, 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 3 May 2021.