Von Willebrands DiseaseVWF-phVNCT02466789
Investigating a Von Willebrand Factor (VWF) Functional Screening Assay for Assigning the Phenotypic Variants of Von Willebrand Disease (VWD)
An observational study of Von Willebrands Disease, run by Jonathan Roberts at 1 site in United States.
Trial summary
The purpose of this study is to improve the investigators ability to diagnose von Willebrand Disease (VWD), a common inherited bleeding disorder. This study will look at a new screening blood test used to determine if a person has VWD. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Von Willebrands Disease
- How many people
- 134 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2015to 30 Jun 2022
- Phase
- Observational
- 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 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: New subjects undergoing evaluation for the diagnosis of VWD that are determined to have VWF:ag or VWF:RCo <50 IU/dl and or a VWF:RCo/VWF:ag ratio of <0.7. Also subjects will be included if Type 2N VWD is suspected
Who may be able to join
- New subjects undergoing evaluation for the diagnosis of VWD determined to have a VWF:Ag or VWF: RCo < 50 IU/dl and or a VWF:RCo/VWF:Ag of <0.7. Also subjects will be included if Type 2 N VWD is clinically suspected
Who cannot join
- Those subjects whose lab results do not meet the inclusion criteria
Trial locations and contacts
1 site in 1 country.
United States1 site
Bleeding and Clotting Disorders Institute
Peoria, Illinois, 61615
About this study
The purpose of this study is to improve the investigators ability to diagnose von Willebrand Disease (VWD), a common inherited bleeding disorder. This study will look at a new screening blood test used to determine if a person has VWD. This new screening blood test can determine a diagnosis more rapidly than current blood tests. Also this test could be available at local hospital labs rather than require samples to be sent to bigger more specialized labs.
Read the detailed description
This investigation will be a prospective, multicenter trial to validate the clinical utility of a novel screening assay as a diagnostic screening assay for VWD variants: Type 1C, 2A, 2B, 2M and 2N. Once the subject is enrolled into the study, a minimum of 0.5ml of citrated plasma will be collected and analyzed at the Bleeding and Clotting Disorders Institute Laboratory in Peoria Illinois. Results will be collected: phenotype function profiles will be determined, statistically analyzed and compared to the qualitative data from Blood Center of Wisconsin. Data is expected to correlate as previously shown in prior studies and will confirm the utility of this assay.
- Conditions
- Von Willebrands Disease
- Keywords
- Bleeding Disorder, inherited, Von Willebrands Disease, Hematologic Test, VWF, VWD
- Medical subject headings
- von Willebrand Diseases, Hemostatic Disorders
What is being measured
The primary outcome is the main question the study is designed to answer.
Validate the novel ELISA-based VWF functional screening assay as a diagnostic screening assay to assign VWD phenotypes 1C, 2A, 2B, 2M and 2N.
Measured: planned analysis at 2 years and 4 years with study duration estimated at 4 years
Secondary outcomes (1)
Further development of the VWF functional screening assay through investigating the incorporation of VWF:CB6 (binding to collagen VI)
Measured: study duration 4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jun 2015 (estimated)
- Study startJul 2015
- Primary completion31 Dec 2021
- Study completion30 Jun 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (10)Papers the sponsor has linked to this study.
- Other trials for Von Willebrands 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
- Observational
- Observational model
- Ecologic or community
- Time perspective
- Prospective
- Enrolment
- 134 participants (actual)
Sponsor and investigators
- Lead sponsor
- Jonathan Roberts
- Sponsor type
- University, hospital or other
- Collaborators
- Versiti Blood Health
- Responsible party
- Sponsor-investigator, Jonathan Roberts, Bleeding and Clotting Disorders Institute Peoria, Illinois
- Investigators
- Jonathan Roberts, MD, Principal investigator, Bleeding and Clotting Disorders Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02466789
- Sponsor's protocol ID
- H14-25321
- Acronym
- VWF-phV
- Status last verified
- Aug 2022
- First submitted
- 5 Jun 2015
- Last updated
- 10 Aug 2022
- Results posted
- No
- Data monitoring committee
- No
Publications
Nichols WL, Hultin MB, James AH, Manco-Johnson MJ, Montgomery RR, Ortel TL, Rick ME, Sadler JE, Weinstein M, Yawn BP. von Willebrand disease (VWD): evidence-based diagnosis and management guidelines, the National Heart, Lung, and Blood Institute (NHLBI) Expert Panel report (USA). Haemophilia. 2008 Mar;14(2):171-232. doi: 10.1111/j.1365-2516.2007.01643.x.
PubMed 18315614Rodeghiero F, Castaman G, Dini E. Epidemiological investigation of the prevalence of von Willebrand's disease. Blood. 1987 Feb;69(2):454-9.
PubMed 3492222Werner EJ, Broxson EH, Tucker EL, Giroux DS, Shults J, Abshire TC. Prevalence of von Willebrand disease in children: a multiethnic study. J Pediatr. 1993 Dec;123(6):893-8. doi: 10.1016/s0022-3476(05)80384-1.
PubMed 8229521Werner EJ, Abshire TC, Giroux DS, Tucker EL, Broxson EH. Relative value of diagnostic studies for von Willebrand disease. J Pediatr. 1992 Jul;121(1):34-8. doi: 10.1016/s0022-3476(05)82537-5.
PubMed 1625090Castaman G, Eikenboom JC, Bertina RM, Rodeghiero F. Inconsistency of association between type 1 von Willebrand disease phenotype and genotype in families identified in an epidemiological investigation. Thromb Haemost. 1999 Sep;82(3):1065-70.
PubMed 10494765Rodeghiero F, Castaman G, Tosetto A. von Willebrand factor antigen is less sensitive than ristocetin cofactor for the diagnosis of type I von Willebrand disease--results based on an epidemiological investigation. Thromb Haemost. 1990 Nov 30;64(3):349-52.
PubMed 2096485Bazhan SV, Zhdanov VP, Koshil' OI. [Experience in organizing rehabilitative treatment for infectious patients]. Voen Med Zh. 1991 Jul;(7):35-6. No abstract available. Russian.
PubMed 1835556Castaman G, Tosetto A, Goodeve A, Federici AB, Lethagen S, Budde U, Batlle J, Meyer D, Mazurier C, Goudemand J, Eikenboom J, Schneppenheim R, Ingerslev J, Habart D, Hill F, Peake I, Rodeghiero F. The impact of bleeding history, von Willebrand factor and PFA-100((R)) on the diagnosis of type 1 von Willebrand disease: results from the European study MCMDM-1VWD. Br J Haematol. 2010 Nov;151(3):245-51. doi: 10.1111/j.1365-2141.2010.08333.x. Epub 2010 Aug 25.
PubMed 20738304Federici AB, Mannucci PM, Castaman G, Baronciani L, Bucciarelli P, Canciani MT, Pecci A, Lenting PJ, De Groot PG. Clinical and molecular predictors of thrombocytopenia and risk of bleeding in patients with von Willebrand disease type 2B: a cohort study of 67 patients. Blood. 2009 Jan 15;113(3):526-34. doi: 10.1182/blood-2008-04-152280. Epub 2008 Sep 19.
PubMed 18805962Federici AB, Canciani MT. Clinical and laboratory versus molecular markers for a correct classification of von Willebrand disease. Haematologica. 2009 May;94(5):610-5. doi: 10.3324/haematol.2009.005751.
PubMed 19407316
Study at a glance
From the registry record, last updated by the sponsor on 10 Aug 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Investigating a Von Willebrand Factor (VWF) Functional Screening Assay for Assigning the Phenotypic Variants of Von Willebrand Disease (VWD)
- ClinicalTrials.gov ID
- NCT02466789
- Acronym / sponsor ID
- VWF-phV / H14-25321
- Conditions
- Von Willebrands Disease
- Keywords
- Bleeding Disorder, inherited, Von Willebrands Disease, Hematologic Test, VWF, VWD
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 134 participants (actual)
- Lead sponsor
- Jonathan RobertsUniversity, hospital or other
- Collaborators
- Versiti Blood Health
- Study start
- Jul 2015
- Primary completion
- 31 Dec 2021 (actual)
- Study completion
- 30 Jun 2022 (actual)
- First posted
- 9 Jun 2015 (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 purpose of this study is to improve the investigators ability to diagnose von Willebrand Disease (VWD), a common inherited bleeding disorder. This study will look at a new screening blood test used to determine if a person has VWD. This new screening blood test can determine a diagnosis more rapidly than current blood tests. Also this test could be available at local hospital labs rather than require samples to be sent to bigger more specialized labs.
Detailed description
This investigation will be a prospective, multicenter trial to validate the clinical utility of a novel screening assay as a diagnostic screening assay for VWD variants: Type 1C, 2A, 2B, 2M and 2N. Once the subject is enrolled into the study, a minimum of 0.5ml of citrated plasma will be collected and analyzed at the Bleeding and Clotting Disorders Institute Laboratory in Peoria Illinois. Results will be collected: phenotype function profiles will be determined, statistically analyzed and compared to the qualitative data from Blood Center of Wisconsin. Data is expected to correlate as previously shown in prior studies and will confirm the utility of this assay.
Study design
- Observational model
- Ecologic or community
- Time perspective
- Prospective
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- New subjects undergoing evaluation for the diagnosis of VWD that are determined to have VWF:ag or VWF:RCo <50 IU/dl and or a VWF:RCo/VWF:ag ratio of <0.7. Also subjects will be included if Type 2N VWD is suspected
Key inclusion criteria (1 listed)
- New subjects undergoing evaluation for the diagnosis of VWD determined to have a VWF:Ag or VWF: RCo < 50 IU/dl and or a VWF:RCo/VWF:Ag of <0.7. Also subjects will be included if Type 2 N VWD is clinically suspected
Key exclusion criteria (1 listed)
- Those subjects whose lab results do not meet the inclusion criteria
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Validate the novel ELISA-based VWF functional screening assay as a diagnostic screening assay to assign VWD phenotypes 1C, 2A, 2B, 2M and 2N.
Time frame: planned analysis at 2 years and 4 years with study duration estimated at 4 years
1 secondary outcome
Further development of the VWF functional screening assay through investigating the incorporation of VWF:CB6 (binding to collagen VI)
Time frame: study duration 4 years
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 10 Aug 2022.
- Background: PubMed 18315614Nichols WL, Hultin MB, James AH, Manco-Johnson MJ, Montgomery RR, Ortel TL, Rick ME, Sadler JE, Weinstein M, Yawn BP. von Willebrand disease (VWD): evidence-based diagnosis and management guidelines, the National Heart, Lung, and Blood Institute (NHLBI) Expert Panel report (USA). Haemophilia. 2008 Mar;14(2):171-232. doi: 10.1111/j.1365-2516.2007.01643.x.
- Background: PubMed 3492222Rodeghiero F, Castaman G, Dini E. Epidemiological investigation of the prevalence of von Willebrand's disease. Blood. 1987 Feb;69(2):454-9.
- Background: PubMed 8229521Werner EJ, Broxson EH, Tucker EL, Giroux DS, Shults J, Abshire TC. Prevalence of von Willebrand disease in children: a multiethnic study. J Pediatr. 1993 Dec;123(6):893-8. doi: 10.1016/s0022-3476(05)80384-1.
- Background: PubMed 1625090Werner EJ, Abshire TC, Giroux DS, Tucker EL, Broxson EH. Relative value of diagnostic studies for von Willebrand disease. J Pediatr. 1992 Jul;121(1):34-8. doi: 10.1016/s0022-3476(05)82537-5.
- Background: PubMed 10494765Castaman G, Eikenboom JC, Bertina RM, Rodeghiero F. Inconsistency of association between type 1 von Willebrand disease phenotype and genotype in families identified in an epidemiological investigation. Thromb Haemost. 1999 Sep;82(3):1065-70.
- Background: PubMed 2096485Rodeghiero F, Castaman G, Tosetto A. von Willebrand factor antigen is less sensitive than ristocetin cofactor for the diagnosis of type I von Willebrand disease--results based on an epidemiological investigation. Thromb Haemost. 1990 Nov 30;64(3):349-52.
- Background: PubMed 1835556Bazhan SV, Zhdanov VP, Koshil' OI. [Experience in organizing rehabilitative treatment for infectious patients]. Voen Med Zh. 1991 Jul;(7):35-6. No abstract available. Russian.
- Background: PubMed 20738304Castaman G, Tosetto A, Goodeve A, Federici AB, Lethagen S, Budde U, Batlle J, Meyer D, Mazurier C, Goudemand J, Eikenboom J, Schneppenheim R, Ingerslev J, Habart D, Hill F, Peake I, Rodeghiero F. The impact of bleeding history, von Willebrand factor and PFA-100((R)) on the diagnosis of type 1 von Willebrand disease: results from the European study MCMDM-1VWD. Br J Haematol. 2010 Nov;151(3):245-51. doi: 10.1111/j.1365-2141.2010.08333.x. Epub 2010 Aug 25.
- Background: PubMed 18805962Federici AB, Mannucci PM, Castaman G, Baronciani L, Bucciarelli P, Canciani MT, Pecci A, Lenting PJ, De Groot PG. Clinical and molecular predictors of thrombocytopenia and risk of bleeding in patients with von Willebrand disease type 2B: a cohort study of 67 patients. Blood. 2009 Jan 15;113(3):526-34. doi: 10.1182/blood-2008-04-152280. Epub 2008 Sep 19.
- Background: PubMed 19407316Federici AB, Canciani MT. Clinical and laboratory versus molecular markers for a correct classification of von Willebrand disease. Haematologica. 2009 May;94(5):610-5. doi: 10.3324/haematol.2009.005751.
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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.
Jonathan Roberts, MD principal investigator
Bleeding and Clotting Disorders Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Von Willebrands Disease, run by Jonathan Roberts at 1 site 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 1 inclusion and 1 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 10 Aug 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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.
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?
Validate the novel ELISA-based VWF functional screening assay as a diagnostic screening assay to assign VWD phenotypes 1C, 2A, 2B, 2M and 2N. (time frame: planned analysis at 2 years and 4 years with study duration estimated at 4 years). 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 NCT02466789, which also lists 10 linked publications.
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 10 Aug 2022.