- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02466789
Investigating a Von Willebrand Factor (VWF) Functional Screening Assay for Assigning the Phenotypic Variants of Von Willebrand Disease (VWD) (VWF-phV)
August 9, 2022 updated by: Jonathan Roberts
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.
Study Overview
Status
Completed
Conditions
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 Type
Observational
Enrollment (Actual)
134
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
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Illinois
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Peoria, Illinois, United States, 61615
- Bleeding and Clotting Disorders Institute
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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
Ages Eligible for Study
2 years and older (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Sampling Method
Non-Probability Sample
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
Description
Inclusion Criteria:
- 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
Exclusion Criteria:
- Those subjects whose lab results do not meet the inclusion criteria
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
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
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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
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planned analysis at 2 years and 4 years with study duration estimated at 4 years
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Secondary Outcome Measures
Outcome Measure |
Time Frame |
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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
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study duration 4 years
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Collaborators
Investigators
- Principal Investigator: Jonathan Roberts, MD, Bleeding and Clotting Disorders Institute
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
- Federici 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.
- 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.
- Rodeghiero F, Castaman G, Dini E. Epidemiological investigation of the prevalence of von Willebrand's disease. Blood. 1987 Feb;69(2):454-9.
- Werner 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.
- Werner 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.
- Castaman 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.
- Rodeghiero 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.
- Bazhan 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.
- Castaman 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.
- Federici 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.
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
July 1, 2015
Primary Completion (Actual)
December 31, 2021
Study Completion (Actual)
June 30, 2022
Study Registration Dates
First Submitted
June 5, 2015
First Submitted That Met QC Criteria
June 8, 2015
First Posted (Estimate)
June 9, 2015
Study Record Updates
Last Update Posted (Actual)
August 10, 2022
Last Update Submitted That Met QC Criteria
August 9, 2022
Last Verified
August 1, 2022
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- H14-25321
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.
Clinical Trials on Von Willebrands Disease
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TakedaCompletedVon Willebrand Disease (VWD)Canada
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TakedaCompletedVon Willebrand Disease (VWD)Germany