Investigating a Von Willebrand Factor (VWF) Functional Screening Assay for Assigning the Phenotypic Variants of Von Willebrand Disease (VWD)

Von Willebrands Disease Clinical Trial

— VWF-phV  

Official title:

Investigating a Von Willebrand Factor (VWF) Functional Screening Assay for Assigning the Phenotypic Variants of Von Willebrand Disease (VWD)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02466789
• Other study ID #: H14-25321
• Status: Completed
• Start date: July 2015
• Est. completion date: June 30, 2022

Study information

• Verified date: August 2022
• Source: Bleeding and Clotting Disorders Institute Peoria, Illinois
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical 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. 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 134
• Est. completion date: June 30, 2022
• Est. primary completion date: December 31, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Von Willebrand Diseases
• Von Willebrands Disease

Locations

Country	Name	City	State
United States	Bleeding and Clotting Disorders Institute	Peoria	Illinois

Sponsors (2)

Lead Sponsor	Collaborator
Jonathan Roberts	Versiti

Country where clinical trial is conducted

United States, 

References & Publications (10)

Bazhan SV, Zhdanov VP, Koshil' OI. [Experience in organizing rehabilitative treatment for infectious patients]. Voen Med Zh. 1991 Jul;(7):35-6. Russian. — View Citation

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. — View Citation

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(®) 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. — View Citation

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. — View Citation

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. — View Citation

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. — View Citation

Rodeghiero F, Castaman G, Dini E. Epidemiological investigation of the prevalence of von Willebrand's disease. Blood. 1987 Feb;69(2):454-9. — View Citation

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. — View Citation

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. — View Citation

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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Validate the novel ELISA-based VWF functional screening assay as a diagnostic screening assay to assign VWD phenotypes 1C, 2A, 2B, 2M and 2N.		planned analysis at 2 years and 4 years with study duration estimated at 4 years
Secondary	Further development of the VWF functional screening assay through investigating the incorporation of VWF:CB6 (binding to collagen VI)		study duration 4 years