Low Von Willebrand in Ireland Cohort Study

Von Willebrand Factor, Deficiency Clinical Trial

— LOVIC  

Official title:

Low Von Willebrand in Ireland Cohort Study

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03167320
• Other study ID #: LOVIC01
• Status: Recruiting
• Start date: October 2014
• Est. completion date: July 2022

Study information

• Verified date: March 2019
• Source: St. James's Hospital, Ireland
• Contact: Michelle Lavin, FRCPath
• Phone: +35314162141
• Email: nchcd[@]stjames.ie
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

The Low Von Willebrand in Ireland Cohort (LoVIC) study focuses on the bleeding phenotype and biological mechanisms underlying low Von Willebrand Factor (VWF) levels.

Description:

All patients with bleeding disorders in Ireland are registered on a national bleeding disorder database and attend the National Coagulation Centre in St. James's Hospital, Dublin, Ireland or the paediatric centre, Our Lady's Children's Hospital Crumlin for annual review. At review eligible patients will be invited to participate in the Low Von Willebrand in Ireland Cohort (LOVIC) study.

Following consent, an extensive bleeding assessment tool will be administered by a coagulation haematologist to all participants from which the International Society of Thrombosis and Haemostasis Bleeding Assessment Tool (ISTH BAT) and the Condensed Molecular and Clinical Markers for the Diagnosis and Management of Type 1 Von Willebrands Disease (MCMDM-1 VWD) scores can be derived. In addition, blood will be drawn for von Willebrand factor (VWF) measurements, VWF propeptide, platelet VWF. Citrated plasma and DNA will be stored for each patient. The relationship between laboratory parameters, (including von Willebrand factor, platelet VWF, FVIII and concomitant coagulation disorders) and the clinical phenotype in patients with low VWF will be studied. We will assess the effect of the laboratory parameters on the severity of bleeding tendency. In the future mutation analysis of the VWF gene will be performed in all participants in the LOVIC study.

Historical patient records and laboratory results will be reviewed and DDAVP (1-desamino-8-D-arginine vasopressin) fall off studies documented where available. If no previous DDAVP fall off study has been performed patients will be invited to attend.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 400
• Est. completion date: July 2022
• Est. primary completion date: July 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Years and older

Eligibility

Inclusion Criteria:

• Two lowest VWF levels (VWF Antigen and/or VWF Ristocetin cofactor activity and/or VWF Collagen Binding) >30 IU/dL <50 IU/dL.

Exclusion Criteria:

• Pregnant patients

• Hospitalised patients/acutely unwell patients

Related Conditions & MeSH terms

• Von Willebrand Diseases
• Von Willebrand Factor, Deficiency

Locations

Country	Name	City	State
Ireland	St. James's Hospital	Dublin

Sponsors (1)

Lead Sponsor	Collaborator
St. James's Hospital, Ireland

Country where clinical trial is conducted

Ireland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Irish patients with Low Von Willebrand Factor with abnormal bleeding scores	The ISTH-BAT and Condensed MCMDM-1 VWD score of all participants will be determined at enrollment using a physician directed questionnaire using only symptoms prior to their diagnosis with Low VWF. This will help elucidate the bleeding phenotype, if any, associated with Low VWF.	at enrolment
Secondary	The number of patients with Low VWF with abnormal plasma VWF clearance	For each individual enrolled the Von Willebrand factor propeptide (VWF:pp, U/dL), Von Willebrand factor antigen (VWF:Ag IU/dL) and Factor VIII:C (FVIII:C IU/dL) levels at enrolment will be determined. From this data the plasma VWF clearance will be ascertained using the plasma VWF:pp/VWF:Ag ratio. In addition, the FVIII:C/VWF:Ag ratio will be calculated to determine the contribution of altered VWF synthesis to Low VWF.	2 years
Secondary	The rate of response to DDAVP in Irish patients with low Von Willebrand factor levels	For each individual with no contraindication a DDAVP trial will be performed with plasma VWF levels taken pre and at 1 and 4 hours post DDAVP. The rate of plasma VWF level fall off for each trial will be determined and the area under the curve (AUC) calculated. Complete response will be defined as a three fold increase from baseline.	3 years
Secondary	The number of patients with Low VWF with reduced plasma VWF synthesis	For each individual enrolled the Von Willebrand factor antigen (VWF:Ag IU/dL) and Factor VIII:C (FVIII:C IU/dL) levels at enrolment will be determined. From this data the plasma FVIII:C/VWF:Ag ratio will be calculated to determine the contribution of altered VWF synthesis to Low VWF.	3 years