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Low Von Willebrand in Ireland Cohort Study (LOVIC)

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ClinicalTrials.gov Identifier: NCT03167320
Recruitment Status : Recruiting
First Posted : May 25, 2017
Last Update Posted : May 25, 2017
Sponsor:
Information provided by (Responsible Party):
James O'Donnell, St. James's Hospital, Ireland

Brief 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.

Condition or disease
Von Willebrand Factor, Deficiency

Detailed Description:

All patients with bleeding disorders in Ireland are registered on a national bleeding disorder database and attend the National Centre for Hereditary Coagulation Disorders in St. James's Hospital, Dublin, Ireland 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.


Study Type : Observational
Estimated Enrollment : 200 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Low Von Willebrand in Ireland Cohort Study
Study Start Date : October 2014
Estimated Primary Completion Date : October 2018
Estimated Study Completion Date : October 2019

Resource links provided by the National Library of Medicine


Group/Cohort
LOVIC
Irish patients with low Von Willebrand levels will be have both venous blood sampling and a bleeding score administered at study entry. A DDAVP (1-desamino-8-D-arginine vasopressin) fall off study was organised for those patients in the cohort with no previous fall offs available and no contraindications to DDAVP.



Primary Outcome Measures :
  1. Number of Irish patients with Low Von Willebrand Factor with abnormal bleeding scores [ Time Frame: at enrolment ]
    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.


Secondary Outcome Measures :
  1. The number of patients with Low VWF with abnormal plasma VWF clearance [ Time Frame: 2 years ]
    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. The rate of response to DDAVP in Irish patients with low Von Willebrand factor levels [ Time Frame: 3 years ]
    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. The number of patients with Low VWF with reduced plasma VWF synthesis [ Time Frame: 3 years ]
    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.


Biospecimen Retention:   Samples With DNA
Peripheral venous blood samples will be taken at study enrolment and stored for further analysis, including samples for DNA analysis.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Irish adults >18 years of age with low Von Willebrand Factor (VWF) levels defined as two lowest VWF levels (VWF Antigen and/or VWF Ristocetin cofactor activity and/or VWF Collagen Binding) >30 IU/dL <50 IU/dL.
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03167320


Contacts
Contact: Michelle Lavin, FRCPath +35314162141 nchcd@stjames.ie

Locations
Ireland
St. James's Hospital Recruiting
Dublin, Ireland, D8
Contact: Michelle Lavin, FRCPath    +35314162141    nchcd@stjames.ie   
Sub-Investigator: Niamh M O'Connell, FRCPath,PhD         
Sponsors and Collaborators
St. James's Hospital, Ireland
Investigators
Principal Investigator: James S O'Donnell, MD,PhD St. James's Hospital, Dublin Ireland

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: James O'Donnell, George Gabriel Stokes Professor of Haematology, Director Haemostasis Research Group, St. James's Hospital, Ireland
ClinicalTrials.gov Identifier: NCT03167320     History of Changes
Other Study ID Numbers: LOVIC01
First Posted: May 25, 2017    Key Record Dates
Last Update Posted: May 25, 2017
Last Verified: May 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Additional relevant MeSH terms:
Von Willebrand Diseases
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Blood Platelet Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn