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REAL-LIFE DATA OF CONSTITUTIONAL VON WILLEBRAND DISEASE IN WESTERN FRANCE (HOPSCOTcH-WILL)

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ClinicalTrials.gov Identifier: NCT03875924
Recruitment Status : Not yet recruiting
First Posted : March 15, 2019
Last Update Posted : March 19, 2019
Sponsor:
Collaborators:
CHU de Brest
University Hospital, Angers
CH Le Mans
Rennes University Hospital
Information provided by (Responsible Party):
Nantes University Hospital

Brief Summary:

Von Willebrand disease (VWD) is the most common constitutional bleeding disorder in the world, caused by missing or defective von Willebrand factor (VWF). In France, VWD affects approximatively 7,000 patients.

There are many types of VWD. The severest forms are characterized by the occurrence of extremely serious bleedings, requiring in-stays with clotting factors (CF) treatments in specialized hospital units and/or an ambulatory substitutive therapy; both of them are highly expensive.

In France, Hemostasis Treatment Centers (HTC) have the opportunity to record these kinds of data in a database called NHEMO (Net-Hemostasis = care database for constitutional bleeding disorders). Further ahead, the data can be coded, dumped into and extracted from the research database BERHLINGO and analyzed.

The HOPSCOTcH-WILL study will be a retrospective, non-interventional, multicenter (national) cohort study & will provide an overview of the real-life management of patients with VWD in western France requiring a substitutive treatment with VWF, as well as a description of the characteristics of their hemorrhagic events.

Model : Observationnal, real world evidence study. Time Horizon : 2015-2018. HTC (France): Western University Hospitals (BERHLINGO network) = Nantes University Hospital (promotion), Angers University Hospital, Brest University Hospital, Le Mans Regional Hospital & Rennes University Hospital


Condition or disease Intervention/treatment
Von Willebrand Diseases Other: VWD treatments

  Show Detailed Description

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Study Type : Observational
Estimated Enrollment : 300 participants
Observational Model: Cohort
Time Perspective: Retrospective
Official Title: Multicenter Retrospective Study From 5 Hemostasis Treatment Centers in Western France: Severe Hemorrhagic Treated Occurrences' Patterns and Global Substitutive COagulation Factors THerapy in the Inherited Von WILLebrand Disease
Estimated Study Start Date : April 1, 2019
Estimated Primary Completion Date : June 30, 2019
Estimated Study Completion Date : June 30, 2019

Resource links provided by the National Library of Medicine


Group/Cohort Intervention/treatment
VWD BERHLINGO
Patients with constitutional von Willebrand Disease, of any severity, with or without inhibitor followed in one of the investigator centers
Other: VWD treatments
Modalities and types of treatments in VWD patients for severe hemorrhagic events: INN, quantities, duration of treatment




Primary Outcome Measures :
  1. Consumptions of clotting factors/desmopressine for the treatment of severe hemorrhagic events in VWD patients of any severity (in and outpatients care consumptions) [ Time Frame: Every treatment over 48 months ]

Secondary Outcome Measures :
  1. Consumptions of clotting factors/desmopressine for the treatment of severe hemorrhagic events in VWD patients, by type of VWD (in and outpatients care consumptions) [ Time Frame: Every treatment over 48 months ]
  2. Consumptions of clotting factors/desmopressine for the treatment of severe hemorrhagic events in VWD patients, according the history of inhibitor (in and outpatients care consumptions) [ Time Frame: Every treatment over 48 months ]
  3. Consumptions of clotting factors/desmopressine for the treatment of severe hemorrhagic events in VWD patients, by regimen of treatment (in and outpatients care consumptions) [ Time Frame: Every treatment over 48 months ]
  4. Consumptions of clotting factors/desmopressine for the treatment of severe hemorrhagic events in VWD patients, by type of treatment (pdVWF, FVIII, pdVWF/FVIII, desmopressin) [ Time Frame: Every treatment over 48 months ]
  5. Consumptions of clotting factors/desmopressine for the treatment of severe hemorrhagic events in VWD patients, by type of hemorrhage [ Time Frame: Every treatment over 48 months ]
  6. Consumptions of clotting factors/desmopressine for the treatment of severe hemorrhagic events in VWD patients, by age and sex [ Time Frame: Every treatment over 48 months ]
  7. Cost of illness study from the French payer perspective for the von Willebrand Disease [ Time Frame: Over 48 months ]
  8. Estimation of the annual treated bleeding rate (ABR) in VWD patients [ Time Frame: Over 48 months ]


Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

On 16th July 2018, 919 constitutional VWD* patients are included in the NHEMO database for the 5 Western France BERHLINGO HTC = Angers, Brest, Le Mans, Nantes & Rennes. In case of hemorrhagic events:

  • 16.4% (n=151) are likely to be treated with desmopressin only,
  • 20.8% (n=191) with CF only - i.e. von Willebrand Factor (VWF) and/or Factor VIII (FVIII) and /or by-passing agents -,
  • 21.6% (n=199) with desmopressin and/or CF. To be taken into account: 41.2% of patients have no treatment mentioned or are in a phase of evaluation of their modalities of treatment (=desmopressin test to be done or desmopressin treatment to be debated before use, essentially).

It is estimated that out of these potential treated patients, 300 could have been treated with CF at least once during the 4-years period.

*According to the CRMW criteria for constitutional VWD

Criteria

Inclusion Criteria:

  • Constitutional VWD patient, of any severity, with or without inhibitors (according to the CRMW criteria*),
  • Patient included in the research database BERHLINGO
  • Patient treated or not by desmopressin or VWF/FVIII/by-passing agents available on the French market (at baseline)
  • Patient who agrees to participate in the HOPSCOTcH and followed in one of the 5 investigator HTC
  • Patient who are not under guardianship

    • CRMW criteria for constitutional VWD definition:

Severe forms: VWF:Ag and VWF:RCo <5 UI/dL VWD 2A or 2M: VWF:RCo/VWF:Ag < 0.7 and/or ratio VWF:CB/VWF:Ag < 0.7 VWD 2B: unexplained thrombopenia and/or positive RIPA < 0.8 mg/mL (for any value of VWF:RCo/VWF:Ag) VWD 2N: FVIII:C/VWF:Ag < 0.6 and reduced to very reduced VWF:FVIIIB VWF:Ag <30 UI/dL (in the absence of every previous criteria)

Exclusion Criteria:

  • Patients Under guardianship

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): NCT03875924


Contacts
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Contact: Marc Trossaert, MD, PhD +33(0)240087428

Locations
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France
Angers University Hospital Not yet recruiting
Angers, France
Contact: Philippe Beurrier, MD         
Brest University Hospital Not yet recruiting
Brest, France
Contact: Brigitte Pan-Petesch, MD, PhD         
Le Mans Regional Hospital Not yet recruiting
Le Mans, France
Contact: Vincent Cussac, MD         
Nantes University Hospital Not yet recruiting
Nantes, France
Contact: Marc Trossaërt, MD, PhD         
Rennes University Hospital Not yet recruiting
Rennes, France
Contact: Benoit Guillet, MD, PhD         
Sponsors and Collaborators
Nantes University Hospital
CHU de Brest
University Hospital, Angers
CH Le Mans
Rennes University Hospital

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Responsible Party: Nantes University Hospital
ClinicalTrials.gov Identifier: NCT03875924     History of Changes
Other Study ID Numbers: RC19_0075
First Posted: March 15, 2019    Key Record Dates
Last Update Posted: March 19, 2019
Last Verified: March 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

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