VON WILLEBRAND DISEASE PROPHYLAXIS NETWORK (vWD PN) The VWD International Prophylaxis (VIP) Study

Phase 4

Recruiting

Conditions: Von Willebrand disease
10064477

Registration Number: NL-OMON31532

Lead Sponsor: Von Willebrand disease prophylaxis network

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Recruiting

Sex: Not specified

Target Recruitment: 10

Inclusion Criteria

A. Type 1: eligible for participation if
1. < or = 20% RCo and/or < or = 20% FVIII; and
2. DDAVP non-responsive, defined as occurrence of bleeding episodes not responding satisfactorily to desmopressin, or deemed non-responsive a priori by the investigator; and
3. Bleeding indication criteria are met (page 9-11 protocol) . ;B. Type 2: eligible for participation if
1. DDAVP non-responsive, defined as occurrence of bleeding episodes not responding satisfactorily to desmopressin, or deemed non-responsive a priori by the investigator; or Type 2B; and
2. Bleeding indication criteria are met (page 9-11 protocol) ;C. Type 3: eligible for participation if
1. Bleeding indication criteria are met (page 9-11 protocol)

Exclusion Criteria

A. They have acquired VWD;B. They have a history of inhibitory antibodies to VWF;C. They are already on prophylaxis, defined as receiving factor infusions at least once per week to prevent or decrease the severity of bleeding with the intention of maintaining this regimen for, on average, 45 or more weeks per year; or are receiving factor infusions on a regular basis to prevent or decrease the severity of menorrhagia.

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Bleeding episodes. The primary outcome for the study is bleeding episodes<br /><br>associated with VWD. Participants will be provided with diaries on which they<br /><br>will prospectively record the date, location, and cause of hemorrhages<br /><br>occurring during the study. Treatment for breakthrough bleeding episodes and<br /><br>prophylactic doses (date, time, dosage) of VWD product will be recorded as<br /><br>well.<br /><br><br /><br>For the menorraghia study the bleeding will be scored using a standardized<br /><br>score (PBAC).<br /><br><br /><br>For the arthroscopy study evaluatieon will be performed by a physical<br /><br>therapist using teh WFH PE scale and a pain scale. Radiologic evaluation will<br /><br>be performed using the Petterson score<br /><br><br /><br>Health related quaity of life will be assessed by the HRQOL-4.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Health economics:<br /><br><br /><br>prospective evaluation of absence of scholl or work<br /><br><br /><br>product usage<br /><br>hospitalizations<br /><br><br /><br>laboratory analysis : inhibitors to VWF<br /><br><br /><br>Safety variables:<br /><br>serious adverse events<br /><br>other adverse events</p><br>