Clinical trial NCT06651255: Algorithm-based Management to Reduce the Recurrence of GI Bleeding and Severe Epistaxis in Von Willebrand Disease
Official title: Algorithm-based Management to Reduce the Recurrence of Gastrointestinal Bleeding and Severe Epistaxis in Von Willebrand Disease: WILL-MANAGE Trial
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: NA
Conditions: Von Willebrand Disease (VWD)
Keywords: Von Willebrand disease
gastrointestinal bleeding
angiodysplasia
epistaxis
Lead sponsor: University Hospital, Lille
Sponsor class: OTHER
First posted: 2024-10-21
Enrollment: 66
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: WILL MANAGE is a prospective multicenter controlled open label randomized trial comparing an algorithm-based multidisciplinary management to a standard of care to reduce the incidence of bleeding (GI bleeding or severe epistaxis) recurrence in von Willebrand disease.


Eligibility criteria: Inclusion Criteria:

* Males and females adults aged \> 18 years
* With a constitutional von Willebrand Disease (according to the phenotypic criteria of the French Reference Center of von Willebrand Disease)
* With a GI bleeding \[either overt (any hematemesis, any melena or hematochezia with at least a 2g/dL drop in hemoglobin) or occult (iron deficiency anemia with at least 2g/dL drop in hemoglobin)\] with the presence of angiodysplasia or a negative finding on digestive conventional endoscopy OR with a severe epistaxis (requiring red blood cells transfusion or treatment with VWF concentrates)
* Be affiliated to a social security scheme
* Written informed consent obtained

Exclusion Criteria:

* Acquired von Willebrand Disease
* Presence of an inhibitor to VWF or a contra indication to VWF concentrates
* Contra-indication to videocapsule endoscopy
* Refusal of the procedures part of the study
* Pregnant women or breastfeeding
* Short-life expectancy
* Liver cirrhosis Child-Pugh C or diagnosed portal hypertension
* Cancer currently undergoing chemotherapy
* Inability to provide informed consent
* Patient under justice protection

Primary outcomes:
measure: cumulative incidence of bleeding (GI bleeding or severe epistaxis) recurrence during follow-up
timeFrame: Until the visit 7 (72 months ±2months)
Secondary outcomes:
measure: Number of GI bleeding and severe epistaxis recurrence during follow-up
timeFrame: Until the visit 7 (72 months ±2months)
measure: Number of days of hospitalization for GI bleeding or severe epistaxis recurrence during follow-up
timeFrame: Until the visit 7 (72 months ±2months)
measure: Number of transfused red blood cells units, the total amount of iron supplementation (mg) and number of units of VWF concentrates administred (IU/kg) during follow-up
timeFrame: Until the visit 7 (72 months ±2months)
measure: Change in haemoglobin and serum ferritin levels from inclusion to 3-years
timeFrame: from inclusion to 3-years
measure: Change in bleeding score (ISTH BAT) from inclusion to 3-years. The bleeding score will be calculated for the 3-year period before the evaluation.
timeFrame: from inclusion to 3-years
measure: Change in quality of life assessed by a standardized questionnaire validated for VWD (WISH-QoL scores) from inclusion to 3 years
timeFrame: from inclusion to 3-years
Other outcomes:
Locations:
city: Lille
country: France
facility: CHU Lille
state: Nord
city: Amiens
country: France
facility: CHU Amiens
city: Brest
country: France
facility: CHRU Brest
city: Bron
country: France
facility: Hôpital cardiologique Louis Pradel
city: Caen
country: France
facility: CHU Caen
city: Chambery
country: France
facility: CH Chambery
city: Clermont-Ferrand
country: France
facility: CHU Clermont-Ferrand
city: Dijon
country: France
facility: CHU Dijon
city: Le Kremlin-Bicêtre
country: France
facility: AP-HP Hôpital Kremlin Bicetre
city: Marseille
country: France
facility: AP-HM Hôpital Timone
city: Montpellier
country: France
facility: CHU Montpellier
city: Nantes
country: France
facility: CHU Nantes
city: Rennes
country: France
facility: CHU Rennes
city: Rouen
country: France
facility: CHU Rouen
city: Strasbourg
country: France
facility: CHU Strasbourg
city: Toulouse
country: France
facility: CHU Toulouse
city: Tours
country: France
facility: CHU Tours
Study officials:
affiliation: Lille University Hospital
name: Sophie Susen, MD, Professor
role: PRINCIPAL_INVESTIGATOR
References: