Efficacy, safety and pharmaco-economic assessment ofsecondary long term prophylaxis with highly purified, standardized, doubly virus inactivated FVIII/VWF concentrates in patients with severe, inherited VWDand frequent bleedings

Phase 1

• Conditions: Patients with severe, inherited Von Willebrand’s disease and frequent bleedings; MedDRA version: 16.0Level: PTClassification code 10047715Term: Von Willebrand's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2006-001383-23-DE
• Lead Sponsor: Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2013-05-21
• Last Update Posted: 31 January 2017

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 24

Inclusion Criteria

Patients =6 years old
Documented unresponsiveness to DDAVP. All the patients, excluding type 3 and type 2B, should have been exposed to a documented infusion trial with DDAVP. Responsiveness to DDAVP is defined as an increase in plasma FVIII:C and VWF:RCo levels of at least three fold over baseline and in absolute reaching at least 30 IU/dl for both values
OR
contraindication to DDAVP (namely type 2B VWD patients or other patients who display significant side effects to DDAVP)
-Frequent, spontaneous bleedings i.e. at least 5 episodes in whatever anatomical site in the last 12 months, (unless patients currently under prophylaxis with VWF/FVIII concentrates; see below) severe enough to require treatment with FVIII/VWF concentrates
Prolonged or excess bleeding during menses will be allowed only when:
i.No other medication can control frequent bleeds
ii.Bleeding exceeds 10 consecutive days
OR
Recurrent spontaneous bleedings, severe enough to require treatment with FVIII/VWF concentrates of the following types:
?Epistaxes (at least 5 episodes in the last 12 months)
?Hemarthroses (at least 3 episodes at the same joint in the last 12 months)
?Gastrointestinal bleeding (at least 2 episodes in the last 12 months, with a drop ? 2 g/dl of haemoglobin in 24-48 hours) due to unexplained reason or in association with underlying gastrointestinal angiodysplasia

Patients under long term prophylaxis with VWF/FVIII concentrates at the evaluation can be included in the study (after a wash out period): the selection criteria are the same and must be referred to the 12 months period preceding the start of the current prophylaxis treatment
-Willingness to participate in the study, expressed by signed, written informed consent

Are the trial subjects under 18? yes
Number of subjects for this age range: 2
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 21
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

Exclusion Criteria

-a life expectancy < 1 year
-presence of alloantibodies to VWF or FVIII
-acquired Von Willebrand’s syndrome (AVWS)
-co-morbidity with other haemorrhagic diathesis, excluded those linked to VWD as the complication of treatment (for instance thrombocytopenia in Type 2B VWD)
-advanced liver cirrhosis with:
?cirrhosis related coagulation abnormalities (thrombocytopenia, defined as platelet count < 50.000/mm3, INR >1,7, prolonged prothrombin time > 4 seconds versus normal)
?portal hypertension with history of variceal bleeding
-pregnancy and lactation
-any known need for invasive procedures or elective surgery scheduled in the following 3 months (recruitment in these cases should be postponed)
-proven co-morbidity for other causes of gastrointestinal bleeding not related to the studied disease (as drug induced haemorrhagic gastropathy, upper GI tract ulcers or cancer, or operable conditions, e.g. haemorrhoids) with the exception of concomitant angiodysplasia which meets the inclusion criteria
-gastrointestinal bleeding due to trauma, invasive diagnostic or surgical procedures
-concomitant autoimmune anaemia and/or autoimmune thrombocytopenia

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified