Efficacy, safety and pharmaco-economic assessment of secondary long term prophylaxis with highly purified, standardized, doubly virus inactivated FVIII/VWF concentrates in patients with severe, inherited VWD and frequent bleedings.
- Conditions
- Severe, inherited von Willebrand disease (VWD) and frequent bleedingsMedDRA version: 14.1 Level: PT Classification code 10047715 Term: Von Willebrand's disease System Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2006-001383-23-GB
- Lead Sponsor
- Prof Flora Peyvandi, MD
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 24
1. 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 and a BT of 12 minutes or less after 2 hours from administration of 0,3 mcg/kg of DDAVP
OR
contraindication to DDAVP (namely type 2B VWD patients or other patients who display significant side effects to DDAVP)
2. Frequent, spontaneous bleedings i.e. at least 5 episodes in whatever anatomical site in the last 12 months, 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)
- Haemarthroses (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
3. 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:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range
1. a life expectancy < 1 year
2. presence of allo-antibodies to VWF or FVIII
3. acquired Von Willebrand’s syndrome (AVWS)
4. co-morbidity with other haemorrhagic diathesis, excluded those linked to VWD as the complication of treatment (for instance thrombocytopenia in Type 2B VWD)
5. 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
6. pregnancy and lactation
7. any known need for invasive procedures or elective surgery scheduled in the following 3 months (recruitment in these cases should be postponed)
8. 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
9. gastrointestinal bleeding due to trauma, invasive diagnostic or surgical procedures
10. concomitant autoimmune anaemia and/or autoimmune thrombocytopenia
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method