A study to evaluate the safety and effectiveness of the Recombinant Von Willebrand Factor administred with or without Advate for subjects with severe von Willebrand disease (a hereditary coagulation abnormality) who will undergo major,minor or oral surgery procedures.

Phase 1

• Conditions: Hereditary severe von Willebrand Disease in adult patients who will undergo major and minor elective surgical procedures; MedDRA version: 17.1Level: LLTClassification code 10055168Term: Von Willebrand's factor deficiencySystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2014-003575-38-ES
• Lead Sponsor: Baxter Innovations GmbH

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2014-12-16
• Last Update Posted: 3 October 2016

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 15

Inclusion Criteria

1. Diagnosis of severe VWD as listed and elective surgical procedure planned:
· VWD with a history of requiring substitution therapy with von Willebrand factor concentrate to control bleeding.
· Type 1 (VWF:RCo < 20 IU/dL) or
· Type 2A (as verified by multimer pattern), Type 2B (as diagnosed by genotype), Type 2N (Factor VIII coagulation activity [FVIII:C] <10% and historically documented genetics), Type 2M or
· Type 3 (von Willebrand factor antigen [VWF:Ag] ? 3 IU/dL)
2. If type 3 VWD (VWF:Ag ? 3 IU/dL), subject has a medical history of at least 20 EDs to VWF/FVIII
coagulation factor concentrates (including cryoprecipitate or fresh frozen plasma).
3. If type 1 or type 2 VWD, subject has a medical history of 5 EDs or a past major surgery requiring
VWF/FVIII coagulation factor concentrates (including cryoprecipitate or fresh frozen plasma).
4. At least 18 years of age
5. If female of childbearing potential, subject presents with a negative pregnancy test
6. If applicable, subject agrees to employ adequate birth control measures for the duration of the study
7. Willing and able to comply with the requirements of the protocol.
Are the trial subjects under 18? no
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 14
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Subjects who meet ANY of the following criteria are not eligible for this study:
1. Diagnosis of pseudo VWD or another hereditary or acquired coagulation disorder (eg qualitative and quantitative platelet disorders or elevated PT/ international normalized ratio [INR] ? 1.4)
2. History or presence of a VWF inhibitor at screening
3. History or presence of a factor VIII (FVIII) inhibitor with a titer ? 0.4 BU (Nijmegen-modified Bethesda assay ) or ? 0.6 BU (by Bethesda assay)
4. Known hypersensitivity to any of the components of the study drugs, such as to mouse or hamster proteins
5. Medical history of immunological disorders, excluding seasonal allergic rhinitis/conjunctivitis, mild asthma, food allergies or animal allergies
6. Medical history of a thromboembolic event
7. HIV positive with an absolute CD4 count ? 200/mm3
8. Platelet count < 100,000/mL
9. Diagnosis of significant liver disease, as evidenced by, but not limited to, any of the following: serum alanine aminotransferase (ALT) 5 times the upper limit of normal; hypoalbuminemia; portal vein hypertension (e.g. presence of otherwise unexplained splenomegaly, history of esophageal varices) or liver cirrhosis classified as Child B or C
10. Diagnosis of renal disease, with a serum creatinine level ? 2 .5mg/dL
11. Subject has been treated with an immunomodulatory drug, excluding topical treatment (e.g. ointments, nasal sprays), within 30 days prior to signing the informed consent
12. Subject is pregnant or lactating at the time informed content is obtained
13. Subject has participated in another clinical study involving an investigational product (IP), other than rVWF with or without ADVATE, or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study. (Eligible patients participating in the rVWF Prophy study (071301) may be enrolled).
14. Progressive fatal disease and/or life expectancy of less than 3 months
15. Subject is identified by the investigator as being unable or unwilling to cooperate with study procedures
16. Subject suffers from a mental condition rendering him/her unable to understand the nature, scope and possible consequences of the study and/or evidence of an uncooperative attitude
17. Subject is in prison or compulsory detention by regulatory and/or juridical order
18. Member of the study team conducting this study or in a dependent relationship with one of the study team members. Dependent relationships include close relatives (i.e., children, partner/spouse, siblings, parents) as well as employees

Study & Design

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