This study in an open label study that is conducted in many centers around the world to investigate whether Wilate is safe and works in patients that need surgery and have von Willebrand disease.

• Register: EUCTR
• Last refreshed on: 28 August 2014
• Main ID: EUCTR2010-021162-30-BG
• Date of registration: 07/12/2011
• Prospective Registration: Yes
• Primary sponsor: Octapharma AG
• Public title: This study in an open label study that is conducted in many centers around the world to investigate whether Wilate is safe and works in patients that need surgery and have von Willebrand disease.
• Scientific title: Prospective, Open-Label, Multi-Center, Phase III Clinical Study To Investigate The Efficacy And Safety Of Human Factor VWF/VIII Concentrate (Wilate®) In Subjects With Inherited Von Willebrand Disease (VWD) Who Undergo Surgical Procedures.
• Date of first enrolment: 20/12/2011
• Target sample size: 41
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2010-021162-30
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no Number of treatment arms in the trial: 1

Countries of recruitment

Bulgaria, India, Italy, Russian Federation, Turkey, United States

Contacts

• Name: Sigurd Knaub
• Affiliation: Octapharma AG

• Name: Sigurd Knaub
• Affiliation: Octapharma AG

Key inclusion & exclusion criteria

Inclusion criteria:
1. Male and female subjects who are at least 6 years of age.
2.Diagnosed with congenital VWD (any type) where VWF:RCo is below 40% at screening or the subject has a diagnosis of Type 1, 2 or 3 VWD and a history of VWF:RCo below 40% documented in their medical notes at enrolment.
3.Require therapy with a VWF product to treat any potential surgical procedure.
4. Negative for anti-human immunodeficiency virus (HIV); if positive, viral load <200 particles/µL or <400,000 copies/mL and CD4+ count > 200/µL.
5. The subject and/or their legally acceptable representative understands the nature of the study, gives written informed consent to participate in the study and is willing and able to comply with the protocol.
Are the trial subjects under 18? yes
Number of subjects for this age range: 3
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 14

Exclusion criteria:
1. Known coagulation disorder other than congenital VWD.
2. Any VWF containing product administered within 3 days prior to the screening visit.
3. Any subject where it is planned to infuse the investigational product via continuous infusion.
4. Have a known history of, or are suspected to have VWF or FVIII inhibitors.
5. Emergency surgery or any surgery with a degree of urgency not permitting completion of baseline assessment required by the study protocol.
6. Suffering an acute or chronic medical condition, other than VWD, which may in the opinion of the Investigator affect the conduct of the study.
7. Subjects with active hepatic disease (alanine aminotransferase [ALT] or aspartate aminotransferase [AST] levels >5 times the upper limit of normal)8. Have a known or suspected hypersensitivity or previous evidence of severe side effects to Wilate® or other VWF/FVIII concentrates.
9. Subjects receiving immune-modulating drugs (other than anti-retroviral chemotherapy) such as alpha-interferon, prednisone (equivalent to >10 mg/day), or similar drugs at study start.
10. Pregnant women within the first 20 weeks of gestation.
11. Subjects having evidence or a history (within the previous 12 months) of abuse of any drug substance, licit or illicit.
12. Participation in another interventional clinical study currently or during the past 4 weeks.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Surgery in Inherited Von Willebrand Disease (VWD)
MedDRA version: 14.1 Level: PT Classification code 10047715 Term: Von Willebrand's disease System Organ Class: 10010331 - Congenital, familial and genetic disorders

Intervention(s)

Product Name: WILATE 500 I.U.
Product Code: WILATE 500 I.U.
Pharmaceutical Form: Lyophilisate for solution for injection
Current Sponsor code: Wilate
Other descriptive name: Human von Willebrand factor (VWF) and coagulation factor VIII (FVIII)
Concentration unit: IU international unit(s)
Concentration type: equal
Concentration number: 500-

Product Name: WILATE 1000 I.U.
Product Code: WILATE 1000 I.U.
Pharmaceutical Form: Lyophilisate for solution for injection
Current Sponsor code: Wilate
Other descriptive name: Human von Willebrand factor (VWF) and coagulation factor VIII (FVIII)
Concentration unit: IU international unit(s)
Concentration type: equal
Concentration number: 1000-

Primary Outcome(s)

Main Objective: The primary objective of the study is to evaluate the overall hemostatic efficacy of Wilate® in preventing excessive intra- and post-operative bleeding in pediatric and adult subjects with VWD who require a Von Willebrand Factor (VWF) product and undergo a surgical procedure.

Timepoint(s) of evaluation of this end point: Overall hemostatic efficacy of Wilate® in the treatment in VWD subjects who undergo a surgical procedure based on both the intra-operative assessment of the surgeon at the end of the surgical procedure (last suture) and the assessment by the Investigator covering the post-surgical period from the end of the procedure to 24 hours following the last infusion.

Primary end point(s): Overall hemostatic efficacy (success or failure) of Wilate® in the treatment of VWD subjects who undergo a surgical procedure, based on both the intra-operative assessment of the surgeon and the assessment by the Investigator covering the post-surgical period from the end of the procedure to 24 hours following the last infusion, both of which use a 4-point ordinal hemostatic efficacy scale. The overall efficacy is assessed according to a pre-defined algorithm.

Secondary Objective: • To evaluate the intra- and post-operative surgical hemostatic efficacy of Wilate® in preventing excessive bleeding in pediatric and adult subjects with VWD who require a VWF product and undergo a surgical procedure.
• To assess the safety of Wilate® used in VWD subjects who undergo surgical procedures.

• To document the capability of Wilate® to normalize the coagulation defect in VWD as demonstrated by an increase of the plasma activity of von Willebrand Factor Ristocetin cofactor (VWF:RCo) and Factor VIII:Coagulant (FVIII:C).

• To analyze the actual dosage and duration of treatment in surgical procedures.

Secondary Outcome(s)

Secondary end point(s): • Assessment of intra-operative hemostatic efficacy according to 4-point ordinal efficacy scales.
• Assessment of post-operative hemostatic efficacy according to 4-point ordinal efficacy scales.
• Documentation of the actual dosage and duration of treatment during surgical procedures.
• Measurement of VWF:RCo and FVIII:C plasma activity during treatment.
• The nature and incidence of adverse events (AEs).
• Assessment of the in vivo recovery (IVR) of VWF:RCo, VWF:Ag, and FVIII:C.

Timepoint(s) of evaluation of this end point: • The intra-operative efficacy of Wilate® during the surgical procedures will be assessed by a 4-point ordinal efficacy scale by the surgeon at the end of the surgical procedure (last suture).
• Documentation of the actual dosage and duration of treatment during surgical procedures will be assessed at the end of the surgical procedure (last suture).
• Measurement of VWF:RCo and FVIII:C plasma activity during treatment will be assessed during full course of treatment
• The nature and incidence of adverse events (AE) will be assessed at the end of the study
• Assessment of the in vivo recovery (IVR) of VWF:RCo, VWF:Ag, and FVIII will be calculated at baseline from samples obtained pre infusion, 0.5, and 1 hour post-infusion.

Secondary ID(s)

Wil-24

Source(s) of Monetary Support

Octapharma AG