Wilate in Subjects With Von Willebrand Disease Who Undergo Surgery

Phase 3

Completed

Brief Summary: Proportion of surgeries in which the primary endpoint (overall assessment) is classified as success.

Recruitment & Eligibility

Inclusion Criteria

Diagnosed with congenital VWD (von Willebrand Disease)
Require therapy with a VWF (von Willebrand Factor) product to treat any major surgical procedure

Exclusion Criteria

Known coagulation disorder other than VWD
Known history of, or suspected VWF or FVIII inhibitors
Subjects with hepatic liver disease
Known or suspected hypersensitivity or previous evidence of severe side effects to wilate or other VWF/FVIII concentrates
Pregnant women in the first 20 weeks of gestation

Arm && Interventions

Group	Intervention	Description
human VWF/FVIII concentrate	human VWF/FVIII concentrate	-

Primary Outcome Measures

Name	Time	Method
Overall Hemostatic Efficacy (Success or Failure) of Wilate, Based on the Intra-operative Assessment of the Surgeon and the Post-operative Assessment by the Investigator Using a 4-point Ordinal Efficacy Scale.	30 Days	Efficacy of Wilate in surgical procedures was assessed intra-operatively by the surgeon and post-operatively by the investigator. The IDMC additionally conducted an independent adjudication of all hemostatic efficacy results ('secondary adjudication') and adjudicated the surgeons'/investigators' assessments of the intra- and post-operative assessments where there were discrepancies between the two assessments ('primary adjudication'). It was specified in the SAP that the study will be terminated early and success claimed if the two-sided 98.75% confidence interval (CI) for the overall success rate excludes and is greater than 0.60 (equivalent to 25 or more successes out of the 30 procedures).

Secondary Outcome Measures

Name	Time	Method
Post-operative Efficacy Assessment	up to 30 days	Post-operative efficacy was assessed by the investigator, covering the time period from the end of the procedure up to 24 hours following the last infusion of study medication. This assessment took the post-operative bleeding and oozing into consideration
Assessment of Intra-operative Hemostatic Efficacy	1 Day	The efficacy of Wilate during surgical procedures was assessed by a 4-point ordinal efficacy scale by the surgeon at the end of the surgical procedure and took the predicted versus actual blood loss and transfusion requirements into consideration. Outcome measure 1 takes the results of outcome measure 2 and 3 into consideration and is an overall assessment covering intra- and post-operative efficacy.

Locations (15): SHAT Joan Pavel
🇧🇬
Sofia, Bulgaria
Louis Turcanu Childrens Emergency Hospital
🇷🇴
Timisoara, Romania
Christian Medical College
🇮🇳
Vellore, Tamil Nadu, India
Sultan Quaboos University Hospital
🇴🇲
Muscat, Oman
Instytut Hematologii i Transfuzjologii
🇵🇱
Warsaw, Poland
Azienda Ospedaliero Universitaria Careggi
🇮🇹
Florence, Italy
ULSS6 Vicenza Ematologia
🇮🇹
Vicenza, Italy
UNC-CH Comprehensive Hemophilia Center
🇺🇸
Chapel Hill, North Carolina, United States
Blood Center of Wisconsin
🇺🇸
Milwaukee, Wisconsin, United States
Fundeni Clinical Institute
🇷🇴
Bucharest, Romania
Indiana Hemophilia and Thrombosis Center
🇺🇸
Indianapolis, Indiana, United States
Granda Ospedale Maggiore Policlinico
🇮🇹
Milano, Italy
Hemophilia Comprehensive Care Center
🇿🇦
Johannesburg, South Africa
Sahyadri Specialty Hospital
🇮🇳
Pune, India
Ege University
🇹🇷
Izmir, Turkey