Wilate in Subjects With Von Willebrand Disease Who Undergo Surgery (WONDERS)

Prospective, Open-Label, Multi-Center, Phase III CLinical Study to Investigate the Efficacy and Safety of Human Factor VWF/FVIII Concentrate (Wilate) in Subjects With Inherited Von Willebrand Disease Who Undergo Surgical Procedures

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

Study Overview

• Status: Completed
• Conditions: Prevent Bleeding in Major Surgery
• Intervention / Treatment: Biological: human VWF/FVIII concentrate

• Study Type: Interventional
• Enrollment (Actual): 30
• Phase: Phase 3

Contacts and Locations

Study Locations

• Bulgaria
  • Sofia, Bulgaria, 1233
    • SHAT Joan Pavel
• India
  • Pune, India, 411004
    • Sahyadri Specialty Hospital
  • Tamil Nadu
    • Vellore, Tamil Nadu, India, 632004
      • Christian Medical College
• Italy
  • Florence, Italy, 50133
    • Azienda Ospedaliero Universitaria Careggi
  • Milano, Italy, 20122
    • Granda Ospedale Maggiore Policlinico
  • Vicenza, Italy, 36100
    • ULSS6 Vicenza Ematologia
• Oman
  • Muscat, Oman, PC123
    • Sultan Quaboos University Hospital
• Poland
  • Warsaw, Poland, 0.-776
    • Instytut Hematologii i Transfuzjologii
• Romania
  • Bucharest, Romania, 022328
    • Fundeni Clinical Institute
  • Timisoara, Romania, 300011
    • Louis Turcanu Childrens Emergency Hospital
• South Africa
  • Johannesburg, South Africa, 2193
    • Hemophilia Comprehensive Care Center
• Turkey
  • Izmir, Turkey, 35100
    • Ege University
• United States
  • Indiana
    • Indianapolis, Indiana, United States, 46260
      • Indiana Hemophilia and Thrombosis Center
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27599
      • UNC-CH Comprehensive Hemophilia Center
  • Wisconsin
    • Milwaukee, Wisconsin, United States, 53201
      • Blood Center of Wisconsin

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Prevention
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: human VWF/FVIII concentrate	Biological: human VWF/FVIII concentrate; intravenous infusion. Dose based on subject's individual invivo-recovery

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	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).	30 Days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Assessment of Intra-operative Hemostatic Efficacy	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.	1 Day
Post-operative Efficacy Assessment	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	up to 30 days

Collaborators and Investigators

• Sponsor: Octapharma

Study record dates

Study Major Dates

• Study Start: June 1, 2011
• Primary Completion (Actual): February 1, 2014
• Study Completion (Actual): April 1, 2014

Study Registration Dates

• First Submitted: May 27, 2011
• First Submitted That Met QC Criteria: June 2, 2011
• First Posted (Estimate): June 3, 2011

Study Record Updates

• Last Update Posted (Estimate): March 9, 2015
• Last Update Submitted That Met QC Criteria: March 5, 2015
• Last Verified: March 1, 2015

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Blood Platelet Disorders; Von Willebrand Diseases
• Other Study ID Numbers: Wil-24