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

This study has been completed.
Information provided by (Responsible Party):
ClinicalTrials.gov Identifier:
First received: May 27, 2011
Last updated: March 5, 2015
Last verified: March 2015
Proportion of surgeries in which the primary endpoint (overall assessment) is classified as success.

Condition Intervention Phase
Prevent Bleeding in Major Surgery
Biological: human VWF/FVIII concentrate
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Prevention
Official Title: 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

Resource links provided by NLM:

Further study details as provided by Octapharma:

Primary Outcome Measures:
  • 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. [ Time Frame: 30 Days ] [ Designated as safety issue: No ]
    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:
  • Assessment of Intra-operative Hemostatic Efficacy [ Time Frame: 1 Day ] [ Designated as safety issue: No ]
    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.

  • Post-operative Efficacy Assessment [ Time Frame: up to 30 days ] [ Designated as safety issue: No ]
    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

Enrollment: 30
Study Start Date: June 2011
Study Completion Date: April 2014
Primary Completion Date: February 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: human VWF/FVIII concentrate Biological: human VWF/FVIII concentrate
intravenous infusion. Dose based on subject's individual invivo-recovery


Ages Eligible for Study:   6 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01365546

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

Responsible Party: Octapharma
ClinicalTrials.gov Identifier: NCT01365546     History of Changes
Other Study ID Numbers: Wil-24 
Study First Received: May 27, 2011
Results First Received: January 6, 2015
Last Updated: March 5, 2015
Health Authority: United States: Food and Drug Administration
India: Drugs Controller General of India
South Africa: Medicines Control Council
Italy: National Monitoring Centre for Clinical Trials - Ministry of Health
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products
Romania: Ministry of Public Health
Turkey: Ministry of Health
Bulgaria: Ministry of Health

Additional relevant MeSH terms:
Von Willebrand Diseases
Blood Coagulation Disorders
Blood Coagulation Disorders, Inherited
Blood Platelet Disorders
Coagulation Protein Disorders
Genetic Diseases, Inborn
Hematologic Diseases
Hemorrhagic Disorders

ClinicalTrials.gov processed this record on May 26, 2016