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

• ClinicalTrials.gov Identifier: NCT01365546
• Recruitment Status: Completed
• First Posted: June 3, 2011
• Results First Posted: March 9, 2015
• Last Update Posted: March 9, 2015
• Sponsor: Octapharma
• Information provided by (Responsible Party): Octapharma

Study Description

Brief Summary:

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

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

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 30 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (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
• Study Start Date: June 2011
• Actual Primary Completion Date: February 2014
• Actual Study Completion Date: April 2014

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures :

1. 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 ]
   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 :

1. Assessment of Intra-operative Hemostatic Efficacy [ Time Frame: 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.
2. Post-operative Efficacy Assessment [ Time Frame: 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

Eligibility Criteria

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

Criteria

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

Locations

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

Bulgaria

SHAT Joan Pavel

Sofia, Bulgaria, 1233

India

Christian Medical College

Vellore, Tamil Nadu, India, 632004

Sahyadri Specialty Hospital

Pune, India, 411004

Italy

Azienda Ospedaliero Universitaria Careggi

Florence, Italy, 50133

Granda Ospedale Maggiore Policlinico

Milano, Italy, 20122

ULSS6 Vicenza Ematologia

Vicenza, Italy, 36100

Oman

Sultan Quaboos University Hospital

Muscat, Oman, PC123

Poland

Instytut Hematologii i Transfuzjologii

Warsaw, Poland, 0.-776

Romania

Fundeni Clinical Institute

Bucharest, Romania, 022328

Louis Turcanu Childrens Emergency Hospital

Timisoara, Romania, 300011

South Africa

Hemophilia Comprehensive Care Center

Johannesburg, South Africa, 2193

Turkey

Ege University

Izmir, Turkey, 35100

Sponsors and Collaborators

Octapharma

More Information

• Responsible Party: Octapharma
• ClinicalTrials.gov Identifier: NCT01365546
• Other Study ID Numbers: Wil-24
• First Posted: June 3, 2011
• Results First Posted: March 9, 2015
• Last Update Posted: March 9, 2015
• Last Verified: March 2015

Additional relevant MeSH terms:

Von Willebrand Diseases; Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Coagulation Protein Disorders; Blood Platelet Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn