Prevent Bleeding in Major Surgery Clinical Trial
— WONDERSOfficial 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
Proportion of surgeries in which the primary endpoint (overall assessment) is classified as success.
| Status | Completed |
| Enrollment | 30 |
| Est. completion date | April 2014 |
| Est. primary completion date | February 2014 |
| Accepts healthy volunteers | No |
| Gender | Both |
| Age group | 6 Years and older |
| 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 |
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Prevention
| Country | Name | City | State |
|---|---|---|---|
| Bulgaria | SHAT Joan Pavel | Sofia | |
| India | Sahyadri Specialty Hospital | Pune | |
| India | Christian Medical College | Vellore | Tamil Nadu |
| Italy | Azienda Ospedaliero Universitaria Careggi | Florence | |
| Italy | Granda Ospedale Maggiore Policlinico | Milano | |
| Italy | ULSS6 Vicenza Ematologia | Vicenza | |
| Oman | Sultan Quaboos University Hospital | Muscat | |
| Poland | Instytut Hematologii i Transfuzjologii | Warsaw | |
| Romania | Fundeni Clinical Institute | Bucharest | |
| Romania | Louis Turcanu Childrens Emergency Hospital | Timisoara | |
| South Africa | Hemophilia Comprehensive Care Center | Johannesburg | |
| Turkey | Ege University | Izmir | |
| United States | UNC-CH Comprehensive Hemophilia Center | Chapel Hill | North Carolina |
| United States | Indiana Hemophilia and Thrombosis Center | Indianapolis | Indiana |
| United States | Blood Center of Wisconsin | Milwaukee | Wisconsin |
| Lead Sponsor | Collaborator |
|---|---|
| Octapharma |
United States, Bulgaria, India, Italy, Oman, Poland, Romania, South Africa, Turkey,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | 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 | No |
| Secondary | 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 | No |
| Secondary | 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 | No |