Surveillance of Safety and Efficacy of Wilate in Patients With Von Willebrand Disease
- Conditions
- Von Willebrand Disease
- Interventions
- Other: Patients using Wilate as standard of care
- Registration Number
- NCT01602419
- Lead Sponsor
- Octapharma
- Brief Summary
This is an observational study, hence there is no study hypothesis
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 120
- Patients with a diagnosis of von Willebrand Disease who have been prescribed Wilate
Not provided
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Patients using Wilate as standard of care treatment Patients using Wilate as standard of care This patient population is being treated with Wilate as standard of care treatment
- Primary Outcome Measures
Name Time Method Incidence of Adverse Drug Reactions (ADRs) (%) Throughout the duration of each patient's participation in the study (mean [± standard deviation (SD)]: 575 days [±326]; median [range]: 731 days [2-1185]) Medical Dictionary for Regulatory Activities (MedDRA) primary system organ class preferred term. Incidence rate = number of patients reporting the event / number of patients \* 100
Tolerability Assessment of Wilate Infusions by Reason for Administration During and immediately after each infusion of Wilate during the study. Tolerability was assessed using a 3-point verbal rating scale (excellent; satisfactory; unsatisfactory) according to overall feeling during and after Wilate therapy and occurrence of ADRs.
Tolerability was assessed for infusions given for on-demand and prophylactic treatment, but not for infusions administered for surgeries or for the purpose of thrombogenicity assessment. In some instances, however, investigators also recorded the tolerability of infusions given for surgical prophylaxis. For infusions administered for surgeries, only those with available tolerability assessments are presented. Wilate infusion may have been administered to a patient for more than one reason and may be included in more than one category (e.g., if a patient was under Wilate prophylaxis, they could also receive Wilate for the treatment of a bleeding episode \[BE\] or surgery or menstruation).
- Secondary Outcome Measures
Name Time Method Patient and Investigator Efficacy Analysis Assessment of the Treatment of Bleeding Episodes (BEs) During and immediately after treatment of each BE. Document the efficacy of Wilate in the treatment of acute BEs, breakthrough BEs in patients receiving prophylactic treatment, and menstrual BEs.
Efficacy was rated on a 4-point scale (excellent; good; moderate; none) according to overall haemostasis.Efficacy Analysis for the Prevention of Breakthrough Bleeds During Prophylaxis At the end of the study for each patient (study duration: mean [±SD]: 805 days [±247]; median [range]: 797 days [144-1185]). Efficacy was rated on a 4-point scale (excellent; good; moderate; none) according to the number of breakthrough bleeds per month. The treatment regimen for prophylactic treatment was at the discretion of the investigator and differed for each patient, as did the duration of prophylactic treatment. The prophylaxis efficacy population (EFF-P) was subdivided into 2 groups, prophylaxis on a continuous basis (EFF-PC population) and prophylaxis on an intermittent basis (EFF-PI population). In total, 25 patients received Wilate for prophylaxis and of these, 17 patients received prophylaxis on a continuous basis, which was defined as: (1) patients having received continuous prophylaxis over a period of at least 3 months, with no treatment gaps longer than 14 days; or (2) patients having received continuous prophylaxis for at least 1 year with an average of 1 infusion/per week (these patients may have had gaps of more than 14 days).
Efficacy Analysis of Surgical Prophylaxis During and immediately after each surgery. Efficacy was rated on a 4-point scale (excellent; good; moderate; none) according to overall haemostasis during and after surgery.
Overall Efficacy Assessment by Patient and Physician at the End of the Treatment Period At the end of the study for each patient (study duration: mean [±SD]: 596 days [±336]; median [range]: 732 days [2-1185]). Patient and investigator assessment of the overall efficacy of Wilate performed at the end of the study for each patient. Efficacy was rated on a 4-point scale (excellent; good; moderate; none); criteria for assessment were not defined.
Trial Locations
- Locations (24)
Los Angeles Biomedical Research Institute
🇺🇸Torrance, California, United States
Nicklaus Children's Hospital
🇺🇸Miami, Florida, United States
St. Michael's Hospital
🇨🇦Toronto, Ontario, Canada
Virginia Commonwealth University
🇺🇸Richmond, Virginia, United States
Fundación de la Hemofilia de Salta
🇦🇷Salta, Argentina
Hospital Fundacion Jiminez Diaz
🇪🇸Madrid, Spain
University of Utah
🇺🇸Salt Lake City, Utah, United States
St. John Regional Hospital
🇨🇦Saint John, New Brunswick, Canada
Werlhof-Institut
🇩🇪Hannover, Germany
Hospital Universitario de Burgos
🇪🇸Burgos, Spain
Hospital Pereira Rossell
🇺🇾Montevideo, Uruguay
University Hospital Ostrava
🇨🇿Ostrava-Poruba, Czechia
University of Alberta
🇨🇦Edmonton, Alberta, Canada
Queens University
🇨🇦Kingston, Ontario, Canada
Eastern Regional Health Authority
🇨🇦St. John's, Newfoundland and Labrador, Canada
Maisonneuve-Rosemont Hospital
🇨🇦Montreal, Quebec, Canada
Skåne University Hospital
🇸🇪Malmö, Sweden
Sanatorio Americano
🇺🇾Montevideo, Uruguay
McMaster University
🇨🇦Hamilton, Ontario, Canada
Children's Hospital of Eastern Ontario
🇨🇦Ottawa, Ontario, Canada
Centro Hospitalar Cova da Beira
🇵🇹Covilhã, Portugal
Hospital San Pedro de Alcantara
🇪🇸Cáceres, Spain
Great Ormond Street Hospital for Children
🇬🇧London, United Kingdom
Vancouver General Hospital
🇨🇦Vancouver, British Columbia, Canada