Wilate for Von Willebrand Disease
(EMPOWER Trial)
Recruiting in Palo Alto (17 mi)
Age: 18+
Sex: Female
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 3
Recruiting
Sponsor: Unity Health Toronto
Must be taking: Wilate
Must not be taking: Desmopressin, Hormonal contraceptives
Disqualifiers: Pregnancy, Breastfeeding, Liver disease, others
Pivotal Trial (Near Approval)
Prior Safety Data
Trial Summary
What is the purpose of this trial?The EMPOWER trial is a pilot multi-center, placebo-controlled (normal saline), double-blind (patient and outcome assessor), crossover, 2-year randomized trial in female outpatients with von Willebrand disease (VWD) and heavy menstrual bleeding to determine trial feasibility and viability, and to explore assay sensitivity of the proposed efficacy clinical outcomes for a definitive randomized controlled trial
Will I have to stop taking my current medications?
The trial requires that you stay on your current treatment for heavy menstrual bleeding and iron deficiency anemia for at least 3 cycles before joining and throughout the study. However, you cannot take desmopressin at the same time as Wilate, unless it's for treating excessive bleeding.
What data supports the effectiveness of the drug Wilate for Von Willebrand Disease?
Research shows that Wilate is effective and safe for managing bleeding in patients with Von Willebrand Disease, with high success rates in both surgical and non-surgical settings. In various studies, Wilate was rated as 'excellent' or 'good' in over 96% of cases, and it effectively stopped bleeding episodes quickly, often within one or two days.
12345Is Wilate safe for treating Von Willebrand Disease?
Wilate has been shown to be safe and well-tolerated in both adults and children with Von Willebrand Disease. Studies report no unexpected adverse reactions, no thrombotic events (blood clots), and no development of inhibitory antibodies, indicating a strong safety profile.
13456How is the drug Wilate unique in treating von Willebrand disease?
Wilate is unique because it is a plasma-derived concentrate that contains von Willebrand factor and factor VIII in a natural 1:1 ratio, making it effective for both prophylactic and on-demand treatment of bleeding episodes in von Willebrand disease. It is also double virus-inactivated, ensuring a high safety profile, and has shown excellent efficacy in both adults and children, including those undergoing surgery.
23457Eligibility Criteria
This trial is for adult female outpatients with von Willebrand disease (VWD) who suffer from heavy menstrual bleeding. Participants must have a modified PBAC score over 100, agree to nurse-administered infusions, and use specific feminine hygiene products provided by the sponsor. They should be on stable treatment for their condition and anemia.Inclusion Criteria
I have been diagnosed with inherited von Willebrand disease.
You must agree to only use the feminine hygiene products provided by the study sponsor.
I am a woman over 18 with heavy menstrual bleeding and my doctor recommends Wilate®.
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Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Treatment Period 1
Participants receive either pdVWF:FVIII concentrate or placebo for 4 menstrual cycles
4 cycles
Washout
A washout period where no study-based treatment is delivered
1 cycle
Treatment Period 2
Participants crossover to the comparator treatment for 4 menstrual cycles
4 cycles
Follow-up
Participants are monitored for safety and effectiveness after treatment
4 weeks
Participant Groups
The EMPOWER trial tests a lyophilized concentrate of human coagulation factors against placebo in women with VWD to see if it can reduce heavy menstrual bleeding. It's a double-blind crossover study, meaning patients will receive both the treatment and placebo at different times without knowing which one they're getting.
2Treatment groups
Experimental Treatment
Placebo Group
Group I: pdVWF:FVIII concentrate (Wilate®) Treatment and Standard CareExperimental Treatment1 Intervention
Wilate® at a dose of 30-60 IU VWF:RCo/kg for the two anticipated heaviest days of bleeding every 24-48 hours within the first 4 days of menstruation will be provided. Additional two optional doses 24-48 hours from the last can be provided. A minimum of 2 doses must be provided.
Group II: Placebo and Standard CarePlacebo Group1 Intervention
Patients randomized to the placebo arm will receive intravenous placebo (normal saline) at the same approximate volume and frequency as the study drug.
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
St. Michael's HospitalToronto, Canada
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Who Is Running the Clinical Trial?
Unity Health TorontoLead Sponsor
References
Real-World Data on the Effectiveness and Safety of wilate for the Treatment of von Willebrand Disease. [2021]Background  The efficacy and safety of wilate (human von Willebrand factor/coagulation factor VIII) in patients with von Willebrand disease (VWD) has been demonstrated in clinical trials. Here, we present real-world data on the use of wilate for the routine care of patients with VWD. Objectives  The objectives of this observational, prospective, phase 4 study were to evaluate the safety, tolerability, and effectiveness of wilate in on-demand treatment of bleeding episodes (BEs), long-term prophylaxis, and surgical prophylaxis among patients with any type of VWD. Methods  Patients were enrolled at 31 study centers in 11 countries and followed for up to 2 years. Safety endpoints included adverse drug reactions (ADRs) and drug tolerability. Effectiveness was assessed using annualized bleeding rates (ABRs) during prophylaxis and predefined criteria for the treatment of BEs and surgical prophylaxis. Results  A total of 111 patients (76 [68%] female) including 41 (37%) children were treated with wilate. Twenty-five patients received prophylaxis, 29 on-demand treatment, and 62 surgical prophylaxis. Tolerability was rated by patients as "excellent" for 96.2% of 6,497 infusions. No unexpected ADRs or thrombotic events were reported. Median ABR during prophylaxis was 1.9. Effectiveness was assessed as "excellent" or "good" by patients and investigators for 100% of BEs treated on-demand, 98% (patient rating) and 99% (investigator rating) of breakthrough BEs, and 99% of surgical procedures (investigator rating). Conclusion  wilate was safe, well tolerated, and effective for the prevention and treatment of bleeding in pediatric and adult VWD patients in a real-world setting.
Efficacy and safety of a new generation von Willebrand factor/factor VIII concentrate (Wilate®) in the management of perioperative haemostasis in von Willebrand disease patients undergoing surgery. [2012]The aim of this study was to assess the efficacy of Wilate®, a new generation, plasma-derived, high-purity, double virus-inactivated von Willebrand factor (VWF) and factor VIII (FVIII) concentrate (ratio close to physiological 1:1) in the perioperative management of haemostasis in von Willebrand disease (VWD). Data for VWD patients who received Wilate® for perioperative management were obtained from four European, prospective, open-label, non-controlled, non-randomised, multicentre phase II or III clinical trials. A total of 57 surgical procedures were performed (major: n = 27; minor n = 30) in 32 patients. The majority of patients (n = 19, 59.4%) had type 3 VWD, 9 (28.1%) had type 2 VWD and four (12.5%) had type 1 VWD. During major surgery, median daily FVIII dose and mean number of infusions were 25 IU•kg-1 FVIII (VWF:RCo ~23 IU•kg-1) and 11.0, respectively. Corresponding values for minor surgery were 35 IU•kg-1 (VWF:RCo ~32 IU•kg-1) and 1.5. The efficacy of Wilate® was rated by the investigator as excellent or good in 51 of 53 (96%) procedures. Tolerability was rated as very good or good in 100% of major surgeries (27 of 27) and minor surgeries (29 of 29). Wilate® is an effective and well-tolerated VWF/FVIII replacement therapy in the perioperative management of haemostasis in patients with VWD. It can be administered at a similar FVIII dose, but at a lower VWF dose, as compared to older generation products. Clinical benefits were shown in a population with a high proportion of type 3 VWD patients.
Efficacy and safety of Wilate in paediatric VWD patients under 6 years of age - results of a prospective multicentre clinical study including recovery information. [2013]Treatment with exogenous von Willebrand factor (VWF) is indicated in patients with von Willebrand disease (VWD) in whom treatment with 1-deamino-8-d-arginine vasopressin/desmopressin is contraindicated. Wilate is a new generation plasma-derived concentrate of native VWF and coagulation factor VIII (FVIII) (in a physiological 1:1 ratio) developed for the treatment of VWD. This is the first study to report safety, efficacy and in vivo recovery (IVR) data from 15 paediatric patients less than 6 years of age who received Wilate for either prophylaxis, on-demand treatment or for treatment in surgical procedures during a prospective open-label trial (VWD type 1: 5, type 2A: 1, type 2B: 2, type 3: 6, unknown type: 1 patients). Analysis of IVR for VWF and FVIII suggested an appropriate and consistent rise in coagulation activity after Wilate administration. Overall efficacy was rated as excellent or good for 99.7% [prophylactic infusions] and 100% [bleeding episodes/surgical procedures]. More than 82% of bleeding episodes resolved after 1 day of treatment, and a Wilate dosage of 20-50 IU kg(-1) was sufficient to achieve haemostasis in 97% of bleeding episodes. All surgical procedures were successfully managed with Wilate. No thromboembolic events were observed during the study, and no patient developed anti-VWF antibodies or FVIII inhibitors. In conclusion, this study confirms both the expected IVR profile in paediatric patients and the excellent efficacy, tolerability and safety profile of Wilate observed previously in adults. Wilate showed excellent efficacy in the treatment of bleeding when used prophylactically or on-demand, and in the treatment of surgical procedures.
Treatment and prevention of acute bleedings in von Willebrand disease--efficacy and safety of Wilate, a new generation von Willebrand factor/factor VIII concentrate. [2009]For many patients with von Willebrand disease (VWD), the replacement therapy with von Willebrand factor (VWF)/factor VIII (FVIII) concentrates is the treatment of choice. To evaluate clinical efficacy, safety and tolerability of Wilate, an albumin-free VWF/FVIII concentrate with a ratio of the two haemostatic moieties of approximately 1 to 1, a prospective clinical programme has been designed. The dataset on the treatment and prevention of bleedings is derived from 44 patients (20 males and 24 females) of all VWD types. Thousand and ninety five bleeding episodes were treated with an overall efficacy rating of excellent or good in 96%. The median dose per treatment day was 26 IU FVIII:C per kg. Eighty-one per cent of bleeds were stopped within 1 or 2 days. Gastrointestinal (GI) bleeds needed higher doses (mean 44 IU kg(-1)) and longer treatment (mean 4 days). Efficacy and dosing data from eight children of 12 or less years of age did not differ significantly from the overall study population. Nineteen patients, including six children, were treated prophylactically for more than 3 months (mean 14.8, range 3-46) with a mean prophylactic dose of 27.4 IU kg(-1) and a mean frequency of 1.9 infusions per week. A drop of bleeding frequency from a mean of 4.5 to 1.4 bleeds per month was observed. The overall tolerability was very good. Adverse drug reactions were rare and were mild or moderate in their intensity. The large prospective clinical dataset shows that Wilate is efficacious and safe in the treatment and prevention of haemorrhages in all VWD types in both adult and paediatric patients.
Safety and efficacy of a von Willebrand factor/factor VIII concentrate (Wilate®): a single centre experience. [2014]von Willebrand disease (VWD) is the commonest inherited bleeding disorder. Management of major surgery or bleeding often requires treatment with a plasma-derived (pd) VWF/FVIII containing concentrate. Wilate® is a dual-virally inactivated pd-concentrate, produced specifically for the treatment of VWD, containing physiological (1:1) ratios of VWF: FVIII. We reviewed efficacy and safety of Wilate® usage (2007-2012) at our centre including 2 years following product switching the majority of patients. Clinical and laboratory data of all adult patients treated with Wilate® during the study period were evaluated. Fifty four patients used 3 972 150 IU of Wilate® (1378 infusions) between 1/3/07 and 1/5/12. Efficacy was rated as being excellent or good in 94% of surgical episodes (n = 70; 34 patients) and 98% of bleeding/traumatic episodes (n = 46; 25 patients). Eight patients (2 636 100 IU) were managed on home treatment regimens. Two patients switched to Wilate® prophylaxis in the evaluation period, demonstrating similar efficacy to a previous product. Incremental recoveries (n = 37) were 2.24 IU dL(-1) per IU kg(-1) for FVIII:C, 2.39 IU dL(-1) per IU kg(-1) for VWF:Ag and 1.88 IU dL(-1) per IU kg(-1) for VWF:RCo. Six adverse events occurred in six patients (11.1% patients) over 1378 infusions (0.44%). Half of these were retrospectively felt to be infusion speed related. No notable accumulation of FVIII was seen in patients treated for ≥3 days. There was no treatment failure, thrombosis, transfusion transmitted infection or inhibitory VWF antibodies seen. Our findings confirm safety and efficacy of Wilate® in an adult VWD population with lack of notable FVIII accumulation.
Wilate use in 47 children with von Willebrand disease: the North London paediatric haemophilia network experience. [2014]Children with von Willebrand disease (VWD) in whom DDAVP is ineffective or contraindicated require treatment with a coagulation factor concentrate containing von Willebrand factor (VWF) and factor VIII (FVIII). The aim of this study was to monitor the safety, efficacy and tolerability of Wilate(®) (a VWF:FVIII concentrate with a 1:1 ratio) used across the North London Paediatric Haemophilia Network since May 2010. In total, 47 children (aged 0.0-17.0 years) with type 1 (n = 28), type 2 (n = 7), type 3 (n = 10) and acquired VWS (n = 2) have been treated for bleeds, surgery and/or prophylaxis using 260 000 IU Wilate(®). Analysis of dose and frequency of treatment show expected responses to treatment with mean doses of 55, 50 and 50 IU kg(-1) for bleeds, surgery and prophylaxis respectively. Most bleeds responded to a single treatment. Surgical procedures were covered with clinician approved dosing schedules with 95% (39/41) reported as having excellent or good efficacy. There was no accumulation of FVIII or VWF and no thromboembolic events. This case series confirms the efficacy, safety and tolerability of Wilate(®) in neonates, children and adolescents when used on-demand, prophylactically and in the surgical setting.
Switch to pdVWF:pdFVIII concentrate for prophylaxis in a paediatric patient with Type 3 von Willebrand disease: a case report. [2023]To report the long-term prophylaxis management of a child with type 3 von Willebrand disease by switching to Wilate (Octapharma AG), a plasma-derived, double virus-inactivated concentrate of freeze-dried of a 1 to 1 ratio of active Von Willebrand Factor and Factor VIII (pdVWF:pdFVIII) recently marketed as Eqwilate in France.