Phase 3
N=43
Clinical Study to Investigate the Efficacy and Safety of Wilate During Prophylaxis in Previously Treated Patients With VWD
Von Willebrand Diseases
Bottom Line
View on ClinicalTrials.gov: NCT04052698 ↗Enrolled (actual)
43
Serious AEs
9.3%
Results posted
Oct 2023
Primary outcome: Primary: Total Annualized Bleeding Rate (TABR) — 3.73; 4.28; 6.31; 5.24 bleeding events per year
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Wilate (Drug)
- Age
- Pediatric, Adult, Older Adult · 6+ yrs
- Sex
- All
- Sponsor
- Octapharma
- Primary completion
- Apr 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Total Annualized Bleeding Rate (TABR) |
3.73; 4.28; 6.31; 5.24 | — |
| PRIMARY Comparison of Total Annualized Bleeding Rates (TABR) During Prophylaxis Treatment in Study WIL-31 to On-demand Treatment in the Same Patient Population in the Preceding Study WIL-29 |
33.3751; 5.4914 | <0.0001 sig |
| SECONDARY Spontaneous Annualized Bleeding Rate (SABR) |
2.53; 6; 4.16; 3.23 | — |
| SECONDARY Comparison of Spontaneous Annualized Bleeding Rates (SABR) During Prophylaxis Treatment in Study WIL-31 to On-demand Treatment in the Same Patient Population in the Preceding Study WIL-29. |
24.4168; 3.3925 | <0.0001 sig |
| SECONDARY Wilate Consumption for Prophylaxis (mFAS Population) |
31.06; 31.04; 66.06; 287.26 | — |
| SECONDARY Incremental In Vivo Recovery (IVR) of Von Willebrand Factor Activity (VWF:RCo) |
1.439; 1.273 | — |
| SECONDARY Incremental In Vivo Recovery (IVR) of FVIII |
1.697; 2.140 | — |
| SECONDARY Efficacy of Wilate in the Treatment of Breakthrough Bleeding Events (BEs) |
109; 11; 1; 0 | — |
| SECONDARY Wilate Exposure for Prophylaxis (mFAS Population) |
105.55 | — |
Summary
This is a prospective, non-controlled, international, multi-center phase 3 study investigating the efficacy and safety of Wilate in previously treated adult patients with VWD, to obtain additional data on the safety and efficacy of Wilate in previously treated patients with VWD undergoing regular prophylaxis.
Eligibility Criteria
Inclusion Criteria
Patients who meet all of the following criteria are eligible for the study:
- Aged ≥6 years at the time of screening
- VWD type 1 (baseline von Willebrand factor activity [VWF:Ristocetin Co-factor (RCo)] 5 times of upper limit of normal, creatinine >120 µmol/L)
- Platelet count 10 mg/day), or similar drugs
- Pregnant or breast-feeding at the time of enrolment
- Cervical or uterine conditions causing abnormal uterine bleeding (including infection, dysplasia)
- Treatment with any IMP in another interventional clinical study currently or within 4 weeks before enrolment
- Other coagulation disorders or bleeding disorders due to anatomical reasons
- Known hypersensitivity to any of the components of the study drug
Data sourced from ClinicalTrials.gov (NCT04052698). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.