Phase 3
N=12
Efficacy, PK, Immunogenicity and Safety of Wilate in Severe Von Willebrand Disease (VWD) Patients <6 Years of Age
Von Willebrand Disease
Bottom Line
View on ClinicalTrials.gov: NCT04953884 ↗Enrolled (actual)
12
Serious AEs
33.3%
Results posted
Feb 2026
Primary outcome: Primary: Total Annualised Bleeding Rate (TABR) During Prophylactic Treatment With Wilate. — 4.6; 3.7 Annualized number of bleeding episodes
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Wilate (Drug)
- Age
- Pediatric
- Sex
- All
- Sponsor
- Octapharma
- Primary completion
- Dec 2024
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Total Annualised Bleeding Rate (TABR) During Prophylactic Treatment With Wilate. |
4.6; 3.7 | — |
| SECONDARY Area Under the Curve (AUC) of Wilate for VWF:Ac (VWF:RCo) Over Time |
1100 | — |
| SECONDARY Area Under the Curve (AUC) of Wilate for FVIII (OS) Over Time |
2410 | — |
| SECONDARY AUC Normalised for the Administered Dose (AUCnorm) of Wilate for VWF:Ac (VWF:RCo) Over Time |
13.7 | — |
| SECONDARY AUC Normalised for the Administered Dose (AUCnorm) of Wilate for FVIII:C (OS) Over Time |
30.1 | — |
| SECONDARY Clearance (CL) of Wilate for VWF:Ac (VWF:RCo) Over Time |
0.09 | — |
| SECONDARY Clearance (CL) of Wilate for FVIII:C (OS) Over Time |
0.04 | — |
| SECONDARY Maximum Plasma Concentration (Cmax) of Wilate for VWF:Ac (VWF:RCo) Over Time |
105 | — |
| SECONDARY Maximum Plasma Concentration (Cmax) of Wilate for FVIII:C (OS) Over Time |
114.1 | — |
| SECONDARY Mean Residence Time (MRT) of Wilate for VWF:Ac (VWF:RCo) Over Time |
15.6 | — |
| SECONDARY Mean Residence Time (MRT) of Wilate for FVIII:C (OS) Over Time |
22.5 | — |
| SECONDARY In Vivo Half-life (T1/2) of Wilate for VWF:Ac (VWF:RCo) Over Time |
11.7 | — |
| SECONDARY In Vivo Half-life (T1/2) of Wilate for FVIII:C (OS) Over Time |
15 | — |
| SECONDARY Time to Reach Maximum Plasma Concentration (Tmax) of Wilate for VWF:Ac (VWF:RCo) Over Time |
0.27 | — |
| SECONDARY Time to Reach Maximum Plasma Concentration (Tmax) of Wilate for FVIII:C (OS) Over Time |
0.26 | — |
| SECONDARY Volume of Distribution (Vd) of Wilate for VWF:Ac (VWF:RCo) Over Time |
1.12 | — |
| SECONDARY Volume of Distribution (Vd) of Wilate for FVIII:C (OS) Over Time |
0.82 | — |
| SECONDARY Incremental In-vivo Recovery (IVR) of Wilate for VWF:Ac (VWF:RCo) Over Time |
1.26; 1.75 | — |
| SECONDARY Incremental In-vivo Recovery (IVR) of Wilate for FVIII:C (OS) Over Time |
1.48; 1.69 | — |
| SECONDARY Efficacy of Wilate Measured by the Proportion of BEs Successfully Treated With Wilate |
46; 1; 0; 0 | — |
| SECONDARY The Overall Efficacy of Wilate in Perioperative Prophylaxis Against Excessive Bleeding as Assessed at the End of the Postoperative Period by the Responsible Treating Investigator |
1; 0; 0; 0 | — |
| SECONDARY Consumption of Wilate for Prophylactic Treatment |
54; 120.1 | — |
| SECONDARY Consumption of Wilate for the Treatment of BEs (On-demand Treatment) |
58.7; 55 | — |
| SECONDARY Consumption of Wilate During Surgical Prophylaxis |
62.1 | — |
| SECONDARY Number of Participants With Detectable Inhibitory Antibodies Against VWF and/or FVIII |
1 | — |
| SECONDARY Number of Participants With Detectable Thromboembolic Events |
— | — |
| SECONDARY Change in Haemophilia Joint Health Score |
1.55; 0.80; -0.90 | — |
Summary
The WIL-33 study aimed to determine the efficacy, pharmacokinetics, immunogenicity and safety of wilate as routine prophylaxis in up to 12 paediatric patients (eight evaluable) with severe von Willebrand Disease VWD (defined as screening von Willebrand factor ristocetin cofactor activity [VWF:RCo] <20%) under the age of 6 years, over a period of 12 months.
Eligibility Criteria
Inclusion Criteria
- Patients aged 10 mg/day), or similar drugs
- Treatment with any investigational medicinal product (IMP) in another interventional clinical study currently or within four weeks before enrolment
- Other coagulation disorders or bleeding disorders
- Known hypersensitivity to any of the components of the study drug
Data sourced from ClinicalTrials.gov (NCT04953884). 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.