Trial · NCT04953884
Efficacy, PK, Immunogenicity and Safety of Wilate in Severe Von Willebrand Disease (VWD) Patients <6 Years of Age
NCT04953884, Phase 3, Completed, Octapharma; results first posted 2026-02-20
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. Efficacy, PK, Immunogenicity and Safety of Wilate in Severe Von Willebrand Disease (VWD) Patients <6 Years of Age. Trials, record NCT04953884. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04953884
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Efficacy, PK, Immunogenicity and Safety of Wilate in Severe Von Willebrand Disease (VWD) Patients <6 Years of Age
- NCT number
- NCT04953884
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 12 participants, actual
- Lead sponsor
- Octapharma
- Conditions
- Von Willebrand Disease
- Interventions
- Wilate
- First posted
- 2021-07-08
- Last updated
- 2026-02-20
- Results first posted
- 2026-02-20
Source ClinicalTrials.gov
Registered outcomes27
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Total Annualised Bleeding Rate (TABR) During Prophylactic Treatment With Wilate. | During 12 months of prophylactic treatment | TABR is defined as the total number of bleeding episodes (BEs) including spontaneous, traumatic, and other bleeds, occurring during the period from the first prophylactic dose of wilate to the study completion visit, divided by the duration (in years) between these two time points. Bleeding episodes that occurred during surgery periods were excluded from the calculation of TABR. Total BEs refers to all bleeding episodes that occurred during the study period, regardless of whether they were treated with wilate or not. Treated BEs are a subset of total BEs comprising of bleeding episodes that were treated with wilate. |
| Secondary | Area Under the Curve (AUC) of Wilate for VWF:Ac (VWF:RCo) Over Time | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate | Mean AUC of VWF:Ac after PK injection of wilate as measured by chromogenic assay. |
| Secondary | Area Under the Curve (AUC) of Wilate for FVIII (OS) Over Time | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate | Mean AUC of FVIII after PK injection of wilate as measured by chromogenic assay. |
| Secondary | AUC Normalised for the Administered Dose (AUCnorm) of Wilate for VWF:Ac (VWF:RCo) Over Time | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate | Mean AUC of VWF:Ac after PK injection of wilate, normalised for the actual administered dose (IU/kg), over time. AUC norm was measured and reported as \[h\*kg/IU\]/(dL/IU) which could also be reported as h\*kg/dL. |
| Secondary | AUC Normalised for the Administered Dose (AUCnorm) of Wilate for FVIII:C (OS) Over Time | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate | Mean AUC of FVIII after PK injection of wilate, normalised for the actual administered dose (IU/kg), over time. AUC norm was measured and reported as \[h\*kg/IU\]/(dL/IU) which could also be reported as h\*kg/dL. |
| Secondary | Clearance (CL) of Wilate for VWF:Ac (VWF:RCo) Over Time | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate | Mean clearance (CL) of VWF:Ac, measured by ristocetin cofactor assay \[VWF:RCo\]) after PK injection of wilate, calculated as the ratio of dose administered to the area under the plasma concentration-time curve (AUC), over time. Clearance was defined as the rate of drug elimination divided by plasma concentration, giving a volume of plasma from which drug is completely removed per unit of time. |
| Secondary | Clearance (CL) of Wilate for FVIII:C (OS) Over Time | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate | Mean clearance (CL) of FVIII:C, measured by one-stage assay \[OS\]) after PK injection of wilate, calculated as the ratio of dose administered to the area under the plasma concentration-time curve (AUC), over time. |
| Secondary | Maximum Plasma Concentration (Cmax) of Wilate for VWF:Ac (VWF:RCo) Over Time | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate | Mean maximum plasma concentration (Cmax) of VWF:Ac, measured by ristocetin cofactor assay \[VWF:RCo\]) after PK injection of wilate, as determined from the observed peak value in the plasma concentration-time profile. |
| Secondary | Maximum Plasma Concentration (Cmax) of Wilate for FVIII:C (OS) Over Time | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate | Mean maximum plasma concentration (Cmax) of FVIII:C, measured by one-stage assay \[OS\]) after PK injection of wilate, as determined from the observed peak value in the plasma concentration-time profile. |
| Secondary | Mean Residence Time (MRT) of Wilate for VWF:Ac (VWF:RCo) Over Time | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate | Mean residence time (MRT) of VWF:Ac, measured by ristocetin cofactor assay \[VWF:RCo\]) after PK injection of wilate, calculated as the average time a VWF:Ac molecule remains in the body, derived from the ratio of area under the first moment curve (AUMC) to AUC. |
1 to 10 of 27Source ClinicalTrials.gov
See all 27 registered outcomesOutcomes27
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Total Annualised Bleeding Rate (TABR) During Prophylactic Treatment With Wilate. | During 12 months of prophylactic treatment | The analysis population consists of all patients in the full analysis set (FAS) | Annualized number of bleeding episodes | MEAN |
| SECONDARY | Area Under the Curve (AUC) of Wilate for VWF:Ac (VWF:RCo) Over Time | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate | The analysis population consists of all patients in the PK per protocol population (PK-PP). | h*IU/dL | MEAN |
| SECONDARY | Area Under the Curve (AUC) of Wilate for FVIII (OS) Over Time | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate | Of the 10 patients in the PK per protocol population (PK-PP), evaluable data for FVIII measurements was available for only 8 patients. | h*IU/dL | MEAN |
| SECONDARY | AUC Normalised for the Administered Dose (AUCnorm) of Wilate for VWF:Ac (VWF:RCo) Over Time | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate | The analysis population consists of all 10 patients in the PK per protocol population (PK-PP). | h*kg/dL | MEAN |
| SECONDARY | AUC Normalised for the Administered Dose (AUCnorm) of Wilate for FVIII:C (OS) Over Time | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate | Of the 10 patients in the PK per protocol population (PK-PP), evaluable data for FVIII measurements was available for only 8 patients. | h*kg/dL | MEAN |
| SECONDARY | Clearance (CL) of Wilate for VWF:Ac (VWF:RCo) Over Time | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate | The analysis population consists of all 10 patients in the PK per protocol population (PK-PP). | dL/h/kg | MEAN |
| SECONDARY | Clearance (CL) of Wilate for FVIII:C (OS) Over Time | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate | Of the 10 patients in the PK per protocol population (PK-PP), evaluable data for FVIII measurements was available for only 8 patients. | dL/h/kg | MEAN |
| SECONDARY | Maximum Plasma Concentration (Cmax) of Wilate for VWF:Ac (VWF:RCo) Over Time | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate | The analysis population consists of all 10 patients in the PK per protocol population (PK-PP). | IU/dL | MEAN |
| SECONDARY | Maximum Plasma Concentration (Cmax) of Wilate for FVIII:C (OS) Over Time | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate | Of the 10 patients in the PK per protocol population (PK-PP), evaluable data for FVIII measurements was available for only 8 patients. | IU/dL | MEAN |
| SECONDARY | Mean Residence Time (MRT) of Wilate for VWF:Ac (VWF:RCo) Over Time | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate | The analysis population consists of all 10 patients in the PK per protocol population (PK-PP). | hours | MEAN |
1 to 10 of 27Source ClinicalTrials.gov
See all 27 outcomesResults by group40
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Total Annualised Bleeding Rate (TABR) During Prophylactic Treatment With Wilate. | Wilate Treatment | Total BEs | 4.6 | Standard Deviation 6.1 | Annualized number of bleeding episodes |
| Total Annualised Bleeding Rate (TABR) During Prophylactic Treatment With Wilate. | Wilate Treatment | Treated BEs | 3.7 | Standard Deviation 5.1 | Annualized number of bleeding episodes |
| Area Under the Curve (AUC) of Wilate for VWF:Ac (VWF:RCo) Over Time | Wilate Treatment | 1100 | Standard Deviation 532 | h*IU/dL | |
| Area Under the Curve (AUC) of Wilate for FVIII (OS) Over Time | Wilate Treatment | 2410 | Standard Deviation 1122 | h*IU/dL | |
| AUC Normalised for the Administered Dose (AUCnorm) of Wilate for VWF:Ac (VWF:RCo) Over Time | Wilate Treatment | 13.7 | Standard Deviation 6.7 | h*kg/dL | |
| AUC Normalised for the Administered Dose (AUCnorm) of Wilate for FVIII:C (OS) Over Time | Wilate Treatment | 30.1 | Standard Deviation 14.2 | h*kg/dL | |
| Clearance (CL) of Wilate for VWF:Ac (VWF:RCo) Over Time | Wilate Treatment | 0.09 | Standard Deviation 0.04 | dL/h/kg | |
| Clearance (CL) of Wilate for FVIII:C (OS) Over Time | Wilate Treatment | 0.04 | Standard Deviation 0.03 | dL/h/kg | |
| Maximum Plasma Concentration (Cmax) of Wilate for VWF:Ac (VWF:RCo) Over Time | Wilate Treatment | 105 | Standard Deviation 19.7 | IU/dL | |
| Maximum Plasma Concentration (Cmax) of Wilate for FVIII:C (OS) Over Time | Wilate Treatment | 114.1 | Standard Deviation 27.1 | IU/dL |
1 to 10 of 40Source ClinicalTrials.gov
See all 40 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Wilate Treatment | 12 | |
| Overall Study | Full analysis set (FAS) | Wilate Treatment | 12 | |
| Overall Study | Safety analysis set (SAF) | Wilate Treatment | 12 | |
| Overall Study | Study population of patients undergoing pharmacokinetic analysis (PK) | Wilate Treatment | 11 | |
| Overall Study | Study population of patients undergoing pharmacokinetic analysis as Per-protocol (PK-PP) | Wilate Treatment | 10 | |
| Overall Study | Per-protocol population (PP) | Wilate Treatment | 8 | |
| Overall Study | Surgery set (SURG) | Wilate Treatment | 1 | |
| Overall Study | COMPLETED | Wilate Treatment | 11 | |
| Overall Study | NOT COMPLETED | Wilate Treatment | 1 |
Source ClinicalTrials.gov
Adverse events32
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Bronchitis | Infections and infestations | Wilate Treatment | 1 | 12 | 1 |
| other | Conjunctivitis | Infections and infestations | Wilate Treatment | 1 | 12 | 1 |
| other | Ear infection | Infections and infestations | Wilate Treatment | 1 | 12 | 1 |
| other | Gastroenteritis | Infections and infestations | Wilate Treatment | 1 | 12 | 1 |
| other | Nasopharyngitis | Infections and infestations | Wilate Treatment | 2 | 12 | 2 |
| other | Pharyngitis | Infections and infestations | Wilate Treatment | 1 | 12 | 1 |
| other | Pneumonia | Infections and infestations | Wilate Treatment | 1 | 12 | 1 |
| other | Pulpitis dental | Infections and infestations | Wilate Treatment | 1 | 12 | 1 |
| other | Respiratory tract infection | Infections and infestations | Wilate Treatment | 1 | 12 | 1 |
| other | Respiratory tract infection viral | Infections and infestations | Wilate Treatment | 1 | 12 | 1 |
1 to 10 of 32Source ClinicalTrials.gov
See all 32 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Wilate Treatment | 0 | 12 |
| Total, other adverse events | Wilate Treatment | 10 | 12 |
| Total, serious adverse events | Wilate Treatment | 4 | 12 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04953884
GET https://conductscience.org/api/trials/v1/NCT04953884/registered?page=2