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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Wilate treatment | Experimental | PK: Single dose of 80 IU/kg body weight (BW). Prophylactic treatment: 30-50 IU/kg BW administered 2-3 times per week at recommended dose over 12 months. Minor haemorrhage: loading dose 30-50 IU/kg BW followed by a maintenance dose of 30-40 IU/kg BW every 12-24 hours to achieve von Willebrand factor activity (VWF:Ac) and Factor VIII coagulant activity (FVIII:C) trough levels of >30%. Major haemorrhage: loading dose 50-80 IU/kg BW then maintenance dose of 30-50 IU/kg BW every 12-24 hours to achieve VWF:Ac and FVIII:C trough levels of >50%. Minor surgery: loading dose of 40-60 IU/kg BW then maintenance dose of 20-30 IU/kg BW every 12-24 hours for up to 3 days, to achieve VWF:Ac peak levels of 50% after loading dose and trough levels >30% during maintenance. Major surgery: loading dose of 60-80 IU/kg BW then a maintenance dose of 30-40 IU/kg BW every 12-24 hours for up to 6 days or longer, to achieve VWF:Ac peak levels of 100% after loading dose and trough levels >50% during maintenance |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Wilate | Drug | Wilate is a plasma-derived, stable, highly purified, double virus inactivated concentrate of freeze-dried active VWF and factor VIII (FVIII) prepared from cryoprecipitate and intended for the treatment of patients with VWD and/or haemophilia A |
| Measure | Description | Time Frame |
|---|---|---|
| Total Annualised Bleeding Rate (TABR) During Prophylactic Treatment With Wilate. | 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. | During 12 months of prophylactic treatment |
| Measure | Description | Time Frame |
|---|---|---|
| Area Under the Curve (AUC) of Wilate for VWF:Ac (VWF:RCo) Over Time | Mean AUC of VWF:Ac after PK injection of wilate as measured by chromogenic assay. | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate |
| Area Under the Curve (AUC) of Wilate for FVIII (OS) Over Time |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Tulane University | New Orleans | Louisiana | 70118 | United States | ||
| University Hospital Ostrava Department for Pediatric Medicine |
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| ID | Title | Description |
|---|---|---|
| FG000 | Wilate Treatment | PK: Single dose of 80 IU/kg BW. Prophylactic treatment: 30-50 IU/kg BW administered 2-3 times per week at the recommended dose of over 12 months. Minor haemorrhage: loading dose 30-50 IU/kg BW followed by a maintenance dose of 30-40 IU/kg BW every 12-24 hours to achieve VWF:Ac and FVIII:C trough levels of >30%. Major haemorrhage: loading dose 50-80 IU/kg BW followed by a maintenance dose of 30-50 IU/kg BW every 12-24 hours to achieve VWF:Ac and FVIII:C trough levels of >50%. Minor surgery: loading dose of 40-60 IU/kg BW followed by a maintenance dose of 20-30 IU/kg BW every 12-24 hours for up to 3 days, to achieve VWF:Ac peak levels of 50% after loading dose and trough levels >30% during maintenance. Major surgery: loading dose of 60-80 IU/kg BW followed by a maintenance dose of 30-40 IU/kg BW every 12-24 hours for up to 6 days or longer, to achieve VWF:Ac peak levels of 100% after loading dose and trough levels >50% during maintenance Wilate: Wilate is a plasma-derived, stable, highly purified, double virus inactivated concentrate of freeze-dried active VWF and factor VIII (FVIII) prepared from cryoprecipitate and intended for the treatment of patients with von Willebrand disease (VWD) and/or haemophilia A |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Wilate Treatment | PK: Single dose of 80 IU/kg body weight (BW). Prophylactic treatment: 30-50 IU/kg BW administered 2-3 times per week at the recommended dose of over 12 months. Minor haemorrhage: loading dose 30-50 IU/kg BW followed by a maintenance dose of 30-40 IU/kg BW every 12-24 hours to achieve von Willebrand factor activity (VWF:Ac) and FVIII:C trough levels of >30%. Major haemorrhage: loading dose 50-80 IU/kg BW followed by a maintenance dose of 30-50 IU/kg BW every 12-24 hours to achieve VWF:Ac and FVIII:C trough levels of >50%. Minor surgery: loading dose of 40-60 IU/kg BW followed by a maintenance dose of 20-30 IU/kg BW every 12-24 hours for up to 3 days, to achieve VWF:Ac peak levels of 50% after loading dose and trough levels >30% during maintenance. Major surgery: loading dose of 60-80 IU/kg BW followed by a maintenance dose of 30-40 IU/kg BW every 12-24 hours for up to 6 days or longer, to achieve VWF:Ac peak levels of 100% after loading dose and trough levels >50% during maintenance wilate: wilate is a plasma-derived, stable, highly purified, double virus inactivated concentrate of freeze-dried active VWF and factor VIII (FVIII) prepared from cryoprecipitate and intended for the treatment of patients with von Willebrand disease (VWD) and/or haemophilia A |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Patients VWD type was confirmed via central laboratory testing using screening samples from patients prior to confirmed enrollment in the study. This included the VWF Ristocetin Cofactor (VWF:RCo) assay to measure VWF activity and VWF multimeter analysis that included assessment / quantification of VWF antigen levels. Patients with a quantitative defect (i.e., low VWF) were diagnosed with Type 3 and those with a * qualitative* defect (lack of functional VWF) Type 2. |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Total Annualised Bleeding Rate (TABR) During Prophylactic Treatment With Wilate. | 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. | The analysis population consists of all patients in the full analysis set (FAS) | Posted | Mean | Standard Deviation | Annualized number of bleeding episodes | During 12 months of prophylactic treatment |
|
From the first administration of study drug and continuing until study a completion which was up to 12 months +2 weeks.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Wilate Treatment | PK: Single dose of 80 IU/kg body weight (BW). Prophylactic treatment: 30-50 IU/kg BW administered 2-3 times per week at the recommended dose of over 12 months. Minor haemorrhage: loading dose 30-50 IU/kg BW followed by a maintenance dose of 30-40 IU/kg BW every 12-24 hours to achieve von Willebrand factor activity (VWF:Ac) and FVIII:C trough levels of >30%. Major haemorrhage: loading dose 50-80 IU/kg BW followed by a maintenance dose of 30-50 IU/kg BW every 12-24 hours to achieve VWF:Ac and FVIII:C trough levels of >50%. Minor surgery: loading dose of 40-60 IU/kg BW followed by a maintenance dose of 20-30 IU/kg BW every 12-24 hours for up to 3 days, to achieve VWF:Ac peak levels of 50% after loading dose and trough levels >30% during maintenance. Major surgery: loading dose of 60-80 IU/kg BW followed by a maintenance dose of 30-40 IU/kg BW every 12-24 hours for up to 6 days or longer, to achieve VWF:Ac peak levels of 100% after loading dose and trough levels >50% during maintenance wilate: wilate is a plasma-derived, stable, highly purified, double virus inactivated concentrate of freeze-dried active VWF and factor VIII (FVIII) prepared from cryoprecipitate and intended for the treatment of patients with von Willebrand disease (VWD) and/or haemophilia A |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pyrexia | General disorders | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Bronchitis | Infections and infestations | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Sigurd Knaub | Octapharma AG | +41 55 451 21 89 | Sigurd.Knaub@octapharma.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Dec 5, 2022 | Aug 28, 2025 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Feb 12, 2025 | Aug 28, 2025 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D014842 | von Willebrand Diseases |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| ID | Term |
|---|---|
| D014841 | von Willebrand Factor |
| ID | Term |
|---|---|
| D001779 | Blood Coagulation Factors |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
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Mean AUC of FVIII after PK injection of wilate as measured by chromogenic assay. |
| At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate |
| AUC Normalised for the Administered Dose (AUCnorm) of Wilate for VWF:Ac (VWF:RCo) Over Time | 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. | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate |
| AUC Normalised for the Administered Dose (AUCnorm) of Wilate for FVIII:C (OS) Over Time | 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. | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate |
| Clearance (CL) of Wilate for VWF:Ac (VWF:RCo) Over Time | 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. | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate |
| Clearance (CL) of Wilate for FVIII:C (OS) Over Time | 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. | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate |
| Maximum Plasma Concentration (Cmax) of Wilate for VWF:Ac (VWF:RCo) Over Time | 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. | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate |
| Maximum Plasma Concentration (Cmax) of Wilate for FVIII:C (OS) Over Time | 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. | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate |
| Mean Residence Time (MRT) of Wilate for VWF:Ac (VWF:RCo) Over Time | 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. | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate |
| Mean Residence Time (MRT) of Wilate for FVIII:C (OS) Over Time | Mean residence time (MRT) of FVIII:C, measured by one-stage assay [OS]) after PK injection of wilate, calculated as the average time a FVIII:C molecule remains in the body, derived from the ratio of area under the first moment curve (AUMC) to AUC. | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate |
| In Vivo Half-life (T1/2) of Wilate for VWF:Ac (VWF:RCo) Over Time | Mean in vivo half-life (T1/2) VWF:Ac, measured by ristocetin cofactor assay [VWF:RCo]) after PK injection of wilate, calculated as the time required for the plasma concentration to decrease by half during the terminal elimination phase. | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate |
| In Vivo Half-life (T1/2) of Wilate for FVIII:C (OS) Over Time | Mean in vivo half-life (T1/2) of FVIII:C, measured by one-stage assay [OS]) after PK injection of wilate, calculated as the time required for the plasma concentration to decrease by half during the terminal elimination phase. | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate |
| Time to Reach Maximum Plasma Concentration (Tmax) of Wilate for VWF:Ac (VWF:RCo) Over Time | Mean time to reach maximum plasma concentration (Tmax) of VWF:Ac, measured by ristocetin cofactor assay [VWF:RCo]) after PK injection of wilate, defined as the time from dosing to the observed peak plasma concentration. | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate |
| Time to Reach Maximum Plasma Concentration (Tmax) of Wilate for FVIII:C (OS) Over Time | Mean time to reach maximum plasma concentration (Tmax) of FVIII:C, measured by one-stage assay [OS]) after PK injection of wilate, defined as the time from dosing to the observed peak plasma concentration. | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate |
| Volume of Distribution (Vd) of Wilate for VWF:Ac (VWF:RCo) Over Time | Mean volume of distribution at steady state (Vd) of VWF:Ac, measured by ristocetin cofactor assay [VWF:RCo]) after PK injection of wilate, calculated as clearance (CL) multiplied by mean residence time (MRT). | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate |
| Volume of Distribution (Vd) of Wilate for FVIII:C (OS) Over Time | Mean volume of distribution at steady state (Vd) of FVIII:C, measured by one-stage assay [OS]) after PK injection of wilate, calculated as clearance (CL) multiplied by mean residence time (MRT). | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate |
| Incremental In-vivo Recovery (IVR) of Wilate for VWF:Ac (VWF:RCo) Over Time | Mean incremental in vivo recovery (IVR) of VWF:Ac, measured by ristocetin cofactor assay [VWF:RCo]) after PK injection of wilate, calculated as the increase in plasma VWF:Ac concentration per IU/kg of wilate administered. | Measures were taken at the following timepoints: baseline and 1, 2, 3, 6, 9, and 12 months of treatment with IVR values reported at baseline and 12 months. |
| Incremental In-vivo Recovery (IVR) of Wilate for FVIII:C (OS) Over Time | Mean incremental in vivo recovery (IVR) of FVIII:C, measured by one-stage assay [OS]) after PK injection of wilate, calculated as the increase in plasma FVIII:C concentration per IU/kg of wilate administered. | Measures were taken at the following timepoints: baseline and 1, 2, 3, 6, 9, and 12 months of treatment with IVR values reported at baseline and 12 months. |
| Efficacy of Wilate Measured by the Proportion of BEs Successfully Treated With Wilate | Proportion of BEs successfully treated with wilate, as assessed using a predefined 4-point ordinal haemostatic efficacy scale. Each BE was rated at the end of treatment as "excellent", "good", "moderate", or "none" based on the time to bleeding cessation and the dose required. A BE is considered successfully treated if the efficacy rating is "excellent" (bleeding stopped within 3 days for minor bleeds, within 7 days for major bleeds, or within 10 days for gastrointestinal bleeds) or "good" (bleeding stopped, but time and/or dose slightly exceeded expectations). The outcome is reported as the percentage of BEs with a rating of "excellent" or "good" out of all treated BEs | Up to 12 months of treatment |
| 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 | Overall efficacy of wilate in perioperative prophylaxis against excessive bleeding, as assessed at the end of the postoperative period by the responsible treating investigator using a predefined 4-point ordinal haemostatic efficacy scale. The assessment is based on the presence or absence of postoperative bleeding or oozing, the need for additional dosing, and the ability to control bleeding with wilate. Excellent: No postoperative bleeding or oozing not due to surgical complications; all postoperative bleeding due to complications controlled with wilate as anticipated for the procedure. Good: No postoperative bleeding or oozing not due to surgical complications; control of postoperative bleeding due to complications required increased dosing or additional injections of wilate not originally anticipated. Moderate: Some postoperative bleeding or oozing not due to surgical complications; control required increased dosing or additional injections of wilate not originally anticipated | Up to 12 months of treatment |
| Consumption of Wilate for Prophylactic Treatment | Mean dose of wilate administered for routine prophylactic treatment, reported as (a) mean dose per injection (IU/kg) and (b) mean dose per week (IU/kg). Dose per injection is calculated as the total amount of wilate (IU) administered per injection divided by the patient's body weight (kg). Dose per week was calculated as the total amount of wilate (IU) administered for prophylaxis in a week, divided by the patient's body weight (kg). Both measures were averaged across all prophylactic infusions during the study period | Up to 12 months of treatment |
| Consumption of Wilate for the Treatment of BEs (On-demand Treatment) | Mean dose of wilate administered for the on-demand treatment of BEs, reported as (a) mean dose per BE (IU/kg) and (b) mean dose per injection (IU/kg). Dose per BE is calculated as the total amount of wilate (IU) administered per BE treatment divided by the patient's body weight (kg). Dose per injection is calculated as the total amount of wilate (IU) administered for BE treatment in a week, divided by the patient's body weight (kg). Both measures are averaged across all treated BEs during the study period | Up to 12 months of treatment |
| Consumption of Wilate During Surgical Prophylaxis | Mean dose of wilate administered for surgical prophylaxis, reported as dose per exposure day (ED) in IU/kg. Dose per exposure day is calculated as the total amount of wilate (IU) administered for surgical prophylaxis on each day, divided by the patient's body weight (kg). The measure is averaged across all exposure days during the perioperative period | Up to 12 months of treatment |
| Number of Participants With Detectable Inhibitory Antibodies Against VWF and/or FVIII | Number of patients with detectable inhibitory antibodies to von Willebrand factor (VWF) and factor VIII (FVIII) during the study testing. Inhibitor testing is performed at baseline, every 3 months, and at any time if inhibitor development is suspected. | Up to 12 months of treatment |
| Number of Participants With Detectable Thromboembolic Events | Incidence of thromboembolic events, defined as the proportion of patients who experience one or more thromboembolic events (such as deep vein thrombosis, pulmonary embolism, or other clinically relevant thromboses) during the study period. Thromboembolic events are monitored and recorded as adverse events throughout the study, and are confirmed by clinical assessment and/or diagnostic imaging as appropriate. The outcome is reported as the number and percentage of patients with at least one thromboembolic event during the study | Up to 12 months of treatment |
| Change in Haemophilia Joint Health Score | Change from baseline in the Haemophilia Joint Health Score (HJHS) total score, assessed at baseline and at the end of the study (12 months). The HJHS is a validated clinical tool used to evaluate joint health in patients with bleeding disorders, measured over a scale of 0-124, with higher scores indicating worse joint health. This score is comprised of the assessments of 6 joints according to 8 criteria (each joint is scored from 0 to 20, giving a possible assessment range for all joints of 0 to 120) plus the Global Gait Score (measured from 0 to 4). The outcome is reported as the mean change in total HJHS score from baseline to study completion for each participant. An increase (positive value) indicates an increase in HJHS over the course of the study and worsening of joint health. A decrease (negative value) reflects a reduction in HJHS over the study duration and improvements in joint health. Assessments are performed by trained investigators using the standard HJHS protocol. | At baseline and at 12 months of treatment |
| Ostrava |
| Czechia |
| University Hospital Motol, Department of Paediatric Haematology and Oncology | Prague | Czechia |
| Gerinnungszentrum Rhein-Ruhr Ambulanz und Fachlaboratorium für Gerinnungserkrankungen/Hämophilie | Duisburg | Germany |
| IMSP Mother and Child Institute | Chisinau | Moldova |
| PHI University Clinic for Child Diseases | Skopje | North Macedonia |
| FSBI National Research Medical Center of Pediatric Hematology, Oncology and Immunology | Moscow | Russia |
| Morozovskaya Children's Hospital | Moscow | Russia |
| Danylo Halytsky Lviv National Medical University, Communal Institution of Lviv Regional Council "Western Ukrainian Specialized Children's Medical Centre" | Lviv | Ukraine |
| Mean |
| Standard Deviation |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| Blood Group | Count of Participants | Participants |
|
| VWD type | Count of Participants | Participants |
|
| VWF inhibitor history | Count of Participants | Participants |
|
| FVIII inhibitor history | Count of Participants | Participants |
|
| Family history of VWD | Count of Participants | Participants |
|
TABR was assessed in the FAS.
Prophylactic treatment: 30-50 IU/kg BW administered 2-3 times per week at the recommended dose of over 12 months.
wilate: wilate is a plasma-derived, stable, highly purified, double virus inactivated concentrate of freeze-dried active VWF and factor VIII (FVIII) prepared from cryoprecipitate and intended for the treatment of patients with von Willebrand disease (VWD) and/or haemophilia A
|
|
| Secondary | Area Under the Curve (AUC) of Wilate for VWF:Ac (VWF:RCo) Over Time | Mean AUC of VWF:Ac after PK injection of wilate as measured by chromogenic assay. | The analysis population consists of all patients in the PK per protocol population (PK-PP). | Posted | Mean | Standard Deviation | h*IU/dL | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate |
|
|
|
| Secondary | Area Under the Curve (AUC) of Wilate for FVIII (OS) Over Time | Mean AUC of FVIII after PK injection of wilate as measured by chromogenic assay. | Of the 10 patients in the PK per protocol population (PK-PP), evaluable data for FVIII measurements was available for only 8 patients. | Posted | Mean | Standard Deviation | h*IU/dL | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate |
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|
|
| Secondary | AUC Normalised for the Administered Dose (AUCnorm) of Wilate for VWF:Ac (VWF:RCo) Over Time | 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. | The analysis population consists of all 10 patients in the PK per protocol population (PK-PP). | Posted | Mean | Standard Deviation | h*kg/dL | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate |
|
|
|
| Secondary | AUC Normalised for the Administered Dose (AUCnorm) of Wilate for FVIII:C (OS) Over Time | 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. | Of the 10 patients in the PK per protocol population (PK-PP), evaluable data for FVIII measurements was available for only 8 patients. | Posted | Mean | Standard Deviation | h*kg/dL | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate |
|
|
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| Secondary | Clearance (CL) of Wilate for VWF:Ac (VWF:RCo) Over Time | 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. | The analysis population consists of all 10 patients in the PK per protocol population (PK-PP). | Posted | Mean | Standard Deviation | dL/h/kg | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate |
|
|
|
| Secondary | Clearance (CL) of Wilate for FVIII:C (OS) Over Time | 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. | Of the 10 patients in the PK per protocol population (PK-PP), evaluable data for FVIII measurements was available for only 8 patients. | Posted | Mean | Standard Deviation | dL/h/kg | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate |
|
|
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| Secondary | Maximum Plasma Concentration (Cmax) of Wilate for VWF:Ac (VWF:RCo) Over Time | 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. | The analysis population consists of all 10 patients in the PK per protocol population (PK-PP). | Posted | Mean | Standard Deviation | IU/dL | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate |
|
|
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| Secondary | Maximum Plasma Concentration (Cmax) of Wilate for FVIII:C (OS) Over Time | 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. | Of the 10 patients in the PK per protocol population (PK-PP), evaluable data for FVIII measurements was available for only 8 patients. | Posted | Mean | Standard Deviation | IU/dL | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate |
|
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| Secondary | Mean Residence Time (MRT) of Wilate for VWF:Ac (VWF:RCo) Over Time | 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. | The analysis population consists of all 10 patients in the PK per protocol population (PK-PP). | Posted | Mean | Standard Deviation | hours | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate |
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| Secondary | Mean Residence Time (MRT) of Wilate for FVIII:C (OS) Over Time | Mean residence time (MRT) of FVIII:C, measured by one-stage assay [OS]) after PK injection of wilate, calculated as the average time a FVIII:C molecule remains in the body, derived from the ratio of area under the first moment curve (AUMC) to AUC. | Of the 10 patients in the PK per protocol population (PK-PP), evaluable data for FVIII was available for only 8 patients. | Posted | Mean | Standard Deviation | hours | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate |
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| Secondary | In Vivo Half-life (T1/2) of Wilate for VWF:Ac (VWF:RCo) Over Time | Mean in vivo half-life (T1/2) VWF:Ac, measured by ristocetin cofactor assay [VWF:RCo]) after PK injection of wilate, calculated as the time required for the plasma concentration to decrease by half during the terminal elimination phase. | The analysis population consists of all 10 patients in the PK per protocol population (PK-PP). | Posted | Mean | Standard Deviation | hours | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate |
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| Secondary | In Vivo Half-life (T1/2) of Wilate for FVIII:C (OS) Over Time | Mean in vivo half-life (T1/2) of FVIII:C, measured by one-stage assay [OS]) after PK injection of wilate, calculated as the time required for the plasma concentration to decrease by half during the terminal elimination phase. | Of the 10 patients in the PK per protocol population (PK-PP), evaluable data for FVIII measurements was available for only 8 patients. | Posted | Mean | Standard Deviation | hours | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate |
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| Secondary | Time to Reach Maximum Plasma Concentration (Tmax) of Wilate for VWF:Ac (VWF:RCo) Over Time | Mean time to reach maximum plasma concentration (Tmax) of VWF:Ac, measured by ristocetin cofactor assay [VWF:RCo]) after PK injection of wilate, defined as the time from dosing to the observed peak plasma concentration. | The analysis population consists of all 10 patients in the PK per protocol population (PK-PP). | Posted | Mean | Standard Deviation | hours | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate |
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| Secondary | Time to Reach Maximum Plasma Concentration (Tmax) of Wilate for FVIII:C (OS) Over Time | Mean time to reach maximum plasma concentration (Tmax) of FVIII:C, measured by one-stage assay [OS]) after PK injection of wilate, defined as the time from dosing to the observed peak plasma concentration. | Of the 10 patients in the PK per protocol population (PK-PP), evaluable data for FVIII measurements was available for only 8 patients. | Posted | Mean | Standard Deviation | hours | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate |
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| Secondary | Volume of Distribution (Vd) of Wilate for VWF:Ac (VWF:RCo) Over Time | Mean volume of distribution at steady state (Vd) of VWF:Ac, measured by ristocetin cofactor assay [VWF:RCo]) after PK injection of wilate, calculated as clearance (CL) multiplied by mean residence time (MRT). | The analysis population consists of all 10 patients in the PK per protocol population (PK-PP). | Posted | Mean | Standard Deviation | dL/kg | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate |
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| Secondary | Volume of Distribution (Vd) of Wilate for FVIII:C (OS) Over Time | Mean volume of distribution at steady state (Vd) of FVIII:C, measured by one-stage assay [OS]) after PK injection of wilate, calculated as clearance (CL) multiplied by mean residence time (MRT). | Of the 10 patients in the PK per protocol population (PK-PP), evaluable data for FVIII measurements was available for only 8 patients. | Posted | Mean | Standard Deviation | dL/kg | At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate |
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| Secondary | Incremental In-vivo Recovery (IVR) of Wilate for VWF:Ac (VWF:RCo) Over Time | Mean incremental in vivo recovery (IVR) of VWF:Ac, measured by ristocetin cofactor assay [VWF:RCo]) after PK injection of wilate, calculated as the increase in plasma VWF:Ac concentration per IU/kg of wilate administered. | Analysis was performed on the full analysis set (FAS). At 12 months, data was available for only 11 patients as 1 patient terminated the study early due to an AE. | Posted | Mean | 95% Confidence Interval | kg/dL | Measures were taken at the following timepoints: baseline and 1, 2, 3, 6, 9, and 12 months of treatment with IVR values reported at baseline and 12 months. |
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| Secondary | Incremental In-vivo Recovery (IVR) of Wilate for FVIII:C (OS) Over Time | Mean incremental in vivo recovery (IVR) of FVIII:C, measured by one-stage assay [OS]) after PK injection of wilate, calculated as the increase in plasma FVIII:C concentration per IU/kg of wilate administered. | Analysis was performed on the full analysis set; however, at baseline there were only 9 patients with valid FVIII data. At 12 months, valid FVIII data was available for only 8 patients as 1 patient terminated the study early due to an AE. | Posted | Mean | 95% Confidence Interval | kg/dL | Measures were taken at the following timepoints: baseline and 1, 2, 3, 6, 9, and 12 months of treatment with IVR values reported at baseline and 12 months. |
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| Secondary | Efficacy of Wilate Measured by the Proportion of BEs Successfully Treated With Wilate | Proportion of BEs successfully treated with wilate, as assessed using a predefined 4-point ordinal haemostatic efficacy scale. Each BE was rated at the end of treatment as "excellent", "good", "moderate", or "none" based on the time to bleeding cessation and the dose required. A BE is considered successfully treated if the efficacy rating is "excellent" (bleeding stopped within 3 days for minor bleeds, within 7 days for major bleeds, or within 10 days for gastrointestinal bleeds) or "good" (bleeding stopped, but time and/or dose slightly exceeded expectations). The outcome is reported as the percentage of BEs with a rating of "excellent" or "good" out of all treated BEs | BEs were recorded in the FAS set. Of the 12 patients, a total of 59 BEs were recorded in 10 of the 12 patients. OF these 59 BEs, 47 were treated with wilate. | Posted | Number | number of treated BEs | Up to 12 months of treatment | BEs | BEs |
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| 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 | Overall efficacy of wilate in perioperative prophylaxis against excessive bleeding, as assessed at the end of the postoperative period by the responsible treating investigator using a predefined 4-point ordinal haemostatic efficacy scale. The assessment is based on the presence or absence of postoperative bleeding or oozing, the need for additional dosing, and the ability to control bleeding with wilate. Excellent: No postoperative bleeding or oozing not due to surgical complications; all postoperative bleeding due to complications controlled with wilate as anticipated for the procedure. Good: No postoperative bleeding or oozing not due to surgical complications; control of postoperative bleeding due to complications required increased dosing or additional injections of wilate not originally anticipated. Moderate: Some postoperative bleeding or oozing not due to surgical complications; control required increased dosing or additional injections of wilate not originally anticipated | Analysis was measured in the SURG population. | Posted | Count of Participants | Participants | Up to 12 months of treatment |
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| Secondary | Consumption of Wilate for Prophylactic Treatment | Mean dose of wilate administered for routine prophylactic treatment, reported as (a) mean dose per injection (IU/kg) and (b) mean dose per week (IU/kg). Dose per injection is calculated as the total amount of wilate (IU) administered per injection divided by the patient's body weight (kg). Dose per week was calculated as the total amount of wilate (IU) administered for prophylaxis in a week, divided by the patient's body weight (kg). Both measures were averaged across all prophylactic infusions during the study period | Analysis was measured in the FAS population. | Posted | Mean | Standard Deviation | IU/kg | Up to 12 months of treatment |
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| Secondary | Consumption of Wilate for the Treatment of BEs (On-demand Treatment) | Mean dose of wilate administered for the on-demand treatment of BEs, reported as (a) mean dose per BE (IU/kg) and (b) mean dose per injection (IU/kg). Dose per BE is calculated as the total amount of wilate (IU) administered per BE treatment divided by the patient's body weight (kg). Dose per injection is calculated as the total amount of wilate (IU) administered for BE treatment in a week, divided by the patient's body weight (kg). Both measures are averaged across all treated BEs during the study period | The study population consists of all the patients in the FAS. | Posted | Mean | Standard Deviation | IU/kg | Up to 12 months of treatment |
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| Secondary | Consumption of Wilate During Surgical Prophylaxis | Mean dose of wilate administered for surgical prophylaxis, reported as dose per exposure day (ED) in IU/kg. Dose per exposure day is calculated as the total amount of wilate (IU) administered for surgical prophylaxis on each day, divided by the patient's body weight (kg). The measure is averaged across all exposure days during the perioperative period | Analysis measured in SURG population. | Posted | Mean | Standard Deviation | IU/kg | Up to 12 months of treatment |
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| Secondary | Number of Participants With Detectable Inhibitory Antibodies Against VWF and/or FVIII | Number of patients with detectable inhibitory antibodies to von Willebrand factor (VWF) and factor VIII (FVIII) during the study testing. Inhibitor testing is performed at baseline, every 3 months, and at any time if inhibitor development is suspected. | Analysis was performed on the FAS. | Posted | Oct 2025 | Count of Participants | Participants | Up to 12 months of treatment |
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| Secondary | Number of Participants With Detectable Thromboembolic Events | Incidence of thromboembolic events, defined as the proportion of patients who experience one or more thromboembolic events (such as deep vein thrombosis, pulmonary embolism, or other clinically relevant thromboses) during the study period. Thromboembolic events are monitored and recorded as adverse events throughout the study, and are confirmed by clinical assessment and/or diagnostic imaging as appropriate. The outcome is reported as the number and percentage of patients with at least one thromboembolic event during the study | Analysis measured in the SAF. | Posted | Count of Participants | Participants | Up to 12 months of treatment |
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| Secondary | Change in Haemophilia Joint Health Score | Change from baseline in the Haemophilia Joint Health Score (HJHS) total score, assessed at baseline and at the end of the study (12 months). The HJHS is a validated clinical tool used to evaluate joint health in patients with bleeding disorders, measured over a scale of 0-124, with higher scores indicating worse joint health. This score is comprised of the assessments of 6 joints according to 8 criteria (each joint is scored from 0 to 20, giving a possible assessment range for all joints of 0 to 120) plus the Global Gait Score (measured from 0 to 4). The outcome is reported as the mean change in total HJHS score from baseline to study completion for each participant. An increase (positive value) indicates an increase in HJHS over the course of the study and worsening of joint health. A decrease (negative value) reflects a reduction in HJHS over the study duration and improvements in joint health. Assessments are performed by trained investigators using the standard HJHS protocol. | Analysis measured in FAS and of 12 patients, only 11 patients with evaluable data. | Posted | Mean | Standard Deviation | score on a scale | At baseline and at 12 months of treatment |
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| 0 |
| 12 |
| 4 |
| 12 |
| 10 |
| 12 |
| Soft tissue infection | Infections and infestations | Systematic Assessment |
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| Pneumonia | Infections and infestations | Systematic Assessment |
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| Iron deficiency anaemia | Blood and lymphatic system disorders | Systematic Assessment |
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| Conjunctivitis | Infections and infestations | Systematic Assessment |
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| Ear infection | Infections and infestations | Systematic Assessment |
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| Gastroenteritis | Infections and infestations | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | Systematic Assessment |
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| Pharyngitis | Infections and infestations | Systematic Assessment |
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| Pneumonia | Infections and infestations | Systematic Assessment |
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| Pulpitis dental | Infections and infestations | Systematic Assessment |
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| Respiratory tract infection | Infections and infestations | Systematic Assessment |
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| Respiratory tract infection viral | Infections and infestations | Systematic Assessment |
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| Rhinitis | Infections and infestations | Systematic Assessment |
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| Scarlet fever | Infections and infestations | Systematic Assessment |
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| Soft tissue infection | Infections and infestations | Systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | Systematic Assessment |
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| Iron deficiency anaemia | Blood and lymphatic system disorders | Systematic Assessment |
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| Vitamin D deficiency | Metabolism and nutrition disorders | Systematic Assessment |
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| Attention deficit hyperactivity disorder | Psychiatric disorders | Systematic Assessment |
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| Hypertension | Vascular disorders | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Dental caries | Gastrointestinal disorders | Systematic Assessment |
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| Mouth swelling | Gastrointestinal disorders | Systematic Assessment |
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| Toothache | Gastrointestinal disorders | Systematic Assessment |
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| Dermatitis contact | Skin and subcutaneous tissue disorders | Systematic Assessment |
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| Pain | General disorders | Systematic Assessment |
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| Pyrexia | General disorders | Systematic Assessment |
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| Limb injury | Injury, poisoning and procedural complications | Systematic Assessment |
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| Lip injury | Injury, poisoning and procedural complications | Systematic Assessment |
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| Skin laceration | Injury, poisoning and procedural complications | Systematic Assessment |
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Not provided
Not provided
| D020147 | Coagulation Protein Disorders |
| D001791 | Blood Platelet Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D001685 |
| Biological Factors |
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| Title | Measurements |
|---|
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| None |
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| None |
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| Change from baseline |
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