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| ID | Type | Description | Link |
|---|---|---|---|
| 2018-003453-16 | EudraCT Number |
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| Name | Class |
|---|---|
| Takeda Development Center Americas, Inc. | INDUSTRY |
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The main aim of the study is to check effectiveness of rVWF (vonicog alfa) prophylaxis based on the annualized bleeding rate (ABR) of spontaneous (not related to trauma) bleeding episodes in pediatric and adult participants during the first 12 months on study treatment.
The participants will be treated with rVWF for a maximum of 3 years. Their von Willebrand Disease will be treated according to Investigational product (IP) dosing directions.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| On-Demand | Experimental | Participants will receive recombinant von Willebrand factor (rVWF) (with or without ADVATE). |
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| Prophylaxis | Experimental | Participants will receive recombinant von Willebrand factor (rVWF). |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| rVWF | Biological | Recombinant von Willebrand factor |
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| Measure | Description | Time Frame |
|---|---|---|
| Spontaneous Annualized Bleeding Rate (sABR) | sABR was derived as [number of treated bleeds] / [duration in years]. Bleeds with unknown causality were considered as spontaneous. Bleeds were categorized based on the investigator assessment of cause. sABR during the first 12 months of prophylactic treatment with rVWF (vonicog alfa) was reported. | Up to 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | An adverse event (AE): any untoward medical occurrence in a participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to medicinal product. TEAE was defined as any AE that started after the first administration of study drug in this continuation study. Serious TEAEs: any untoward medical occurrence that: 1) results in death, 2) is life-threatening, 3) requires inpatient hospitalization or prolongation of existing hospitalization, 4) results in persistent or significant disability/incapacity, 5) leads to a congenital anomaly/birth defect in the offspring of the participant or 6) is medically important. |
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Inclusion Criteria:
The participant will not be considered eligible for the study without meeting all of the criteria below.
Participants who have completed Study 071301 or Study 071102 (or participants who have completed the surgery arm treatment in Study 071102 and want to continue to receive on-demand (OD) treatment) and are willing to immediately transition into this study, must meet the following 2 criteria to be eligible for this study:
New participants (Cohort 4) who meet the above 2 and ALL the following additional criteria are eligible for this study:
- Participant has a documented diagnosis of severe von Willebrand disease (VWD) (baseline von Willebrand factor: Ristocetin cofactor (VWF:RCo) <20 International Units per deciliter [IU/dL]) with a history of requiring substitution therapy with von Willebrand factor (vWF) concentrate to control bleeding:
Diagnosis is confirmed by genetic testing and multimer analysis, documented in participant history or at screening.
Exclusion Criteria:
The participant will be excluded from the study if any of the following exclusion criteria are met.
Delay criteria Only for Cohort 4, if the participant presents with an acute bleeding episodes or acute illness (eg, influenza, flu-like syndrome, allergic rhinitis/conjunctivitis, and non-seasonal asthma) the screening visit will be postponed until the participant has recovered. For all other participants, end of study (EOS) visit for 071102 or 071301 will be completed per protocol and the completed EOS in Study 071102 or 071301 will also serve as the screening visit for this continuation study (SHP677-304).
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Arkansas Children's Hospital Research Institute | Little Rock | Arkansas | 72202 | United States | ||
| University of Colorado Health |
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| Label | URL |
|---|---|
| To obtain more information on the study, click here/on this link | View source |
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Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
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IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/ For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
A total of 38 participants with diagnosis of Von Willebrand disease were enrolled. Only participants who received treatment in study(N=35) were included in analysis. These participants received recombinant von Willebrand factor (rVWF) [vonicog alfa], 50±10 international units per kilogram (IU/kg),intravenous(IV) infusion in either prophylaxis or on demand cohorts. Some participants received supportive treatment with ADVATE for treating bleeding episodes (BEs) if deemed necessary by investigator.
Participants took part in the study at 23 investigative sites globally from 1 April 2019 to 30 January 2025.
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| ID | Title | Description |
|---|---|---|
| FG000 | Cohort 1: Prophylaxis | Adult participants who transitioned from the phase 3 prophylaxis parent study 071301 (NCT02973087) received the same prophylactic dose, 50±10 IU/kg, IV infusion of vonicog alfa twice weekly as in parent study 071301. |
| FG001 | Cohort 2: Prophylaxis |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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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 20, 2021 | Jul 4, 2025 |
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| rFVIII | Biological | Recombinant Factor VIII |
|
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| Up to 5.8 years |
| Number of Participants Based on Severity of TEAEs | An AE is defined as any untoward medical occurrence in a participant administered IP that does not necessarily have a causal relationship with the treatment. TEAE was defined as any AE that started after the first administration of study drug in this continuation study. Severity of TEAEs was determined by following definitions: Mild: No limitation of usual activities; Moderate: Some limitation of usual activities and may required therapeutic intervention; Severe: Inability to carry out usual activities with sequelae, which required therapeutic intervention. Number of participants with TEAEs based on severity of TEAEs were reported. | Up to 5.8 years |
| Number of Participants Based on Causality of TEAEs | An AE is defined as any untoward medical occurrence in a participant administered IP that does not necessarily have a causal relationship with the treatment. TEAE was defined as any AE that started after the first administration of study drug in this continuation study. A physician/investigator made the assessment of relationship to investigational product for each AE. Number of participants with TEAEs based on causality were reported. | Up to 5.8 years |
| Number of Participants With Thromboembolic Events | Thromboembolism defined as formation of a clot (thrombus) in a blood vessel that breaks loose, is carried by the blood stream and could plug another vessel. Number of participants with thromboembolic events as TEAEs of special interest were reported. | Up to 5.8 years |
| Number of Participants With Hypersensitivity Reactions | Hypersensitivity (also called hypersensitivity reaction or intolerance) defined as undesirable reactions produced by the normal immune system, including allergies and autoimmunity. Potential hypersensitivity events were identified by broad search criteria and then medically assessed. Number of participants with hypersensitivity reactions as TEAEs of special interest was calculated. | Up to 5.8 years |
| Number of Participants Who Developed Neutralizing Antibodies to Von Willebrand Factor (VWF) | Three functional VWF assays for von Willebrand factor collagen binding (VWF:CB), von Willebrand factor: Ristocetin Cofactor (VWF:RCo) and von Willebrand factor VIII B (VWF:FVIIIB) were used to test the presence of neutralizing anti-VWF antibodies. Neutralizing antibodies to VWF:RCo, VWF:CB and VWF:FVIIIB activities were measured by assays based on the Bethesda assay established for quantitative analysis of FVIII inhibitors (Nijmegen modification of the Bethesda assay). Only confirmed neutralizing anti -VWF antibodies were considered inhibitors. Number of participants who developed neutralizing antibodies to rVWF were assessed. | Up to 5.8 years |
| Number of Participants Who Developed Neutralizing Antibodies to Factor VIII (FVIII) | Three functional VWF assays for von Willebrand factor collagen binding (VWF:CB), von Willebrand factor: Ristocetin Cofactor (VWF:RCo) and von Willebrand factor VIII B (VWF:FVIIIB) were used to test the presence of neutralizing anti-VWF antibodies. Neutralizing antibodies to VWF:RCo, VWF:CB and VWF:FVIIIB activities was measured by assays based on the Bethesda assay established for quantitative analysis of FVIII inhibitors (Nijmegen modification of the Bethesda assay). Only confirmed neutralizing anti -VWF antibodies were considered inhibitors. Number of participants who developed neutralizing antibodies to FVIII were assessed. | Up to 5.8 years |
| Number of Participants Who Developed Total Binding Antibodies to Von Willebrand Factor (VWF) | The presence of total binding anti-VWF antibodies was determined by an enzyme-linked immunosorbent assay (ELISA) employing polyclonal anti-human Immunoglobulin (Ig) antibodies (IgG, IgM and IgA). Number of participants who developed of total binding antibodies to rVWF were assessed. | Up to 5.8 years |
| Number of Participants Who Developed Total Binding Antibodies to Factor VIII (FVIII) | Binding antibodies against FVIII were analyzed using a proprietary enzyme immunoassay. Number of participants who developed of total binding antibodies to FVIII were assessed. | Up to 5.8 years |
| Number of Participants Who Developed Binding Antibodies to Chinese Hamster Ovary (CHO) Proteins | Total Ig antibodies (IgG, IgA, IgM) against CHO protein were analyzed using ELISA. Number of participants who developed binding antibodies to CHO proteins were assessed. | Up to 5.8 years |
| Number of Participants Who Developed Binding Antibodies to Mouse Immunoglobulin G (IgG) | Detection and quantification of IgG antibodies originating from human plasma that were directed against mouse-IgG (HAMA: human anti- mouse antibodies) were assessed using ELISA (Medac, Hamburg, Germany). Number of participants who developed binding antibodies to Mouse IgG were assessed. | Up to 5.8 years |
| Number of Participants Who Develop Binding Antibodies to Recombinant Furin (rFurin) | Total Ig antibodies (IgG, IgA, IgM) against human furin were analyzed using ELISA. Number of participants who developed binding antibodies to rFurin were assessed. | Up to 5.8 years |
| Number of Participants With Clinically Significant Changes in Vital Signs | Vital signs included blood pressure (systolic and diastolic), pulse rate, respiratory rate and body temperature. Number of participants with clinically significant change from baseline in vital signs were assessed. | Up to 5.8 years |
| Number of Participants With Clinically Significant Changes in Laboratory Parameters | Clinical laboratory parameters included serum chemistry, hematology and urinalysis assessments. Number of participants with clinically significant change from baseline in clinical laboratory parameters were assessed. | Up to 5.8 years |
| Spontaneous Annualized Bleeding Rate (sABR) Under Prophylactic Treatment | sABR was derived as [number of treated bleeds] / [duration in years]. Bleeds with unknown causality were considered as spontaneous. Bleeds were categorized based on the investigator assessment of cause. sABR during prophylaxis treatment with rVWF (vonicog alfa) while enrolled in the study were reported. | Up to 5.8 years |
| Number of Participants Categorized Based on Weekly Number of Infusions | Categorized as ≥ 0 to < 1 infusion per week, ≥ 1 to < 2 infusions per week, ≥ 2 to < 3 infusions per week, ≥ 3 infusions per week. The number of participants categorized based on number of infusions per week during prophylactic treatment with rVWF (vonicog alfa) were reported. | Up to 5.8 years |
| Number of Participants Categorized Based on Spontaneous Annualized Bleeding Rate (sABR) | The sABR was the number of spontaneous bleeds divided by the observation period in years, where an observation period = (date of completion/termination-date of first dose+1)/365.2425. sABR was categorized based on number of BEs as 0, greater than (>) 0 through 2, >2 through 5, >5 during the prophylactic treatment with rVWF (vonicog alfa). Bleeding at multiple locations related to the same injury was counted as single BE. BEs of unknown cause were counted as spontaneous bleeds. Number of participants categorized based on sABR during prophylactic treatment with rVWF (vonicog alfa) were reported. | Up to 5.8 years |
| Time to First Bleeding Event on Prophylaxis Treatment | Time to event estimates and confidence intervals obtained from Kaplan-Meier analysis. Participants with 0 bleeds during each study period were censored at the date of the last day in that study period. | Up to 5.8 years |
| Spontaneous Annualized Bleeding Rate (sABR) by Location of Bleeding | sABR was derived as [number of treated bleeds] / [duration in years]. sABR for BEs based on location of bleeding: Skin, Muscle, Mucosal Nasal, Mucosal Oral, Joint, Gastrointestinal (GI), Menstrual/Heavy Menstrual, Venipuncture Site, Soft Tissue, Body Cavity, Hematuria, Central Nervous System (CNS) and Other, while on prophylactic treatment with rVWF (vonicog alfa) were reported. | Up to 5.8 years |
| Total Number of Infusions During Prophylactic Treatment | Total number of infusions during prophylactic treatment with rVWF (vonicog alfa) were reported. | Up to 5.8 years |
| Average Number of Infusions Per Week During Prophylactic Treatment | Average number of infusions per week during prophylactic treatment with rVWF (vonicog alfa) were reported. | Up to 5.8 years |
| Total Weight Adjusted Consumption of Recombinant Von Willebrand Factor (rVWF) (Vonicog Alfa) Per Participant During Prophylactic Treatment | For each participant, the body weight-adjusted dose (IU/kg) was derived as the number of units of rVWF infused (IU) divided by the last available body weight (kilogram [kg]) prior to the infusion. Total weight adjusted consumption of rVWF (vonicog alfa) per participant during prophylactic treatment with rVWF (vonicog alfa) was reported as International Units per kilogram (IU/kg). | Up to 5.8 years |
| Number of Participants Who Achieved Transfusion-free Maintenance of Hemoglobin Levels | Transfusion free maintenance of hemoglobin levels during prophylactic treatment with rVWF (vonicog alfa) were reported. | Up to 5.8 years |
| Change From Baseline in Ferritin Levels Over Time | Change from baseline in ferritin levels over time during prophylactic treatment with rVWF (vonicog alfa) were reported. Baseline Ferritin lab assay for rollover participants in cohorts 1, 2 and 3 were not mandatory per protocol at the Screening Visit of this study as the results from End of Study (EOS) visit of parent studies were expected to be utilized for rollover cohorts 1-3. However, many participants in cohort 1 did not have this data collected at EOS Visit of parent study 071301 and no participant in cohorts 2 and 3 had this data collected at EOS Visit of parent studies 071301 and 071102. | Up to 5.8 years |
| Overall Hemostatic Efficacy Rating | Overall Hemostatic Efficacy Rating at resolution of bleed with respect to treatment of BEs for initial 12 months of study in OD cohorts. Hemostatic efficacy for treatment of BEs was rated on 4-point Likert scale as:excellent=full relief of pain&cessation of objective signs of bleeding after single infusion,no additional infusion is required for control of bleeding&administration of further infusion to maintain hemostasis would not affect scoring;good=definite pain relief&/or improvement in signs of bleeding after single infusion,possibly requires >2 infusions for complete resolution&administration of further infusion to maintain hemostasis would not affect scoring;fair=probable&/or slight relief of pain&slight improvement in signs of bleeding after single infusion,required multiple infusions for complete resolution;none=no improvement of signs/symptoms or conditions worsen.Missing=number of unique BEs without any overall hemostatic efficacy rating at resolution of breakthrough BE. | Initial 12 months of study |
| Number of Infusions of Vonicog Alfa | Number of infusions of rVWF (vonicog alfa) utilized to treat BEs during OD treatment while enrolled in the study were reported. | Up to 5.8 years |
| Number of Infusions of ADVATE | Number of infusions of ADVATE (rFVIII, octocog alfa) utilized to treat BEs during OD treatment while enrolled in the study were reported. | Up to 5.8 years |
| Weight-adjusted Consumption of Vonicog Alfa Per Bleeding Episode | Weight-adjusted consumption (IU/kg) was derived as the total units infused (IU) divided by the last available body weight (kg) prior to the infusion. Weight-adjusted consumption of rVWF (vonicog alfa) per bleeding episode during OD treatment while enrolled in the study were reported. | Up to 5.8 years |
| Weight-adjusted Consumption of ADVATE Per Bleeding Episode | Weight-adjusted consumption (IU/kg) was derived as the total units infused (IU) divided by the last available body weight (kg) prior to the infusion. Weight-adjusted consumption of ADVATE (rFVIII, octocog alfa) per bleeding episode during OD treatment while enrolled in the study were reported. | Up to 5.8 years |
| Aurora |
| Colorado |
| 80045 |
| United States |
| University of Florida College of Medicine | Gainesville | Florida | 32610 | United States |
| Indiana Hemophilia and Thrombosis Center | Indianapolis | Indiana | 46260 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| Rainbow Babies and Children's Hospital | Cleveland | Ohio | 44106 | United States |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | United States |
| Medical University of South Carolina (MUSC) | Charleston | South Carolina | 29425 | United States |
| AKH - Medizinische Universität Wien | Vienna | Austria |
| Hopital Cardiologique - CHU Lille | Lille | Nord | France |
| Hôpital Necker - Enfants Malades | Paris | Paris | 75015 | France |
| Groupement Hospitalier Est- Hôpital Louis Pradel | Bron | 69677 | France |
| Groupe Hospitalier Pellegrin - Hôpital Pellegrin | Gironde | France |
| Groupement Hospitalier Sud - Hôpital Bicêtre | Le Kremlin-Bicêtre | 94270 | France |
| Klinikum der Johann Wolfgang Goethe-Universitaet | Frankfurt | Germany |
| Werlhof-Institut GmbH | Hanover | Germany |
| Azienda Ospedaliera Universitaria Careggi | Florence | 50134 | Italy |
| Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico | Milan | Italy |
| Azienda Ospedaliera Pediatrica Santobono Pausillipon | Naples | Italy |
| Fondazione Policlinico Universitario Agostino Gemelli IRCCS | Roma | 00168 | Italy |
| Azienda Ospedaliera Universitaria Policlinico Umberto I - Università di Roma La Sapienza | Roma | 00185 | Italy |
| Ospedale Pediatrico Bambino Gesù | Roma | Italy |
| Erasmus Medisch Centrum | Rotterdam | 3015 AA | Netherlands |
| Erasmus Medisch Centrum | Rotterdam | 3015 CN | Netherlands |
| SAIH "Kemerovo Regional Clinical Hospital" | Kemerovo | 650066 | Russia |
| FSBI of Science "Kirov Scientific and Research Institute of Hematology and Blood Transfusion of FMBA | Kirov | 610017 | Russia |
| Hospital General Universitario de Alicante | Alicante | 03010 | Spain |
| Hospital Universitario La Paz | Madrid | 28046 | Spain |
| Hospital Universitari i Politecnic La Fe | Valencia | 46026 | Spain |
| Istanbul University Oncology Institute | Istanbul | Turkey (Türkiye) |
| Ege University Medical Faculty | Izmir | 35040 | Turkey (Türkiye) |
| Ege University Medical Faculty | Izmir | 35100 | Turkey (Türkiye) |
| Ondokuz Mayis Univ. Med. Fac. | Samsun | 55139 | Turkey (Türkiye) |
Adult participants who transitioned from parent study 071301 (NCT02973087) with no clinically significant BE for the past 6 months started this continuation study at a lower dose/frequency of vonicog alfa once weekly or twice weekly prophylactic dose, 50±10 IU/kg, IV infusion compared to the dose received (50±10 IU/kg, IV infusion, thrice weekly) in parent study 071301. |
| FG002 | Cohort 3: Prophylaxis | Adolescent participants (aged 12 to less than [<]18 years) who transitioned from the phase 3 OD and surgery parent study 071102 (NCT02932618) switched from receiving vonicog alfa OD treatment to receiving prophylactic dose of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| FG003 | Cohort 4: Prophylaxis | Newly enrolled adult and adolescent (aged 12 to <18 years) participants who switched from OD treatment with Von Willebrand Factor (VWF) products started 50±10 IU/kg, IV infusion once weekly prophylaxis with vonicog alfa in this continuation study. |
| FG004 | Cohort 5: On Demand | Pediatric participants of all ages from parent study 071102 (NCT02932618) continued receiving OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| FG005 | Cohort 6: On Demand | Adult participants from parent study 071301 (NCT02973087) switched back from prophylactic treatment in study 071301 to OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| COMPLETED |
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| NOT COMPLETED |
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The Full Analysis Set (FAS) consisted of all participants who satisfied all entry criteria and received any amount of study drug.
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| ID | Title | Description |
|---|---|---|
| BG000 | Cohort 1: Prophylaxis | Adult participants who transitioned from the phase 3 prophylaxis parent study 071301 (NCT02973087) received the same prophylactic dose, 50±10 IU/kg, IV infusion of vonicog alfa twice weekly as in parent study 071301. |
| BG001 | Cohort 2: Prophylaxis | Adult participants who transitioned from parent study 071301 (NCT02973087) with no clinically significant BE for the past 6 months started this continuation study at a lower dose/frequency of vonicog alfa once weekly or twice weekly prophylactic dose, 50±10 IU/kg, IV infusion compared to the dose received (50±10 IU/kg, IV infusion, thrice weekly) in parent study 071301. |
| BG002 | Cohort 3: Prophylaxis | Adolescent participants (aged 12 to <18 years) who transitioned from the phase 3 OD and surgery parent study 071102 (NCT02932618) switched from receiving vonicog alfa OD treatment to receiving prophylactic dose of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| BG003 | Cohort 4: Prophylaxis | Newly enrolled adult and adolescent (aged 12 to <18 years) participants who switched from OD treatment with Von Willebrand Factor (VWF) products started 50±10 IU/kg, IV infusion once weekly prophylaxis with vonicog alfa in this continuation study. |
| BG004 | Cohort 5: On Demand | Pediatric participants of all ages from parent study 071102 (NCT02932618) continued receiving OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| BG005 | Cohort 6: On Demand | Adult participants from parent study 071301 (NCT02973087) switched back from prophylactic treatment in study 071301 to OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| BG006 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Customized | 2 Participants aged 18 years old in cohort 5 were <18 years of age at the time of enrollment in the parent study 071102 (NCT02932618). They turned 18 years prior to transitioning into this study, their inclusion aligns with the study design, ensuring continuity in cohort assignment, and the age classification at the time of initial enrollment remains relevant for analytical consistency. | Count of Participants | Participants |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| 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 | |||||||||||||||||||||||||||||
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| Primary | Spontaneous Annualized Bleeding Rate (sABR) | sABR was derived as [number of treated bleeds] / [duration in years]. Bleeds with unknown causality were considered as spontaneous. Bleeds were categorized based on the investigator assessment of cause. sABR during the first 12 months of prophylactic treatment with rVWF (vonicog alfa) was reported. | The FAS consisted of all participants who satisfied all the entry criteria and received any amount of study drug. | Posted | Mean | Standard Deviation | spontaneous bleeds per year | Up to 12 months |
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| Secondary | Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | An adverse event (AE): any untoward medical occurrence in a participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to medicinal product. TEAE was defined as any AE that started after the first administration of study drug in this continuation study. Serious TEAEs: any untoward medical occurrence that: 1) results in death, 2) is life-threatening, 3) requires inpatient hospitalization or prolongation of existing hospitalization, 4) results in persistent or significant disability/incapacity, 5) leads to a congenital anomaly/birth defect in the offspring of the participant or 6) is medically important. | The Safety Analysis Set (SAS) consisted of all participants who received any amount of vonicog alfa as obtained from the study drug administration eDiary, study drug administration details eCRF, or PK infusion eCRF. | Posted | Count of Participants | Participants | Up to 5.8 years |
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| Secondary | Number of Participants Based on Severity of TEAEs | An AE is defined as any untoward medical occurrence in a participant administered IP that does not necessarily have a causal relationship with the treatment. TEAE was defined as any AE that started after the first administration of study drug in this continuation study. Severity of TEAEs was determined by following definitions: Mild: No limitation of usual activities; Moderate: Some limitation of usual activities and may required therapeutic intervention; Severe: Inability to carry out usual activities with sequelae, which required therapeutic intervention. Number of participants with TEAEs based on severity of TEAEs were reported. | The SAS consisted of all participants who received any amount of vonicog alfa as obtained from the study drug administration eDiary, study drug administration details eCRF, or PK infusion eCRF. | Posted | Count of Participants | Participants | Up to 5.8 years |
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| Secondary | Number of Participants Based on Causality of TEAEs | An AE is defined as any untoward medical occurrence in a participant administered IP that does not necessarily have a causal relationship with the treatment. TEAE was defined as any AE that started after the first administration of study drug in this continuation study. A physician/investigator made the assessment of relationship to investigational product for each AE. Number of participants with TEAEs based on causality were reported. | The SAS consisted of all participants who received any amount of vonicog alfa as obtained from the study drug administration eDiary, study drug administration details eCRF, or PK infusion eCRF. | Posted | Count of Participants | Participants | Up to 5.8 years |
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| Secondary | Number of Participants With Thromboembolic Events | Thromboembolism defined as formation of a clot (thrombus) in a blood vessel that breaks loose, is carried by the blood stream and could plug another vessel. Number of participants with thromboembolic events as TEAEs of special interest were reported. | The SAS consisted of all participants who received any amount of vonicog alfa as obtained from the study drug administration eDiary, study drug administration details eCRF, or PK infusion eCRF. | Posted | Count of Participants | Participants | Up to 5.8 years |
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| Secondary | Number of Participants With Hypersensitivity Reactions | Hypersensitivity (also called hypersensitivity reaction or intolerance) defined as undesirable reactions produced by the normal immune system, including allergies and autoimmunity. Potential hypersensitivity events were identified by broad search criteria and then medically assessed. Number of participants with hypersensitivity reactions as TEAEs of special interest was calculated. | The SAS consisted of all participants who received any amount of vonicog alfa as obtained from the study drug administration eDiary, study drug administration details eCRF, or PK infusion eCRF. | Posted | Count of Participants | Participants | Up to 5.8 years |
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| Secondary | Number of Participants Who Developed Neutralizing Antibodies to Von Willebrand Factor (VWF) | Three functional VWF assays for von Willebrand factor collagen binding (VWF:CB), von Willebrand factor: Ristocetin Cofactor (VWF:RCo) and von Willebrand factor VIII B (VWF:FVIIIB) were used to test the presence of neutralizing anti-VWF antibodies. Neutralizing antibodies to VWF:RCo, VWF:CB and VWF:FVIIIB activities were measured by assays based on the Bethesda assay established for quantitative analysis of FVIII inhibitors (Nijmegen modification of the Bethesda assay). Only confirmed neutralizing anti -VWF antibodies were considered inhibitors. Number of participants who developed neutralizing antibodies to rVWF were assessed. | The SAS consisted of all participants who received any amount of vonicog alfa as obtained from the study drug administration eDiary, study drug administration details eCRF, or PK infusion eCRF. | Posted | Count of Participants | Participants | Up to 5.8 years |
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| Secondary | Number of Participants Who Developed Neutralizing Antibodies to Factor VIII (FVIII) | Three functional VWF assays for von Willebrand factor collagen binding (VWF:CB), von Willebrand factor: Ristocetin Cofactor (VWF:RCo) and von Willebrand factor VIII B (VWF:FVIIIB) were used to test the presence of neutralizing anti-VWF antibodies. Neutralizing antibodies to VWF:RCo, VWF:CB and VWF:FVIIIB activities was measured by assays based on the Bethesda assay established for quantitative analysis of FVIII inhibitors (Nijmegen modification of the Bethesda assay). Only confirmed neutralizing anti -VWF antibodies were considered inhibitors. Number of participants who developed neutralizing antibodies to FVIII were assessed. | The SAS consisted of all participants who received any amount of vonicog alfa as obtained from the study drug administration eDiary, study drug administration details eCRF, or PK infusion eCRF. | Posted | Count of Participants | Participants | Up to 5.8 years |
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| Secondary | Number of Participants Who Developed Total Binding Antibodies to Von Willebrand Factor (VWF) | The presence of total binding anti-VWF antibodies was determined by an enzyme-linked immunosorbent assay (ELISA) employing polyclonal anti-human Immunoglobulin (Ig) antibodies (IgG, IgM and IgA). Number of participants who developed of total binding antibodies to rVWF were assessed. | The SAS consisted of all participants who received any amount of vonicog alfa as obtained from the study drug administration eDiary, study drug administration details eCRF, or PK infusion eCRF. | Posted | Count of Participants | Participants | Up to 5.8 years |
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| Secondary | Number of Participants Who Developed Total Binding Antibodies to Factor VIII (FVIII) | Binding antibodies against FVIII were analyzed using a proprietary enzyme immunoassay. Number of participants who developed of total binding antibodies to FVIII were assessed. | The SAS consisted of all participants who received any amount of vonicog alfa as obtained from the study drug administration eDiary, study drug administration details eCRF, or PK infusion eCRF. | Posted | Count of Participants | Participants | Up to 5.8 years |
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| Secondary | Number of Participants Who Developed Binding Antibodies to Chinese Hamster Ovary (CHO) Proteins | Total Ig antibodies (IgG, IgA, IgM) against CHO protein were analyzed using ELISA. Number of participants who developed binding antibodies to CHO proteins were assessed. | The SAS consisted of all participants who received any amount of vonicog alfa as obtained from the study drug administration eDiary, study drug administration details eCRF, or PK infusion eCRF. | Posted | Count of Participants | Participants | Up to 5.8 years |
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| Secondary | Number of Participants Who Developed Binding Antibodies to Mouse Immunoglobulin G (IgG) | Detection and quantification of IgG antibodies originating from human plasma that were directed against mouse-IgG (HAMA: human anti- mouse antibodies) were assessed using ELISA (Medac, Hamburg, Germany). Number of participants who developed binding antibodies to Mouse IgG were assessed. | The SAS consisted of all participants who received any amount of vonicog alfa as obtained from the study drug administration eDiary, study drug administration details eCRF, or PK infusion eCRF. | Posted | Count of Participants | Participants | Up to 5.8 years |
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| Secondary | Number of Participants Who Develop Binding Antibodies to Recombinant Furin (rFurin) | Total Ig antibodies (IgG, IgA, IgM) against human furin were analyzed using ELISA. Number of participants who developed binding antibodies to rFurin were assessed. | The SAS consisted of all participants who received any amount of vonicog alfa as obtained from the study drug administration eDiary, study drug administration details eCRF, or PK infusion eCRF. | Posted | Count of Participants | Participants | Up to 5.8 years |
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| Secondary | Number of Participants With Clinically Significant Changes in Vital Signs | Vital signs included blood pressure (systolic and diastolic), pulse rate, respiratory rate and body temperature. Number of participants with clinically significant change from baseline in vital signs were assessed. | The SAS consisted of all participants who received any amount of vonicog alfa as obtained from the study drug administration eDiary, study drug administration details eCRF, or PK infusion eCRF. | Posted | Count of Participants | Participants | Up to 5.8 years |
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| Secondary | Number of Participants With Clinically Significant Changes in Laboratory Parameters | Clinical laboratory parameters included serum chemistry, hematology and urinalysis assessments. Number of participants with clinically significant change from baseline in clinical laboratory parameters were assessed. | The SAS consisted of all participants who received any amount of vonicog alfa as obtained from the study drug administration eDiary, study drug administration details eCRF, or PK infusion eCRF. | Posted | Count of Participants | Participants | Up to 5.8 years |
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| Secondary | Spontaneous Annualized Bleeding Rate (sABR) Under Prophylactic Treatment | sABR was derived as [number of treated bleeds] / [duration in years]. Bleeds with unknown causality were considered as spontaneous. Bleeds were categorized based on the investigator assessment of cause. sABR during prophylaxis treatment with rVWF (vonicog alfa) while enrolled in the study were reported. | The FAS consisted of all participants who satisfied all the entry criteria and received any amount of study drug. | Posted | Mean | Standard Deviation | spontaneous bleeds per year | Up to 5.8 years |
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| Secondary | Number of Participants Categorized Based on Weekly Number of Infusions | Categorized as ≥ 0 to < 1 infusion per week, ≥ 1 to < 2 infusions per week, ≥ 2 to < 3 infusions per week, ≥ 3 infusions per week. The number of participants categorized based on number of infusions per week during prophylactic treatment with rVWF (vonicog alfa) were reported. | The FAS consisted of all participants who satisfied all the entry criteria and received any amount of study drug. | Posted | Count of Participants | Participants | Up to 5.8 years |
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| Secondary | Number of Participants Categorized Based on Spontaneous Annualized Bleeding Rate (sABR) | The sABR was the number of spontaneous bleeds divided by the observation period in years, where an observation period = (date of completion/termination-date of first dose+1)/365.2425. sABR was categorized based on number of BEs as 0, greater than (>) 0 through 2, >2 through 5, >5 during the prophylactic treatment with rVWF (vonicog alfa). Bleeding at multiple locations related to the same injury was counted as single BE. BEs of unknown cause were counted as spontaneous bleeds. Number of participants categorized based on sABR during prophylactic treatment with rVWF (vonicog alfa) were reported. | The FAS consisted of all participants who satisfied all entry criteria and received any amount of study drug. | Posted | Count of Participants | Participants | Up to 5.8 years |
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| Secondary | Time to First Bleeding Event on Prophylaxis Treatment | Time to event estimates and confidence intervals obtained from Kaplan-Meier analysis. Participants with 0 bleeds during each study period were censored at the date of the last day in that study period. | The FAS consisted of all participants who satisfied all entry criteria and received any amount of study drug. | Posted | Median | 95% Confidence Interval | days | Up to 5.8 years |
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| Secondary | Spontaneous Annualized Bleeding Rate (sABR) by Location of Bleeding | sABR was derived as [number of treated bleeds] / [duration in years]. sABR for BEs based on location of bleeding: Skin, Muscle, Mucosal Nasal, Mucosal Oral, Joint, Gastrointestinal (GI), Menstrual/Heavy Menstrual, Venipuncture Site, Soft Tissue, Body Cavity, Hematuria, Central Nervous System (CNS) and Other, while on prophylactic treatment with rVWF (vonicog alfa) were reported. | The FAS consisted of all participants who satisfied all entry criteria and received any amount of study drug. Number analyzed is the number of participants with data available for analyses for the specified category. | Posted | Mean | Standard Deviation | bleeds per year | Up to 5.8 years |
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| Secondary | Total Number of Infusions During Prophylactic Treatment | Total number of infusions during prophylactic treatment with rVWF (vonicog alfa) were reported. | The FAS consisted of all participants who satisfied all the entry criteria and received any amount of study drug. | Posted | Mean | Standard Deviation | infusions | Up to 5.8 years |
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| Secondary | Average Number of Infusions Per Week During Prophylactic Treatment | Average number of infusions per week during prophylactic treatment with rVWF (vonicog alfa) were reported. | The FAS consisted of all participants who satisfied all the entry criteria and received any amount of study drug. | Posted | Mean | Standard Deviation | infusions per week | Up to 5.8 years |
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| Secondary | Total Weight Adjusted Consumption of Recombinant Von Willebrand Factor (rVWF) (Vonicog Alfa) Per Participant During Prophylactic Treatment | For each participant, the body weight-adjusted dose (IU/kg) was derived as the number of units of rVWF infused (IU) divided by the last available body weight (kilogram [kg]) prior to the infusion. Total weight adjusted consumption of rVWF (vonicog alfa) per participant during prophylactic treatment with rVWF (vonicog alfa) was reported as International Units per kilogram (IU/kg). | The FAS consisted of all participants who satisfied all the entry criteria and received any amount of study drug. | Posted | Mean | Standard Deviation | IU/kg | Up to 5.8 years |
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| Secondary | Number of Participants Who Achieved Transfusion-free Maintenance of Hemoglobin Levels | Transfusion free maintenance of hemoglobin levels during prophylactic treatment with rVWF (vonicog alfa) were reported. | The FAS consisted of all participants who satisfied all the entry criteria and received any amount of study drug. | Posted | Count of Participants | Participants | Up to 5.8 years |
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| Secondary | Change From Baseline in Ferritin Levels Over Time | Change from baseline in ferritin levels over time during prophylactic treatment with rVWF (vonicog alfa) were reported. Baseline Ferritin lab assay for rollover participants in cohorts 1, 2 and 3 were not mandatory per protocol at the Screening Visit of this study as the results from End of Study (EOS) visit of parent studies were expected to be utilized for rollover cohorts 1-3. However, many participants in cohort 1 did not have this data collected at EOS Visit of parent study 071301 and no participant in cohorts 2 and 3 had this data collected at EOS Visit of parent studies 071301 and 071102. | The FAS consisted of all participants who satisfied all the entry criteria and received any amount of study drug. Overall number of participants analyzed is the number of participants with data available for analyses at Baseline. Number analyzed is the number of participants with data available for analyses at the specified timepoint. | Posted | Mean | Standard Deviation | picomole per liter (pmol/L) | Up to 5.8 years |
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| Secondary | Overall Hemostatic Efficacy Rating | Overall Hemostatic Efficacy Rating at resolution of bleed with respect to treatment of BEs for initial 12 months of study in OD cohorts. Hemostatic efficacy for treatment of BEs was rated on 4-point Likert scale as:excellent=full relief of pain&cessation of objective signs of bleeding after single infusion,no additional infusion is required for control of bleeding&administration of further infusion to maintain hemostasis would not affect scoring;good=definite pain relief&/or improvement in signs of bleeding after single infusion,possibly requires >2 infusions for complete resolution&administration of further infusion to maintain hemostasis would not affect scoring;fair=probable&/or slight relief of pain&slight improvement in signs of bleeding after single infusion,required multiple infusions for complete resolution;none=no improvement of signs/symptoms or conditions worsen.Missing=number of unique BEs without any overall hemostatic efficacy rating at resolution of breakthrough BE. | The FAS consisted of all participants who satisfied all the entry criteria and received any amount of study drug. Overall number of participants analyzed is the number of participants with treated bleeding episodes and overall number of units analyzed is the number of bleeding episodes treated in the initial 12 months of the study. | Posted | Number | bleeding episodes | Initial 12 months of study | bleeding episodes | bleeding episodes |
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| Secondary | Number of Infusions of Vonicog Alfa | Number of infusions of rVWF (vonicog alfa) utilized to treat BEs during OD treatment while enrolled in the study were reported. | The FAS consisted of all participants who satisfied all the entry criteria and received any amount of study drug. Overall number of units analyzed is the number of bleeding episodes treated with vonicog alfa. | Posted | Mean | Standard Deviation | infusions | Up to 5.8 years | bleeding episodes | bleeding episodes |
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| Secondary | Number of Infusions of ADVATE | Number of infusions of ADVATE (rFVIII, octocog alfa) utilized to treat BEs during OD treatment while enrolled in the study were reported. | The FAS consisted of all participants who satisfied all the entry criteria and received any amount of study drug. Overall number of participants analyzed is the number of participants with ADVATE-treated bleeding episodes. Overall number of units analyzed is the number of bleeding episodes treated with ADVATE. | Posted | Mean | Standard Deviation | infusions | Up to 5.8 years | bleeding episodes | bleeding episodes |
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| Secondary | Weight-adjusted Consumption of Vonicog Alfa Per Bleeding Episode | Weight-adjusted consumption (IU/kg) was derived as the total units infused (IU) divided by the last available body weight (kg) prior to the infusion. Weight-adjusted consumption of rVWF (vonicog alfa) per bleeding episode during OD treatment while enrolled in the study were reported. | The FAS consisted of all participants who satisfied all the entry criteria and received any amount of study drug. Overall number of units analyzed is the number of bleeding episodes treated with vonicog alfa and had consumption data available for analysis of this outcome measure. | Posted | Mean | Standard Deviation | IU/kg per bleeding episode | Up to 5.8 years | bleeding episodes | bleeding episodes |
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| Secondary | Weight-adjusted Consumption of ADVATE Per Bleeding Episode | Weight-adjusted consumption (IU/kg) was derived as the total units infused (IU) divided by the last available body weight (kg) prior to the infusion. Weight-adjusted consumption of ADVATE (rFVIII, octocog alfa) per bleeding episode during OD treatment while enrolled in the study were reported. | The FAS consisted of all participants who satisfied all the entry criteria and received any amount of study drug. Overall number of participants analyzed is the number of participants with ADVATE-treated bleeding episodes. Overall number of units analyzed is the number of bleeding episodes treated with ADVATE. | Posted | Mean | Standard Deviation | IU/kg per bleeding episode | Up to 5.8 years | bleeding episodes | bleeding episodes |
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Up to 5.8 years
The SAS consisted of all participants who received any amount of rVWF as obtained from the IP administration eDiary, Study Drug Administration Details eCRF or Pharmacokinetic Infusion eCRF.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Cohort 1: Prophylaxis: Adult ≥18 Years | Adult participants who transitioned from the phase 3 prophylaxis parent study 071301 (NCT02973087) received the same prophylactic dose, 50±10 IU/kg, IV infusion of vonicog alfa twice weekly as in parent study 071301. | 0 | 10 | 3 | 10 | 8 | 10 |
| EG001 | Cohort 2: Prophylaxis: Adult ≥18 Years | Adult participants who transitioned from parent study 071301 (NCT02973087) with no clinically significant BE for the past 6 months started this continuation study at a lower dose/frequency of vonicog alfa once weekly or twice weekly prophylactic dose, 50±10 IU/kg, IV infusion compared to the dose received (50±10 IU/kg, IV infusion, thrice weekly) in parent study 071301. | 0 | 1 | 0 | 1 | 1 | 1 |
| EG002 | Cohort 3: Prophylaxis: Pediatric 12 to <18 Years | Adolescent participants (aged 12 to <18 years) who transitioned from the phase 3 OD and surgery parent study 071102 (NCT02932618) switched from receiving vonicog alfa OD treatment to receiving prophylactic dose of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. | 0 | 1 | 0 | 1 | 1 | 1 |
| EG003 | Cohort 4: Prophylaxis: Pediatric 12 to <18 Years | Newly enrolled adolescent (aged 12 to <18 years) participants who switched from OD treatment with VWF products started 50±10 IU/kg, IV infusion once weekly prophylaxis with vonicog alfa in this continuation study. | 0 | 2 | 0 | 2 | 2 | 2 |
| EG004 | Cohort 4: Prophylaxis: Adult ≥18 Years | Newly enrolled adult participants who switched from OD treatment with VWF products started 50±10 IU/kg, IV infusion once weekly prophylaxis with vonicog alfa in this continuation study. | 0 | 3 | 1 | 3 | 2 | 3 |
| EG005 | Cohort 5: On Demand: Pediatric <6 Years | Pediatric participants of <6 years of age from Parent Study 071102/NCT02932618 continued receiving OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. | 0 | 3 | 2 | 3 | 3 | 3 |
| EG006 | Cohort 5: On Demand: Pediatric 6 to <12 Years | Pediatric participants of 6 to <12 years age from parent study 071102 (NCT02932618) continued receiving OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. | 0 | 5 | 1 | 5 | 5 | 5 |
| EG007 | Cohort 5: On Demand: Pediatric 12 to <18 Years | Pediatric participants of 12 to <18 years of age from parent study 071102 (NCT02932618) continued receiving OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. | 0 | 6 | 0 | 6 | 6 | 6 |
| EG008 | Cohort 5: On Demand: Adult ≥18 Years | Adult Participants from Parent Study 071102/NCT02932618 continued receiving OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. | 0 | 2 | 0 | 2 | 1 | 2 |
| EG009 | Cohort 6: On Demand: Adult ≥18 Years | Adult participants from parent study 071301 (NCT02973087) switched back from prophylactic treatment in Study 071301 to OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. | 0 | 2 | 1 | 2 | 2 | 2 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Bacteraemia | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Coronavirus infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Fibula fracture | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Haemoglobin decreased | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Medical device site extravasation | General disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Ovarian cyst ruptured | Reproductive system and breast disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Post procedural haemorrhage | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Spinal compression fracture | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Tibia fracture | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal distension | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Acute sinusitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Angular cheilitis | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Anti factor VIII antibody positive | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Arthropod sting | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Astigmatism | Eye disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Atelectasis | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Bile duct stone | Hepatobiliary disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Blood triglycerides increased | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Bone contusion | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Bundle branch block right | Cardiac disorders | MedDRA 27.1 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Cataract | Eye disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Catarrh | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Cerumen impaction | Ear and labyrinth disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Chest discomfort | General disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Chest injury | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Chills | General disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Cholecystitis | Hepatobiliary disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Conjunctivitis allergic | Eye disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Conjunctivitis bacterial | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Coronavirus infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Croup infectious | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Dental caries | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Dermatitis | Skin and subcutaneous tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Device malfunction | Product Issues | MedDRA 27.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Dysmenorrhoea | Reproductive system and breast disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Ecchymosis | Skin and subcutaneous tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Enteritis | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Eosinophilia | Blood and lymphatic system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Epicondylitis | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Faeces discoloured | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Flatulence | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Fracture displacement | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Gingivitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Haematochezia | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Haemobilia | Hepatobiliary disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Haemophilic arthropathy | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Head injury | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Hepatomegaly | Hepatobiliary disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Hypomagnesaemia | Metabolism and nutrition disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Infusion related reaction | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Infusion site rash | General disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Intermenstrual bleeding | Reproductive system and breast disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Iron deficiency | Metabolism and nutrition disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Iron deficiency anaemia | Blood and lymphatic system disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Irritable bowel syndrome | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Joint effusion | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Joint injury | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Juvenile idiopathic arthritis | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Laryngitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Limb injury | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Lymphatic malformation | Congenital, familial and genetic disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Meniscus injury | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Obesity | Metabolism and nutrition disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Onychomycosis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Otitis media acute | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Palpitations | Cardiac disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Periodontitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Perioral dermatitis | Skin and subcutaneous tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Peripheral swelling | General disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Petechiae | Skin and subcutaneous tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Road traffic accident | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| SARS-CoV-2 test positive | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Salivary hypersecretion | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Serum ferritin decreased | Investigations | MedDRA 27.1 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Skin disorder | Skin and subcutaneous tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Skin laceration | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Spinal compression fracture | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Stress fracture | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Sunburn | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Tendonitis | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Tooth deposit | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Tracheobronchitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Traumatic haematoma | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Varicose vein | Vascular disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Viral rhinitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| |
| Vitamin B12 deficiency | Metabolism and nutrition disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
| |
| Wound | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
| |
| Zinc deficiency | Metabolism and nutrition disorders | MedDRA 27.1 | Systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Takeda | +1-877-825-3327 | TrialDisclosures@takeda.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 3, 2025 | Jul 4, 2025 | SAP_001.pdf |
Not provided
| 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 |
| D020147 | Coagulation Protein Disorders |
| D001791 | Blood Platelet Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
Not provided
Not provided
| ID | Term |
|---|---|
| D005169 | Factor VIII |
| C078147 | F8 protein, human |
| ID | Term |
|---|---|
| D001779 | Blood Coagulation Factors |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D011498 | Protein Precursors |
| D001685 | Biological Factors |
Not provided
Not provided
| >=6 to <12 years |
|
| >=12 to <18 years |
|
| >=18 years |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Cohort 2: Prophylaxis |
Adult participants who transitioned from parent study 071301 (NCT02973087) with no clinically significant BE for the past 6 months started this continuation study at a lower dose/frequency of vonicog alfa once weekly or twice weekly prophylactic dose, 50±10 IU/kg, IV infusion compared to the dose received (50±10 IU/kg, IV infusion, thrice weekly) in parent study 071301. |
| OG002 | Cohort 3: Prophylaxis | Adolescent participants (aged 12 to less than [<]18 years) who transitioned from the phase 3 OD and surgery parent study 071102 (NCT02932618) switched from receiving vonicog alfa OD treatment to receiving prophylactic dose of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| OG003 | Cohort 4: Prophylaxis | Newly enrolled adult and adolescent (aged 12 to <18 years) participants who switched from OD treatment with Von Willebrand Factor (VWF) products started 50±10 IU/kg, IV infusion once weekly prophylaxis with vonicog alfa in this continuation study. |
| OG004 | Cohort 5: On Demand | Pediatric participants of all ages from parent study 071102 (NCT02932618) continued receiving OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| OG005 | Cohort 6: On Demand | Adult participants from parent study 071301 (NCT02973087) switched back from prophylactic treatment in study 071301 to OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
|
|
| OG002 | Cohort 3: Prophylaxis | Adolescent participants (aged 12 to less than [<]18 years) who transitioned from the phase 3 OD and surgery parent study 071102 (NCT02932618) switched from receiving vonicog alfa OD treatment to receiving prophylactic dose of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| OG003 | Cohort 4: Prophylaxis | Newly enrolled adult and adolescent (aged 12 to <18 years) participants who switched from OD treatment with Von Willebrand Factor (VWF) products started 50±10 IU/kg, IV infusion once weekly prophylaxis with vonicog alfa in this continuation study. |
| OG004 | Cohort 5: On Demand | Pediatric participants of all ages from parent study 071102 (NCT02932618) continued receiving OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| OG005 | Cohort 6: On Demand | Adult participants from parent study 071301 (NCT02973087) switched back from prophylactic treatment in study 071301 to OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
|
|
| OG002 | Cohort 3: Prophylaxis | Adolescent participants (aged 12 to less than [<]18 years) who transitioned from the phase 3 OD and surgery parent study 071102 (NCT02932618) switched from receiving vonicog alfa OD treatment to receiving prophylactic dose of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| OG003 | Cohort 4: Prophylaxis | Newly enrolled adult and adolescent (aged 12 to <18 years) participants who switched from OD treatment with Von Willebrand Factor (VWF) products started 50±10 IU/kg, IV infusion once weekly prophylaxis with vonicog alfa in this continuation study. |
| OG004 | Cohort 5: On Demand | Pediatric participants of all ages from parent study 071102 (NCT02932618) continued receiving OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| OG005 | Cohort 6: On Demand | Adult participants from parent study 071301 (NCT02973087) switched back from prophylactic treatment in study 071301 to OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
|
|
Adolescent participants (aged 12 to less than [<]18 years) who transitioned from the phase 3 OD and surgery parent study 071102 (NCT02932618) switched from receiving vonicog alfa OD treatment to receiving prophylactic dose of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| OG003 | Cohort 4: Prophylaxis | Newly enrolled adult and adolescent (aged 12 to <18 years) participants who switched from OD treatment with Von Willebrand Factor (VWF) products started 50±10 IU/kg, IV infusion once weekly prophylaxis with vonicog alfa in this continuation study. |
| OG004 | Cohort 5: On Demand | Pediatric participants of all ages from parent study 071102 (NCT02932618) continued receiving OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| OG005 | Cohort 6: On Demand | Adult participants from parent study 071301 (NCT02973087) switched back from prophylactic treatment in study 071301 to OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
|
|
| OG002 |
| Cohort 3: Prophylaxis |
Adolescent participants (aged 12 to less than [<]18 years) who transitioned from the phase 3 OD and surgery parent study 071102 (NCT02932618) switched from receiving vonicog alfa OD treatment to receiving prophylactic dose of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| OG003 | Cohort 4: Prophylaxis | Newly enrolled adult and adolescent (aged 12 to <18 years) participants who switched from OD treatment with Von Willebrand Factor (VWF) products started 50±10 IU/kg, IV infusion once weekly prophylaxis with vonicog alfa in this continuation study. |
| OG004 | Cohort 5: On Demand | Pediatric participants of all ages from parent study 071102 (NCT02932618) continued receiving OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| OG005 | Cohort 6: On Demand | Adult participants from parent study 071301 (NCT02973087) switched back from prophylactic treatment in study 071301 to OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
|
|
| OG002 | Cohort 3: Prophylaxis | Adolescent participants (aged 12 to less than [<]18 years) who transitioned from the phase 3 OD and surgery parent study 071102 (NCT02932618) switched from receiving vonicog alfa OD treatment to receiving prophylactic dose of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| OG003 | Cohort 4: Prophylaxis | Newly enrolled adult and adolescent (aged 12 to <18 years) participants who switched from OD treatment with Von Willebrand Factor (VWF) products started 50±10 IU/kg, IV infusion once weekly prophylaxis with vonicog alfa in this continuation study. |
| OG004 | Cohort 5: On Demand | Pediatric participants of all ages from parent study 071102 (NCT02932618) continued receiving OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| OG005 | Cohort 6: On Demand | Adult participants from parent study 071301 (NCT02973087) switched back from prophylactic treatment in study 071301 to OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
|
|
| OG002 | Cohort 3: Prophylaxis | Adolescent participants (aged 12 to less than [<]18 years) who transitioned from the phase 3 OD and surgery parent study 071102 (NCT02932618) switched from receiving vonicog alfa OD treatment to receiving prophylactic dose of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| OG003 | Cohort 4: Prophylaxis | Newly enrolled adult and adolescent (aged 12 to <18 years) participants who switched from OD treatment with Von Willebrand Factor (VWF) products started 50±10 IU/kg, IV infusion once weekly prophylaxis with vonicog alfa in this continuation study. |
| OG004 | Cohort 5: On Demand | Pediatric participants of all ages from parent study 071102 (NCT02932618) continued receiving OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| OG005 | Cohort 6: On Demand | Adult participants from parent study 071301 (NCT02973087) switched back from prophylactic treatment in study 071301 to OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
|
|
| Cohort 3: Prophylaxis |
Adolescent participants (aged 12 to less than [<]18 years) who transitioned from the phase 3 OD and surgery parent study 071102 (NCT02932618) switched from receiving vonicog alfa OD treatment to receiving prophylactic dose of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| OG003 | Cohort 4: Prophylaxis | Newly enrolled adult and adolescent (aged 12 to <18 years) participants who switched from OD treatment with Von Willebrand Factor (VWF) products started 50±10 IU/kg, IV infusion once weekly prophylaxis with vonicog alfa in this continuation study. |
| OG004 | Cohort 5: On Demand | Pediatric participants of all ages from parent study 071102 (NCT02932618) continued receiving OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| OG005 | Cohort 6: On Demand | Adult participants from parent study 071301 (NCT02973087) switched back from prophylactic treatment in study 071301 to OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
|
|
Adolescent participants (aged 12 to less than [<]18 years) who transitioned from the phase 3 OD and surgery parent study 071102 (NCT02932618) switched from receiving vonicog alfa OD treatment to receiving prophylactic dose of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study.
| OG003 | Cohort 4: Prophylaxis | Newly enrolled adult and adolescent (aged 12 to <18 years) participants who switched from OD treatment with Von Willebrand Factor (VWF) products started 50±10 IU/kg, IV infusion once weekly prophylaxis with vonicog alfa in this continuation study. |
| OG004 | Cohort 5: On Demand | Pediatric participants of all ages from parent study 071102 (NCT02932618) continued receiving OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| OG005 | Cohort 6: On Demand | Adult participants from parent study 071301 (NCT02973087) switched back from prophylactic treatment in study 071301 to OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
|
|
Adolescent participants (aged 12 to less than [<]18 years) who transitioned from the phase 3 OD and surgery parent study 071102 (NCT02932618) switched from receiving vonicog alfa OD treatment to receiving prophylactic dose of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study.
| OG003 | Cohort 4: Prophylaxis | Newly enrolled adult and adolescent (aged 12 to <18 years) participants who switched from OD treatment with Von Willebrand Factor (VWF) products started 50±10 IU/kg, IV infusion once weekly prophylaxis with vonicog alfa in this continuation study. |
| OG004 | Cohort 5: On Demand | Pediatric participants of all ages from parent study 071102 (NCT02932618) continued receiving OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| OG005 | Cohort 6: On Demand | Adult participants from parent study 071301 (NCT02973087) switched back from prophylactic treatment in study 071301 to OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
|
|
| Cohort 3: Prophylaxis |
Adolescent participants (aged 12 to less than [<]18 years) who transitioned from the phase 3 OD and surgery parent study 071102 (NCT02932618) switched from receiving vonicog alfa OD treatment to receiving prophylactic dose of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| OG003 | Cohort 4: Prophylaxis | Newly enrolled adult and adolescent (aged 12 to <18 years) participants who switched from OD treatment with Von Willebrand Factor (VWF) products started 50±10 IU/kg, IV infusion once weekly prophylaxis with vonicog alfa in this continuation study. |
| OG004 | Cohort 5: On Demand | Pediatric participants of all ages from parent study 071102 (NCT02932618) continued receiving OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| OG005 | Cohort 6: On Demand | Adult participants from parent study 071301 (NCT02973087) switched back from prophylactic treatment in study 071301 to OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
|
|
Adolescent participants (aged 12 to less than [<]18 years) who transitioned from the phase 3 OD and surgery parent study 071102 (NCT02932618) switched from receiving vonicog alfa OD treatment to receiving prophylactic dose of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study.
| OG003 | Cohort 4: Prophylaxis | Newly enrolled adult and adolescent (aged 12 to <18 years) participants who switched from OD treatment with Von Willebrand Factor (VWF) products started 50±10 IU/kg, IV infusion once weekly prophylaxis with vonicog alfa in this continuation study. |
| OG004 | Cohort 5: On Demand | Pediatric participants of all ages from parent study 071102 (NCT02932618) continued receiving OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| OG005 | Cohort 6: On Demand | Adult participants from parent study 071301 (NCT02973087) switched back from prophylactic treatment in study 071301 to OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
|
|
Adolescent participants (aged 12 to less than [<]18 years) who transitioned from the phase 3 OD and surgery parent study 071102 (NCT02932618) switched from receiving vonicog alfa OD treatment to receiving prophylactic dose of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study.
| OG003 | Cohort 4: Prophylaxis | Newly enrolled adult and adolescent (aged 12 to <18 years) participants who switched from OD treatment with Von Willebrand Factor (VWF) products started 50±10 IU/kg, IV infusion once weekly prophylaxis with vonicog alfa in this continuation study. |
| OG004 | Cohort 5: On Demand | Pediatric participants of all ages from parent study 071102 (NCT02932618) continued receiving OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| OG005 | Cohort 6: On Demand | Adult participants from parent study 071301 (NCT02973087) switched back from prophylactic treatment in study 071301 to OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
|
|
Adolescent participants (aged 12 to less than [<]18 years) who transitioned from the phase 3 OD and surgery parent study 071102 (NCT02932618) switched from receiving vonicog alfa OD treatment to receiving prophylactic dose of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| OG003 | Cohort 4: Prophylaxis | Newly enrolled adult and adolescent (aged 12 to <18 years) participants who switched from OD treatment with Von Willebrand Factor (VWF) products started 50±10 IU/kg, IV infusion once weekly prophylaxis with vonicog alfa in this continuation study. |
| OG004 | Cohort 5: On Demand | Pediatric participants of all ages from parent study 071102 (NCT02932618) continued receiving OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| OG005 | Cohort 6: On Demand | Adult participants from parent study 071301 (NCT02973087) switched back from prophylactic treatment in study 071301 to OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
|
|
Adolescent participants (aged 12 to less than [<]18 years) who transitioned from the phase 3 OD and surgery parent study 071102 (NCT02932618) switched from receiving vonicog alfa OD treatment to receiving prophylactic dose of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| OG003 | Cohort 4: Prophylaxis | Newly enrolled adult and adolescent (aged 12 to <18 years) participants who switched from OD treatment with Von Willebrand Factor (VWF) products started 50±10 IU/kg, IV infusion once weekly prophylaxis with vonicog alfa in this continuation study. |
|
|
Adolescent participants (aged 12 to less than [<]18 years) who transitioned from the phase 3 OD and surgery parent study 071102 (NCT02932618) switched from receiving vonicog alfa OD treatment to receiving prophylactic dose of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study.
| OG003 | Cohort 4: Prophylaxis | Newly enrolled adult and adolescent (aged 12 to <18 years) participants who switched from OD treatment with Von Willebrand Factor (VWF) products started 50±10 IU/kg, IV infusion once weekly prophylaxis with vonicog alfa in this continuation study. |
|
|
| OG002 | Cohort 3: Prophylaxis | Adolescent participants (aged 12 to less than [<]18 years) who transitioned from the phase 3 OD and surgery parent study 071102 (NCT02932618) switched from receiving vonicog alfa OD treatment to receiving prophylactic dose of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| OG003 | Cohort 4: Prophylaxis | Newly enrolled adult and adolescent (aged 12 to <18 years) participants who switched from OD treatment with Von Willebrand Factor (VWF) products started 50±10 IU/kg, IV infusion once weekly prophylaxis with vonicog alfa in this continuation study. |
|
|
| OG003 | Cohort 4: Prophylaxis | Newly enrolled adult and adolescent (aged 12 to <18 years) participants who switched from OD treatment with Von Willebrand Factor (VWF) products started 50±10 IU/kg, IV infusion once weekly prophylaxis with vonicog alfa in this continuation study. |
|
|
| OG002 | Cohort 3: Prophylaxis | Adolescent participants (aged 12 to less than [<]18 years) who transitioned from the phase 3 OD and surgery parent study 071102 (NCT02932618) switched from receiving vonicog alfa OD treatment to receiving prophylactic dose of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| OG003 | Cohort 4: Prophylaxis | Newly enrolled adult and adolescent (aged 12 to <18 years) participants who switched from OD treatment with Von Willebrand Factor (VWF) products started 50±10 IU/kg, IV infusion once weekly prophylaxis with vonicog alfa in this continuation study. |
|
|
| OG003 | Cohort 4: Prophylaxis | Newly enrolled adult and adolescent (aged 12 to <18 years) participants who switched from OD treatment with Von Willebrand Factor (VWF) products started 50±10 IU/kg, IV infusion once weekly prophylaxis with vonicog alfa in this continuation study. |
|
|
| OG003 | Cohort 4: Prophylaxis | Newly enrolled adult and adolescent (aged 12 to <18 years) participants who switched from OD treatment with Von Willebrand Factor (VWF) products started 50±10 IU/kg, IV infusion once weekly prophylaxis with vonicog alfa in this continuation study. |
|
|
| OG002 |
| Cohort 3: Prophylaxis |
Adolescent participants (aged 12 to less than [<]18 years) who transitioned from the phase 3 OD and surgery parent study 071102 (NCT02932618) switched from receiving vonicog alfa OD treatment to receiving prophylactic dose of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| OG003 | Cohort 4: Prophylaxis | Newly enrolled adult and adolescent (aged 12 to <18 years) participants who switched from OD treatment with Von Willebrand Factor (VWF) products started 50±10 IU/kg, IV infusion once weekly prophylaxis with vonicog alfa in this continuation study. |
|
|
| OG003 | Cohort 4: Prophylaxis | Newly enrolled adult and adolescent (aged 12 to <18 years) participants who switched from OD treatment with Von Willebrand Factor (VWF) products started 50±10 IU/kg, IV infusion once weekly prophylaxis with vonicog alfa in this continuation study. |
|
|
| OG002 | Cohort 3: Prophylaxis | Adolescent participants (aged 12 to less than [<]18 years) who transitioned from the phase 3 OD and surgery parent study 071102 (NCT02932618) switched from receiving vonicog alfa OD treatment to receiving prophylactic dose of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
| OG003 | Cohort 4: Prophylaxis | Newly enrolled adult and adolescent (aged 12 to <18 years) participants who switched from OD treatment with Von Willebrand Factor (VWF) products started 50±10 IU/kg, IV infusion once weekly prophylaxis with vonicog alfa in this continuation study. |
|
|
| OG001 | Cohort 6: On Demand | Adult participants from parent study 071301 (NCT02973087) switched back from prophylactic treatment in study 071301 to OD treatment of vonicog alfa 50±10 IU/kg, IV infusion, once weekly or twice weekly in this continuation study. |
|
|
| bleeding episodes |
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| bleeding episodes |
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| bleeding episodes |
|
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|
| bleeding episodes |
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