Trial · NCT03879135
A Study of Recombinant Von Willebrand Factor (rVWF) in Pediatric and Adult Participants With Severe Von Willebrand Disease (VWD)
NCT03879135, Phase 3, Completed, Baxalta now part of Shire; results first posted 2025-09-03
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study of Recombinant Von Willebrand Factor (rVWF) in Pediatric and Adult Participants With Severe Von Willebrand Disease (VWD). Trials, record NCT03879135. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03879135
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of Recombinant Von Willebrand Factor (rVWF) in Pediatric and Adult Participants With Severe Von Willebrand Disease (VWD)
- NCT number
- NCT03879135
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 38 participants, actual
- Lead sponsor
- Baxalta now part of Shire
- Conditions
- Von Willebrand Disease (VWD)
- Interventions
- rVWF, rFVIII
- First posted
- 2019-03-18
- Last updated
- 2025-09-03
- Other registries' IDs
- eudract:2018-003453-16
- Results first posted
- 2025-09-03
Source ClinicalTrials.gov
Registered outcomes30
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Spontaneous Annualized Bleeding Rate (sABR) | Up to 12 months | 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. |
| Secondary | Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | Up to 5.8 years | 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. |
| Secondary | Number of Participants Based on Severity of TEAEs | Up to 5.8 years | 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. |
| Secondary | Number of Participants Based on Causality of TEAEs | Up to 5.8 years | 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. |
| Secondary | Number of Participants With Thromboembolic Events | Up to 5.8 years | 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. |
| Secondary | Number of Participants With Hypersensitivity Reactions | Up to 5.8 years | 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. |
| Secondary | Number of Participants Who Developed Neutralizing Antibodies to Von Willebrand Factor (VWF) | Up to 5.8 years | 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. |
| Secondary | Number of Participants Who Developed Neutralizing Antibodies to Factor VIII (FVIII) | Up to 5.8 years | 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. |
| Secondary | Number of Participants Who Developed Total Binding Antibodies to Von Willebrand Factor (VWF) | Up to 5.8 years | 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. |
| Secondary | Number of Participants Who Developed Total Binding Antibodies to Factor VIII (FVIII) | Up to 5.8 years | Binding antibodies against FVIII were analyzed using a proprietary enzyme immunoassay. Number of participants who developed of total binding antibodies to FVIII were assessed. |
1 to 10 of 30Source ClinicalTrials.gov
See all 30 registered outcomesOutcomes30
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Spontaneous Annualized Bleeding Rate (sABR) | Up to 12 months | The FAS consisted of all participants who satisfied all the entry criteria and received any amount of study drug. | spontaneous bleeds per year | MEAN |
| SECONDARY | Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | Up to 5.8 years | 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. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants Based on Severity of TEAEs | Up to 5.8 years | 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. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants Based on Causality of TEAEs | Up to 5.8 years | 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. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Thromboembolic Events | Up to 5.8 years | 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. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Hypersensitivity Reactions | Up to 5.8 years | 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. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants Who Developed Neutralizing Antibodies to Von Willebrand Factor (VWF) | Up to 5.8 years | 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. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants Who Developed Neutralizing Antibodies to Factor VIII (FVIII) | Up to 5.8 years | 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. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants Who Developed Total Binding Antibodies to Von Willebrand Factor (VWF) | Up to 5.8 years | 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. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants Who Developed Total Binding Antibodies to Factor VIII (FVIII) | Up to 5.8 years | 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. | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 30Source ClinicalTrials.gov
See all 30 outcomesResults by group273
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Spontaneous Annualized Bleeding Rate (sABR) | Cohort 1: Prophylaxis | 1.430 | Standard Deviation 2.3894 | spontaneous bleeds per year | |
| Spontaneous Annualized Bleeding Rate (sABR) | Cohort 2: Prophylaxis | 1.040 | Standard Deviation NA | spontaneous bleeds per year | |
| Spontaneous Annualized Bleeding Rate (sABR) | Cohort 3: Prophylaxis | 0.000 | Standard Deviation NA | spontaneous bleeds per year | |
| Spontaneous Annualized Bleeding Rate (sABR) | Cohort 4: Prophylaxis | 3.022 | Standard Deviation 2.4746 | spontaneous bleeds per year | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | Cohort 1: Prophylaxis | TEAEs | 8 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | Cohort 2: Prophylaxis | TEAEs | 1 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | Cohort 3: Prophylaxis | TEAEs | 1 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | Cohort 4: Prophylaxis | TEAEs | 4 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | Cohort 5: On Demand | TEAEs | 15 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | Cohort 6: On Demand | TEAEs | 2 | Participants |
1 to 10 of 273Source ClinicalTrials.gov
See all 273 results by groupParticipant flow18
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cohort 1: Prophylaxis | 10 | |
| Overall Study | STARTED | Cohort 2: Prophylaxis | 1 | |
| Overall Study | STARTED | Cohort 3: Prophylaxis | 1 | |
| Overall Study | STARTED | Cohort 4: Prophylaxis | 5 | |
| Overall Study | STARTED | Cohort 5: On Demand | 16 | |
| Overall Study | STARTED | Cohort 6: On Demand | 2 | |
| Overall Study | COMPLETED | Cohort 1: Prophylaxis | 10 | |
| Overall Study | COMPLETED | Cohort 2: Prophylaxis | 1 | |
| Overall Study | COMPLETED | Cohort 3: Prophylaxis | 1 | |
| Overall Study | COMPLETED | Cohort 4: Prophylaxis | 3 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 participant flowAdverse events1,470
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Abdominal distension | Gastrointestinal disorders | Cohort 1: Prophylaxis: Adult ≥18 Years | 1 | 10 | |
| other | Abdominal distension | Gastrointestinal disorders | Cohort 2: Prophylaxis: Adult ≥18 Years | 0 | 1 | |
| other | Abdominal distension | Gastrointestinal disorders | Cohort 3: Prophylaxis: Pediatric 12 to <18 Years | 0 | 1 | |
| other | Abdominal distension | Gastrointestinal disorders | Cohort 4: Prophylaxis: Pediatric 12 to <18 Years | 0 | 2 | |
| other | Abdominal distension | Gastrointestinal disorders | Cohort 4: Prophylaxis: Adult ≥18 Years | 0 | 3 | |
| other | Abdominal distension | Gastrointestinal disorders | Cohort 5: On Demand: Pediatric <6 Years | 0 | 3 | |
| other | Abdominal distension | Gastrointestinal disorders | Cohort 5: On Demand: Pediatric 6 to <12 Years | 0 | 5 | |
| other | Abdominal distension | Gastrointestinal disorders | Cohort 5: On Demand: Pediatric 12 to <18 Years | 0 | 6 | |
| other | Abdominal distension | Gastrointestinal disorders | Cohort 5: On Demand: Adult ≥18 Years | 0 | 2 | |
| other | Abdominal distension | Gastrointestinal disorders | Cohort 6: On Demand: Adult ≥18 Years | 0 | 2 |
1 to 10 of 1,470Source ClinicalTrials.gov
See all 1,470 adverse eventsAdverse event totals30
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cohort 1: Prophylaxis: Adult ≥18 Years | 0 | 10 |
| Total, all-cause mortality | Cohort 2: Prophylaxis: Adult ≥18 Years | 0 | 1 |
| Total, all-cause mortality | Cohort 3: Prophylaxis: Pediatric 12 to <18 Years | 0 | 1 |
| Total, all-cause mortality | Cohort 4: Prophylaxis: Pediatric 12 to <18 Years | 0 | 2 |
| Total, all-cause mortality | Cohort 4: Prophylaxis: Adult ≥18 Years | 0 | 3 |
| Total, all-cause mortality | Cohort 5: On Demand: Pediatric <6 Years | 0 | 3 |
| Total, all-cause mortality | Cohort 5: On Demand: Pediatric 6 to <12 Years | 0 | 5 |
| Total, all-cause mortality | Cohort 5: On Demand: Pediatric 12 to <18 Years | 0 | 6 |
| Total, all-cause mortality | Cohort 5: On Demand: Adult ≥18 Years | 0 | 2 |
| Total, all-cause mortality | Cohort 6: On Demand: Adult ≥18 Years | 0 | 2 |
1 to 10 of 30Source ClinicalTrials.gov
See all 30 adverse event totalsPapers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
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The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03879135
GET https://conductscience.org/api/trials/v1/NCT03879135/registered?page=2