Trial · NCT01410227
Pharmacokinetics, Safety and Efficacy of Recombinant Von Willebrand Factor (rVWF) in the Treatment of Bleeding Episodes in Von Willebrand Disease (VWD)
NCT01410227, Phase 3, Completed, Baxalta now part of Shire; results first posted 2016-04-21
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Pharmacokinetics, Safety and Efficacy of Recombinant Von Willebrand Factor (rVWF) in the Treatment of Bleeding Episodes in Von Willebrand Disease (VWD). Trials, record NCT01410227. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01410227
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Pharmacokinetics, Safety and Efficacy of Recombinant Von Willebrand Factor (rVWF) in the Treatment of Bleeding Episodes in Von Willebrand Disease (VWD)
- NCT number
- NCT01410227
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 49 participants, actual
- Lead sponsor
- Baxalta now part of Shire
- Conditions
- Von Willebrand Disease
- Interventions
- Recombinant von Willebrand factor (rVWF), Placebo, Recombinant factor VIIII (rFVIII)
- First posted
- 2011-08-05
- Last updated
- 2021-05-19
- Other registries' IDs
- eudract:2010-024108-84
- Results first posted
- 2016-04-21
Source ClinicalTrials.gov
Registered outcomes67
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants With Treatment Success for Treated Bleeding Episodes | For 12 months after first infusion of rVWF:rFVIII or rVWF | Treatment success was defined as the extent of control of bleeding episodes (BEs) using a mean efficacy rating score of \<2.5 for a participant's BEs treated with study product (recombinant von Willebrand Factor \[rVWF\] with or without recombinant factor VIII \[rFVIII\]) during the study period. Scores used: Excellent = 1 - actual infusions ≤ estimated number of infusions required to treat BE; no additional VWF required (all BEs); Good = 2 - \>1-2 infusions (minor/moderate BEs) or \<1.5 infusions (major BEs) greater than estimated required to control BE; no additional VWF required (all BEs); Moderate = 3 ≥ 3 infusions (minor/moderate BEs) or ≥ 1.5 infusions (major BEs) greater than estimated required to control BE; no additional VWF required (all BEs); None = 4 - severe uncontrolled bleeding or intensity of bleeding not changed; additional VWF required. Included participants with available primary efficacy rating (prospective-excluding gastrointestinal bleeds) in the Full Analysis Set. |
| Secondary | Percentage of Treated Bleeding Episodes With an Efficacy Rating of "Excellent" or "Good" | For 12 months after first infusion of rVWF:rFVIII or rVWF | Efficacy ratings "excellent" or "good" for the control of bleeding episodes (BEs) with study product (recombinant von Willebrand Factor \[rVWF\] with or without recombinant factor VIII \[rFVIII\]) are defined as follows: Excellent - actual infusions ≤ estimated number of infusions required to treat BE; no additional von Willebrand Factor (VWF) required (all BEs); Good - \>1-2 infusions (minor/moderate BEs) or \<1.5 infusions (major BEs) greater than estimated required to control BE; no additional VWF required (all BEs). The data set included prospectively estimated BEs treated with study product with an available efficacy rating from participants in the Full Analysis Set |
| Secondary | Percentage of Treated Bleeding Episodes With an Efficacy Rating of "Excellent" or "Good", Excluding Gastrointestinal Bleeds | For 12 months after first infusion of rVWF:rFVIII or rVWF | Efficacy ratings of "excellent" or "good" for the control of bleeding episodes (BEs) with study product (recombinant von Willebrand Factor \[rVWF\] with or without recombinant factor VIII \[rFVIII\]) are defined as follows: Excellent - actual infusions ≤ estimated number of infusions required to treat BE; no additional von Willebrand Factor (VWF) required (all BEs); Good - \>1-2 infusions (minor/moderate BEs) or \<1.5 infusions (major BEs) greater than estimated required to control BE; no additional VWF required (all BEs). The data set included prospectively estimated BEs excluding gastrointestinal (GI) bleeds treated with study product with an available efficacy rating from participants in the Full Analysis Set. |
| Secondary | Number of Infusions of rVWF:rFVIII and/or rVWF Per Bleeding Episode | For 12 months after first infusion of rVWF:rFVIII or rVWF | The actual number of infusions of recombinant von Willebrand factor:recombinant factor VIII (rVWF:rFVIII) and/or rVWF required to treat a bleeding episode (BE). BEs were to be initially treated with an infusion of rVWF:rFVIII and subsequently with rVWF with or without rFVIII, based on FVIII levels, if available. In cases, where no FVIII levels were available, the individual participant's PK data was used to determine the rFVIII dose. The data set included prospectively estimated BEs treated with study product with an available efficacy rating from participants in the Full Analysis Set. |
| Secondary | Number of Units of rVWF:rFVIII and/or rVWF Per Bleeding Episode | For 12 months after first infusion of rVWF:rFVIII or rVWF | The number of units is provided as the actual dose \[IU/kg\] of recombinant von Willebrand factor:recombinant factor VIII (rVWF:rFVIII) and/or rVWF required to treat a bleeding episode (BE). BEs were to be initially treated with an infusion of rVWF:rFVIII and subsequently with rVWF with or without rFVIII, based on FVIII levels, if available. In cases, where no FVIII levels were available, the individual participant's PK data was used to determine the rFVIII dose. The data set included prospectively estimated BEs treated with study product of known lot number with an available efficacy rating from participants in the Full Analysis Set. |
| Secondary | Percentage of Participants Who Develop Inhibitory Antibodies to FVIII | For 12 months after first infusion of rVWF:rFVIII or rVWF | Development of neutralizing antibodies (inhibitors) to factor VIII (FVIII) was assessed by the Nijmegen modification of the Bethesda assay. Positive FVIII inhibitor tests were defined as ≥ 0.4 Bethesda units/mL (BU/mL) by the Nijmegen-modified Bethesda assay that is confirmed by a second test performed on an independent sample obtained 2-4 weeks following the first test. Category title includes number of participants \[N\] who provided data for the category. |
| Secondary | Percentage of Participants Who Develop Inhibitory Antibodies to VWF | After signing informed consent until 12 months after first infusion of rVWF:rFVIII or rVWF | Neutralizing antibodies (inhibitors) to Von Willebrand Factor Ristocetin cofactor (VWF:RCo), VWF collagen binding (VWF:CB) and VWF Factor VIII binding (VWF:FVIIIB) activities were measured using Nijmegen modification of the Bethesda assay. One Bethesda Unit (BU) is thereby defined as the amount of inhibitor that decreased the measured activity in the assays to 50% of that of the negative control samples. The assays were validated using human plasma samples from two type 3 VWD patients with low (1-2 BU/mL) and high (\~10 BU/mL) titer inhibitors and plasma samples from non-human primates immunized with human rVWF (\>100 BU/mL). Category title includes number of participants \[N\] who provided data for the category. |
| Secondary | Percentage of Participants Who Develop Binding Antibodies to VWF | After signing informed consent until 12 months after first infusion of rVWF:rFVIII or rVWF | 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). Category title includes number of participants \[N\] who provided data for the category. |
| Secondary | Percentage of Participants Who Develop Binding Antibodies to CHO | After signing informed consent until 12 months after first infusion of rVWF:rFVIII or rVWF | The presence of total binding anti-CHO antibodies was determined by measuring total immunoglobulin (Ig) antibodies (IgG, IgA, IgM) against Chinese Hamster Ovary (CHO) protein using an enzyme-linked immunosorbent assay (ELISA). Category title includes number of participants \[N\] who provided data for the category. |
| Secondary | Percentage of Participants Who Develop Binding Antibodies to rFurin | After signing informed consent until 12 months after first infusion of rVWF:rFVIII or rVWF | The presence of total binding anti-rFurin antibodies was determined by measuring total immunoglobulin (Ig) antibodies (IgG, IgA, IgM) against rFurin protein using an enzyme-linked immunosorbent assay (ELISA). Category title includes number of participants \[N\] who provided data for the category. |
1 to 10 of 67Source ClinicalTrials.gov
See all 67 registered outcomesOutcomes67
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants With Treatment Success for Treated Bleeding Episodes | For 12 months after first infusion of rVWF:rFVIII or rVWF | Percent of participants | NUMBER | |
| SECONDARY | Percentage of Treated Bleeding Episodes With an Efficacy Rating of Excellent or Good | For 12 months after first infusion of rVWF:rFVIII or rVWF | Percent of bleeding episodes | NUMBER | |
| SECONDARY | Percentage of Treated Bleeding Episodes With an Efficacy Rating of Excellent or Good, Excluding Gastrointestinal Bleeds | For 12 months after first infusion of rVWF:rFVIII or rVWF | Percent of bleeding episodes | GEOMETRIC_MEAN | |
| SECONDARY | Number of Infusions of rVWF:rFVIII and/or rVWF Per Bleeding Episode | For 12 months after first infusion of rVWF:rFVIII or rVWF | Number of infusions | MEDIAN | |
| SECONDARY | Number of Units of rVWF:rFVIII and/or rVWF Per Bleeding Episode | For 12 months after first infusion of rVWF:rFVIII or rVWF | IU/kg | MEDIAN | |
| SECONDARY | Percentage of Participants Who Develop Inhibitory Antibodies to FVIII | For 12 months after first infusion of rVWF:rFVIII or rVWF | Percent of participants | NUMBER | |
| SECONDARY | Percentage of Participants Who Develop Inhibitory Antibodies to VWF | After signing informed consent until 12 months after first infusion of rVWF:rFVIII or rVWF | Percent of participants | NUMBER | |
| SECONDARY | Percentage of Participants Who Develop Binding Antibodies to VWF | After signing informed consent until 12 months after first infusion of rVWF:rFVIII or rVWF | Percent of participants | NUMBER | |
| SECONDARY | Percentage of Participants Who Develop Binding Antibodies to CHO | After signing informed consent until 12 months after first infusion of rVWF:rFVIII or rVWF | Percent of participants | NUMBER | |
| SECONDARY | Percentage of Participants Who Develop Binding Antibodies to rFurin | After signing informed consent until 12 months after first infusion of rVWF:rFVIII or rVWF | Percent of participants | NUMBER |
1 to 10 of 67Source ClinicalTrials.gov
See all 67 outcomesResults by group148
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants With Treatment Success for Treated Bleeding Episodes | Full Analysis Set | 100.0 | 90% Confidence Interval 84.7 to 100.0 | Percent of participants | |
| Percentage of Treated Bleeding Episodes With an Efficacy Rating of Excellent or Good | Full Analysis Set | 100.0 | 90% Confidence Interval 97.7 to 100.0 | Percent of bleeding episodes | |
| Percentage of Treated Bleeding Episodes With an Efficacy Rating of Excellent or Good, Excluding Gastrointestinal Bleeds | Full Analysis Set | 100.0 | 90% Confidence Interval 97.7 to 100.0 | Percent of bleeding episodes | |
| Number of Infusions of rVWF:rFVIII and/or rVWF Per Bleeding Episode | Full Analysis Set | 1.0 | 90% Confidence Interval 1.0 to 1.0 | Number of infusions | |
| Number of Units of rVWF:rFVIII and/or rVWF Per Bleeding Episode | Full Analysis Set | 48.2 | 90% Confidence Interval 43.9 to 50.2 | IU/kg | |
| Percentage of Participants Who Develop Inhibitory Antibodies to FVIII | Safety Analysis Set | Before 1st treatment with study product [N=37] | 0 | Percent of participants | |
| Percentage of Participants Who Develop Inhibitory Antibodies to FVIII | Safety Analysis Set | During 1st treatment until study end [N=27] | 0 | Percent of participants | |
| Percentage of Participants Who Develop Inhibitory Antibodies to FVIII | Safety Analysis Set | At final study visit [N=24] | 0 | Percent of participants | |
| Percentage of Participants Who Develop Inhibitory Antibodies to VWF | Safety Analysis Set | Before 1st treatment with study product [N=37] | 0 | Percent of participants | |
| Percentage of Participants Who Develop Inhibitory Antibodies to VWF | Safety Analysis Set | During 1st treatment until study end [N=27] | 0 | Percent of participants |
1 to 10 of 148Source ClinicalTrials.gov
See all 148 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Arm 1: PK50 + Treatment | 8 | |
| Overall Study | STARTED | Arm 2: PK50 Only | 8 | |
| Overall Study | STARTED | Arm 3: PK80 + Treatment | 15 | |
| Overall Study | STARTED | Arm 4: Treatment Only | 6 | |
| Overall Study | COMPLETED | Arm 1: PK50 + Treatment | 4 | |
| Overall Study | COMPLETED | Arm 2: PK50 Only | 8 | |
| Overall Study | COMPLETED | Arm 3: PK80 + Treatment | 13 | |
| Overall Study | COMPLETED | Arm 4: Treatment Only | 5 | |
| Overall Study | NOT COMPLETED | Arm 1: PK50 + Treatment | 4 | |
| Overall Study | NOT COMPLETED | Arm 2: PK50 Only | 0 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events21
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 | Anaemia | Blood and lymphatic system disorders | Safety Analysis Set | 2 | 37 | 2 |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | Safety Analysis Set | 3 | 37 | 4 |
| other | Vertigo | Ear and labyrinth disorders | Safety Analysis Set | 2 | 37 | 3 |
| other | Nausea | Gastrointestinal disorders | Safety Analysis Set | 2 | 37 | 2 |
| other | Vomiting | Gastrointestinal disorders | Safety Analysis Set | 3 | 37 | 4 |
| other | Infusion site paraesthesia | General disorders | Safety Analysis Set | 2 | 37 | 2 |
| other | Upper respiratory tract infection | Infections and infestations | Safety Analysis Set | 3 | 37 | 5 |
| other | Contusion | Injury, poisoning and procedural complications | Safety Analysis Set | 2 | 37 | 2 |
| other | Laceration | Injury, poisoning and procedural complications | Safety Analysis Set | 2 | 37 | 4 |
| other | Arthralgia | Musculoskeletal and connective tissue disorders | Safety Analysis Set | 3 | 37 | 16 |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Safety Analysis Set | ||
| Total, other adverse events | Safety Analysis Set | 14 | 37 |
| Total, serious adverse events | Safety Analysis Set | 7 | 37 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 26239086 | RESULT |
Source PubMed
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.
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