Trial · NCT04053699
Bleeding Incidence in VWD Patients Undergoing On-Demand Treatment
NCT04053699, Completed, Octapharma; results first posted 2023-12-07
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Bleeding Incidence in VWD Patients Undergoing On-Demand Treatment. Trials, record NCT04053699. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04053699
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Bleeding Incidence in VWD Patients Undergoing On-Demand Treatment
- NCT number
- NCT04053699
- Status
- Completed
- Enrollment
- 56 participants, actual
- Lead sponsor
- Octapharma
- Conditions
- Von Willebrand Diseases
- Interventions
- Von Willebrand Factor-Containing Product
- First posted
- 2019-08-12
- Last updated
- 2023-12-07
- Results first posted
- 2023-12-07
Source ClinicalTrials.gov
Registered outcomes11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Total Annualized Bleeding Rate (TABR) | Screening through study completion (6 months) | The total annualized bleeding rate (TABR) will be calculated as the total number of spontaneous bleeds, traumatic BEs, and other BEs occurring in the time period between the start of data collection for each patient and the Study Completion Visit, divided by the duration (in years) between the start of data collection and the Study Completion Visit. Surgery periods, and BEs occurring within these surgery periods, will be excluded from the calculation of TABR. |
| Secondary | Spontaneous Annualized Bleeding Rate (SABR) | Screening through study completion (6 months) | Spontaneous annualized bleeding rate (SABR), calculated in analogy with TABR. This includes all bleeding episodes that occurred spontaneously. |
| Secondary | Consumption of the VWF-containing Product | Screening through study completion (6 months) | Data on the consumption of the VWF-containing product (VWF/FVIII IU/kg per month per patient) used for routine on-demand treatment |
| Secondary | Number of Bleeding Episodes (BEs) Based on a 4-point Efficacy Scale | Screening through study completion (6 months) | The efficacy assessment of bleeding episodes at the end of a BE was evaluated on a 4 point scale by the patient/legal guardian (together with the Investigator in case of on-site treatment) including the four items 'excellent,' 'good,' moderate,' and 'none.' The assessment was excellent when bleeding was completely stopped within 3 days in case of minor bleed, within 7 days in case of major bleed, and within 10 days in case of gastrointestinal bleed; Good when bleeding was completely stopped, but time and/or dose slightly exceeded expectations ; Moderate when bleeding could be stopped only by significantly exceeding time and/or dose expectations; and None when bleeding could be stopped only by using other VWF-containing products. |
| Secondary | Number of Surgery With Successful/Unsuccessful Efficacy Assessment | From start of surgery until end of post-operative period (within 8 days after surgery) | Effectiveness of VWF-containing product in surgical prophylaxis based on the proportion of surgeries successfully treated. Overall treatment efficacy will be assessed at the end of the postoperative period by the treating physician using predefined criteria of 'Excellent', 'Good', 'Moderate/Poor' or 'None'. |
| Secondary | Quality of Life (QoL) Assessed Using the Patient-Reported Outcomes Measurement Information System (PROMIS-29) | At screening visit | QoL assessment based on the results from the PROMIS-29 survey to monitor and evaluate the physical, mental, and social health in all patients, using a scale of a minimum score of 0 and a maximum score of 10, with higher scores representing a better outcome. The survey covers seven domains from the most relevant areas of self-reported health (depression, anxiety, physical function, pain interference, fatigue, sleep disturbance and ability to participate in social roles and activities) for the majority of people with chronic illness. PROMIS scores have a mean of 50 and standard deviation (SD) of 10 in a referent population. Full details of cut off points for each domain can be found here: https://www.healthmeasures.net/score-and-interpret/interpret-scores/promis/promis-score-cut-points |
| Secondary | Quality of Life (QoL) Assessed Using a 36-Item Short Form Health Survey, Version 2 (SF-36v2) | At screening visit | QoL assessment based on the results from the SF-36v2 questionnaire to measure functional health and well-being in patients ≥16 years. SF-36v2 ranks 8 different domains using a scale standardized with a scoring algorithm to obtain a score ranging from 0 to 100.The eight health domains include physical functioning (PF), role physical (RP), bodily pain (BP), general health problems (GH), vitality (VT), social functioning (SF), role emotional (RE) and general mental health (MH). Higher scores indicate better health status, and a mean score of 50 has been articulated as a normative value for all scales. |
| Secondary | Quality of Life (QoL) Assessed Using a 10-item Short Form Health Survey (SF-10) | At screening | QoL assessment based on the results from a SF-10 parent-completed questionnaire for patients ≥5.5 and \<16 years of age, in order to score physical and psychosocial health. SF-10 uses norm-based scoring where scales have a standardized mean value of 50 and standard deviation of 10. |
| Secondary | Joint Health Status Assessed Using Hemophilia Joint Health Score (HJHS) | At screening | Joint health status will be assessed using the Hemophilia Joint Health Score (HJHS), which has been specifically validated for the assessment of the clinical outcome in VWD. HJHS evaluates six index joints to produce a score between 0-124. Higher scores indicate worse joint health. |
| Secondary | Menstrual Bleeding Assessed Using Pictorial Blood Loss Assessment Chart (PBAC) Score | Screening through study completion (6 months) | Bleeding information from each menstrual period while in this study will be collected using the Pictorial Blood Loss Assessment Chart (PBAC). The PBAC will be provided to all female patients of child-bearing potential. The data documented in the PBAC and the investigator-calculated final score will be recorded in the eCRF. The PBAC records pad and tampon use (as either light \[1 point\], medium \[5 points\], or heavy \[10 points\] flow), clots (small \[1 point\] or large \[5 points\]), and flooding episodes (1 point each) which can be recorded as many times as necessary any day of the month. The PBAC is scored from 0 (no bleeding) onwards, with a score of \>100 defining abnormal coagulation and heavy menstrual bleeding (corresponds to \>80ml of blood loss per menstrual cycle). |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 registered outcomesOutcomes11
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Total Annualized Bleeding Rate (TABR) | Screening through study completion (6 months) | Bleeding events per year | MEAN | |
| SECONDARY | Spontaneous Annualized Bleeding Rate (SABR) | Screening through study completion (6 months) | Bleeding events per year | MEAN | |
| SECONDARY | Consumption of the VWF-containing Product | Screening through study completion (6 months) | Four subjects were not treated with VCP, for two subjects the dose administered is unknown for all infusions | IUs per kg per month per patient | MEAN |
| SECONDARY | Number of Bleeding Episodes (BEs) Based on a 4-point Efficacy Scale | Screening through study completion (6 months) | 39 patients who received VWF-containing product in the treatment of BEs | Number of Bleeding Episodes | NUMBER |
| SECONDARY | Number of Surgery With Successful/Unsuccessful Efficacy Assessment | From start of surgery until end of post-operative period (within 8 days after surgery) | 6 patients had 7 surgeries that were treated with VWF | Surgeries | NUMBER |
| SECONDARY | Quality of Life (QoL) Assessed Using the Patient-Reported Outcomes Measurement Information System (PROMIS-29) | At screening visit | T-Scores | MEAN | |
| SECONDARY | Quality of Life (QoL) Assessed Using a 36-Item Short Form Health Survey, Version 2 (SF-36v2) | At screening visit | 31 patients complete SF-36 questionnaire | Units on a scale | MEAN |
| SECONDARY | Quality of Life (QoL) Assessed Using a 10-item Short Form Health Survey (SF-10) | At screening | 25 subjects completed SF-10 questionnaire | Units on a scale | MEAN |
| SECONDARY | Joint Health Status Assessed Using Hemophilia Joint Health Score (HJHS) | At screening | 37 patients from 42 patients in the PP set completed HJHS | Units on a scale | MEAN |
| SECONDARY | Menstrual Bleeding Assessed Using Pictorial Blood Loss Assessment Chart (PBAC) Score | Screening through study completion (6 months) | All subjects of child bearing potential who experienced menstrual cycle | Units on a scale | MEAN |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 outcomesResults by group39
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Total Annualized Bleeding Rate (TABR) | Patients Undergoing On-demand Treatment With a VWF-containing Product | 29.13 | Standard Deviation 22.925 | Bleeding events per year | |
| Spontaneous Annualized Bleeding Rate (SABR) | Patients Undergoing On-demand Treatment With a VWF-containing Product | 21.19 | Standard Deviation 19.337 | Bleeding events per year | |
| Consumption of the VWF-containing Product | Patients Undergoing On-demand Treatment With a VWF-containing Product | 77.94 | Standard Deviation 67.898 | IUs per kg per month per patient | |
| Number of Bleeding Episodes (BEs) Based on a 4-point Efficacy Scale | Patients Undergoing On-demand Treatment With a VWF-containing Product | Excellent | 450 | Number of Bleeding Episodes | |
| Number of Bleeding Episodes (BEs) Based on a 4-point Efficacy Scale | Patients Undergoing On-demand Treatment With a VWF-containing Product | Good | 6 | Number of Bleeding Episodes | |
| Number of Bleeding Episodes (BEs) Based on a 4-point Efficacy Scale | Patients Undergoing On-demand Treatment With a VWF-containing Product | Moderate | 1 | Number of Bleeding Episodes | |
| Number of Bleeding Episodes (BEs) Based on a 4-point Efficacy Scale | Patients Undergoing On-demand Treatment With a VWF-containing Product | None | 0 | Number of Bleeding Episodes | |
| Number of Surgery With Successful/Unsuccessful Efficacy Assessment | Patients Undergoing Treatment With a VWF-containing Product for a Surgical Procedure | Excellent | 7 | Surgeries | |
| Number of Surgery With Successful/Unsuccessful Efficacy Assessment | Patients Undergoing Treatment With a VWF-containing Product for a Surgical Procedure | Good | 0 | Surgeries | |
| Number of Surgery With Successful/Unsuccessful Efficacy Assessment | Patients Undergoing Treatment With a VWF-containing Product for a Surgical Procedure | Moderate | 0 | Surgeries |
1 to 10 of 39Source ClinicalTrials.gov
See all 39 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Full Analysis Set | STARTED | Patients Undergoing On-demand Treatment With a VWF-containing Product | 56 | |
| Full Analysis Set | COMPLETED | Patients Undergoing On-demand Treatment With a VWF-containing Product | 51 | |
| Full Analysis Set | NOT COMPLETED | Patients Undergoing On-demand Treatment With a VWF-containing Product | 5 | |
| Per Protocol Set | STARTED | Patients Undergoing On-demand Treatment With a VWF-containing Product | 42 | |
| Per Protocol Set | COMPLETED | Patients Undergoing On-demand Treatment With a VWF-containing Product | 42 | |
| Per Protocol Set | NOT COMPLETED | Patients Undergoing On-demand Treatment With a VWF-containing Product | 0 |
Source ClinicalTrials.gov
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Patients Undergoing Treatment With a VWF-containing Product | 0 | 51 |
| Total, other adverse events | Patients Undergoing Treatment With a VWF-containing Product | 0 | 51 |
| Total, serious adverse events | Patients Undergoing Treatment With a VWF-containing Product | 0 | 51 |
Source ClinicalTrials.gov
Papers8
The papers the trial lists on ClinicalTrials.gov.
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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