An observational study in Von Willebrand Diseases, sponsored by Octapharma. Completed at 15 sites in 8 countries. Open to participants aged 66 Months and older. Per ClinicalTrials.gov, last updated 2023-12-07.
Sponsored by Octapharma · Observational
The purpose of this study is to prospectively obtain reliable data on the bleeding and treatment pattern of patients with VWD undergoing on-demand treatment with a VWF-containing product over a period of 6 months. The data obtained will be used as a basis for historical comparisons with the bleeding and treatment pattern obtained from a clinical study on the efficacy of prophylactic treatment with a VWF/FVIII concentrate.
63 studies on the registry are indexed under Von Willebrand Diseases; 4 are open to participants now.
This study's enrollment of 56 is below the median of 102 across 31 observational studies indexed under Von Willebrand Diseases.
Browse Von Willebrand Diseases studies →Octapharma is the lead sponsor of 69 studies on the registry; 8 are open to participants now.
Of its 16 completed or terminated interventional studies of FDA-regulated products, 14 (88%) have results posted.
Counted across the registry records on this site, refreshed daily.
Overall, 55 previously treated patients aged ≥5.5 years at the time of enrolment, with type 3, type 2 (except 2N), or severe type 1 VWD will be included into this study. Of these 55 patients, at least 6 should have type 3 VWD and at least 6 should be ≥5.5 to \<16 years of age.
Patients who meet all of the following criteria are eligible for the study:
Exclusion Criteria:
Patients who meet any of the following criteria are not eligible for the study:
Patients with type 3, type 2 (except 2N), or severe type 1 VWD undergoing routine on-demand treatment with a VWF-containing product over a period of 6 months
Drug: Von Willebrand Factor-Containing Product
Active substances: VWF concentrates, VWF/FVIII concentrates, Cryoprecipitate VWF-containing products licensed in each participating country
Total Annualized Bleeding Rate (TABR)
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.
Time frame: Screening through study completion (6 months)
Spontaneous Annualized Bleeding Rate (SABR)
Spontaneous annualized bleeding rate (SABR), calculated in analogy with TABR. This includes all bleeding episodes that occurred spontaneously.
Time frame: Screening through study completion (6 months)
Consumption of the VWF-containing Product
Data on the consumption of the VWF-containing product (VWF/FVIII IU/kg per month per patient) used for routine on-demand treatment
Time frame: Screening through study completion (6 months)
Number of Bleeding Episodes (BEs) Based on a 4-point Efficacy Scale
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.
Time frame: Screening through study completion (6 months)
Number of Surgery With Successful/Unsuccessful Efficacy Assessment
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'.
Time frame: From start of surgery until end of post-operative period (within 8 days after surgery)
Quality of Life (QoL) Assessed Using the Patient-Reported Outcomes Measurement Information System (PROMIS-29)
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
Time frame: At screening visit
Quality of Life (QoL) Assessed Using a 36-Item Short Form Health Survey, Version 2 (SF-36v2)
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.
Time frame: At screening visit
Quality of Life (QoL) Assessed Using a 10-item Short Form Health Survey (SF-10)
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.
Time frame: At screening
Joint Health Status Assessed Using Hemophilia Joint Health Score (HJHS)
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.
Time frame: At screening
Menstrual Bleeding Assessed Using Pictorial Blood Loss Assessment Chart (PBAC) Score
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).
Time frame: Screening through study completion (6 months)
Number of Participants With Adverse Drug Reactions (ADRs) Associated With Use of Wilate
Noxious and unintended reactions arising from the use of Wilate will be monitored throughout the study.
Time frame: Screening through study completion (6 months)
| Milestone | Patients Undergoing On-demand Treatment With a VWF-containing Product |
|---|---|
| Started | 56 |
| Completed | 51 |
| Not completed | 5 |
| Milestone | Patients Undergoing On-demand Treatment With a VWF-containing Product |
|---|---|
| Started | 42 |
| Completed | 42 |
| Not completed | 0 |
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.
| Bleeding events per year | Patients Undergoing On-demand Treatment With a VWF-containing Product |
|---|---|
| Total Annualized Bleeding Rate (TABR) | 29.13 ± 22.925 |
Spontaneous annualized bleeding rate (SABR), calculated in analogy with TABR. This includes all bleeding episodes that occurred spontaneously.
| Bleeding events per year | Patients Undergoing On-demand Treatment With a VWF-containing Product |
|---|---|
| Spontaneous Annualized Bleeding Rate (SABR) | 21.19 ± 19.337 |
Data on the consumption of the VWF-containing product (VWF/FVIII IU/kg per month per patient) used for routine on-demand treatment
| IUs per kg per month per patient | Patients Undergoing On-demand Treatment With a VWF-containing Product |
|---|---|
| Consumption of the VWF-containing Product | 77.94 ± 67.898 |
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.
| Number of Bleeding Episodes | Patients Undergoing On-demand Treatment With a VWF-containing Product |
|---|---|
| Excellent | 450 |
| Good | 6 |
| Moderate | 1 |
| None | 0 |
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'.
| Surgeries | Patients Undergoing Treatment With a VWF-containing Product for a Surgical Procedure |
|---|---|
| Excellent | 7 |
| Good | 0 |
| Moderate | 0 |
| Poor | 0 |
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
| T-Scores | Patients Undergoing On-demand Treatment With a VWF-containing Product |
|---|---|
| Physical Function | 47.24 ± 7.947 |
| Anxiety/Fear | 53.57 ± 9.941 |
| Depression/Sadness | 49.96 ± 9.265 |
| Fatigue | 49.77 ± 11.202 |
| Sleep Disturbance | 49.81 ± 10.373 |
| Ability to Participate in Social Roles/Activities | 52.78 ± 9.817 |
| Pain Interference | 53.87 ± 11.134 |
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.
| Units on a scale | Patients Undergoing Treatment With a VWF-containing Product |
|---|---|
| Bodily Pain | 49.87 ± 27.304 |
| General Health | 49.45 ± 21.857 |
| Mental Health | 62.74 ± 16.923 |
| Physical Functioning | 64.03 ± 24.509 |
| Role Emotional | 62.63 ± 27.035 |
| Role Physical | 51.21 ± 25.435 |
| Social Functioning | 64.52 ± 24.173 |
| Vitality | 58.06 ± 18.128 |
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.
| Units on a scale | Patients Undergoing On-demand Treatment With a VWF-containing Product |
|---|---|
| Physical Summary Score | 28.60 ± 14.961 |
| Psychosocial Summary Score | 46.56 ± 9.428 |
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.
| Units on a scale | Patients Undergoing On-demand Treatment With a VWF-containing Product |
|---|---|
| Joint Health Status Assessed Using Hemophilia Joint Health Score (HJHS) | 6.76 ± 14.611 |
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).
| Units on a scale | Patients Undergoing Treatment With a VWF-containing Product |
|---|---|
| MC01 | 521.7 ± 790.96 |
| MC02 | 279.2 ± 219.69 |
| MC03 | 311.0 ± 261.98 |
| MC04 | 408.8 ± 471.92 |
| MC05 | 289.9 ± 267.53 |
| MC06 | 330.4 ± 327.02 |
| MC07 | 411.6 ± 314.99 |
| MC08 | 348.5 ± 102.53 |
| MC09 | 361.5 ± 400.93 |
Noxious and unintended reactions arising from the use of Wilate will be monitored throughout the study.
| Participants | Patients Undergoing Treatment With a VWF-containing Product |
|---|---|
| Number of Participants With Adverse Drug Reactions (ADRs) Associated With Use of Wilate | 0 |
Collected over 6 Months. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Patients Undergoing Treatment With a VWF-containing Product | 0/51 (0%) | 0/51 (0%) | 0/51 (0%) |
| Age, Customized(Participants) | Patients Undergoing On-demand Treatment With a VWF-containing Product |
|---|---|
| 6-11 years | 15 |
| 12-16 years | 16 |
| >16 years | 25 |
| Sex: Female, Male(Participants) | Patients Undergoing On-demand Treatment With a VWF-containing Product |
|---|---|
| Female | 25 |
| Male | 31 |
| Race (NIH/OMB)(Participants) | Patients Undergoing On-demand Treatment With a VWF-containing Product |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 1 |
| White | 55 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
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