rFVIII for Von Willebrand Disease

Ospedale Pediatrico Bambino Gesù, Roma, Italy
Von Willebrand Disease+1 More ConditionsrFVIII - Biological
Eligibility
Any Age
All Sexes

Study Summary

This trial will test the effectiveness of rVWF in preventing spontaneous bleeds in pediatric and adult patients with von Willebrand Disease.

Eligible Conditions
  • Von Willebrand Disease

Treatment Effectiveness

Phase-Based Effectiveness

2 of 3
Phase 3
This is further along than 85% of similar trials

Study Objectives

2 Primary · 52 Secondary · Reporting Duration: Throughout the study participation period, up to 3 years

Month 12
Spontaneous Annualized Bleeding Rate (ABR)
Spontaneous annualized bleeding rate (ABR)
Month 12
Overall Hemostatic Efficacy Rating
Overall hemostatic efficacy rating
Year 3
Adverse Events (AEs)/Serious Adverse Events (SAEs)
Adverse events (AEs)/serious adverse events (SAEs)
Average Number of Infusions
Average number of infusions
Categorized Spontaneous Annualized Bleeding Rate (ABR)
Categorized Weekly Number of Infusions
Categorized spontaneous annualized bleeding rate (ABR)
Categorized weekly number of infusions
Duration of transfusion-free maintenance of hemoglobin levels
Duration of transfusion-free maintenance of plasma ferritin levels
Ferritin Levels Over Time
Hypersensitivity Reactions
Hypersensitivity reactions
Number of Infusions
Number of Participants With Clinically Significant Changes in Laboratory Parameters
Number of Participants With Clinically Significant Changes in Vital Signs
Number of Participants who Achieve Transfusion-free Maintenance of Hemoglobin Levels
Number of Participants who Develop Binding Antibodies to Chinese hamster ovary (CHO) proteins
Number of Participants who Develop Binding Antibodies to Mouse Immunoglobulin G (IgG)
Number of Participants who Develop Binding Antibodies to recombinant Furin (rFurin)
Number of Participants who Develop Neutralizing Antibodies to Factor VIII (FVIII)
Number of Participants who Develop Neutralizing Antibodies to von Willebrand factor (VWF)
Number of Participants who Develop Total Binding Antibodies to Factor VIII (FVIII)
Number of Participants who Develop Total Binding Antibodies to von Willebrand factor (VWF)
Number of Participants with Clinically Significant Changes in Laboratory Parameters Reported as Adverse Events
Number of Participants with Clinically Significant Changes in Vital Signs Reported as Adverse Events
Number of infusions
Number of participants who achieve transfusion free maintenance of hemoglobin levels
Number of participants who achieve transfusion free maintenance of plasma ferritin levels
Number of participants who develop binding antibodies to CHO proteins
Number of participants who develop binding antibodies to mouse IgG
Number of participants who develop binding antibodies to rFurin
Number of participants who develop neutralizing antibodies to FVIII
Number of participants who develop neutralizing antibodies to VWF
Number of participants who develop total binding antibodies to FVIII
Number of participants who develop total binding antibodies to VWF
Spontaneous ABR by location of bleeding
Spontaneous ABR under prophylactic treatment
Spontaneous Annualized Bleeding Rate (ABR) Under Prophylactic Treatment
Hemorrhage
Thromboembolic Events
Thromboembolic events
Time to First Bleeding Event
Time to first bleeding event
Total Number of Infusions
Total Weight Adjusted Consumption of recombinant von Willebrand factor (rVWF) (vonicog alfa)
Total number of infusions
Total weight adjusted consumption of rVWF (vonicog alfa)
Weight-adjusted Consumption
Weight-adjusted consumption

Trial Safety

Phase-Based Safety

3 of 3
This is further along than 85% of similar trials

Awards & Highlights

No Placebo Group
All patients enrolled in this trial will receive the new treatment.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

Trial Design

2 Treatment Groups

Prophylaxis
1 of 2
On-Demand
1 of 2

Experimental Treatment

71 Total Participants · 2 Treatment Groups

Primary Treatment: rFVIII · No Placebo Group · Phase 3

ProphylaxisExperimental Group · 2 Interventions: rFVIII, rVWF · Intervention Types: Biological, Biological
On-DemandExperimental Group · 2 Interventions: rFVIII, rVWF · Intervention Types: Biological, Biological

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: throughout the study participation period, up to 3 years

Who is running the clinical trial?

Takeda Development Center Americas, Inc.Industry Sponsor
51 Previous Clinical Trials
11,767 Total Patients Enrolled
Baxalta now part of ShireLead Sponsor
110 Previous Clinical Trials
9,018 Total Patients Enrolled
Study DirectorStudy DirectorTakeda
1,107 Previous Clinical Trials
481,694 Total Patients Enrolled

Eligibility Criteria

Age Any Age · All Participants · 0 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:

Frequently Asked Questions

Are patients currently being recruited for this clinical trial?

"The study, which was first advertised on April 1st 2019 and last updated 13 days ago, is looking for participants." - Anonymous Online Contributor

Unverified Answer

rFVIII has been found to be safe for patients, correct?

"There is some efficacy data and multiple rounds of safety data, so rFVIII scored a 3 for safety." - Anonymous Online Contributor

Unverified Answer

What are rFVIII's primary therapeutic applications?

"rFVIII is an effective medication for treating hemorrhagic episodes, von willebrand disease, and bleeding." - Anonymous Online Contributor

Unverified Answer

At how many distinct locations is this clinical trial being conducted?

"There are a number of locations where this study is currently being conducted, these include the Cincinnati Children's Hospital Medical Center in Cincinnati, Rainbow Babies and Children's Hospital in Cleveland, as well as the Medical University of South Carolina (MUSC) in Charleston. There are also 9 other centres where this research is taking place." - Anonymous Online Contributor

Unverified Answer

What are the desired outcomes of this research?

"The primary goal of this study, which will be assessed over the course of 12 months, is to evaluate the spontaneous annualized bleeding rate (ABR). Secondary objectives include measuring the categorized weekly number of infusions and the total number of infusions during prophylactic treatment with rVWF (vonicog alfa), as well as determining the number of participants who develop neutralizing antibodies to von Willebrand factor (VWF)." - Anonymous Online Contributor

Unverified Answer

To what extent has rFVIII been studied in a clinical setting before?

"Currently, there are 9 trials for rFVIII with 5 of them in Phase 3. Although a few of the clinical studies testing rFVIII are based in Dallas, Texas, 126 research centres across the globe are working on this topic." - Anonymous Online Contributor

Unverified Answer

To the best of your knowledge, is this a novel clinical trial?

"rFVIII has been the focus of medical research since 2017. The first study, sponsored by Baxalta (now part of Shire), took place that year and involved 34 patients. After this initial Phase 3 drug approval in 2017, there have been 9 active studies conducted across 50 cities in 19 countries." - Anonymous Online Contributor

Unverified Answer

How many people are willing to participate in this research?

"That is correct, the trial detailed on clinicaltrials.gov is currently looking for 71 individuals to fill out the study at 9 different locations. The first posting was on April 1st, 2019 with the most recent update happening on April 13th, 2020." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.