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Coagulation Factor

rVWF +/− ADVATE for Von Willebrand Disease

Phase 3

Recruiting

Research Sponsored by Baxalta now part of Shire

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Additional criterion for previously untreated participants: Participant has not received prior VWF coagulation factor replacement therapy

Diagnosis of severe von Willebrand disease (VWD) with specific criteria for Type 1, Type 2A, Type 2B, Type 2N, Type 2M, or Type 3

Timeline

Screening 3 weeks

Treatment Varies

Follow Up throughout the study period of approximately 6.5 years

Awards & highlights

Study Summary

This trial will test the effectiveness of a new treatment for von Willebrand Disease in children.

Who is the study for?

Children under 18 with severe hereditary von Willebrand disease (VWD) can join this trial. They must have had at least one bleed requiring treatment in the past year and agree to follow study rules. Girls who can have babies must test negative for pregnancy and use birth control.Check my eligibility

What is being tested?

The trial is testing recombinant von Willebrand Factor (rVWF), alone or with ADVATE, to treat bleeding in kids with VWD. Over 12-18 months, doctors will give treatments based on their usual practice while checking effectiveness and side effects.See study design

What are the potential side effects?

Possible side effects of rVWF include allergic reactions due to mouse or hamster proteins in the drug, risk of blood clots, and general discomforts like headaches or nausea that are common with infusions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have never received VWF coagulation factor replacement therapy.

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I have been diagnosed with severe von Willebrand disease.

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I am younger than 18 years old.

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I am able to have children, not pregnant, and will use birth control.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ throughout the study period of approximately 6.5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~throughout the study period of approximately 6.5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and throughout the study period of approximately 6.5 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Hemostatic Efficacy

Secondary outcome measures

Area Under the Plasma Concentration/Time Curve From 0 to 96 Hours Post-Infusion (AUC0-96h) for Von Willebrand factor: ristocetin cofactor (VWF:Rco), von Willebrand factor: antigen (VWF:Ag) and von Willebrand factor: collagen binding capacity (VWF:CB)

Area Under the Plasma Concentration/Time Curve From 0 to 96 Hours Post-infusion (AUC0-96h) for Factor VIII (FVIII) Activity

Area Under the Plasma Concentration/Time Curve From Time 0 to Infinity (AUC0-inf) for VWF:RCo, VWF:Ag and VWF:CB

+22 more

Side effects data

From 2020 Phase 3 trial • 29 Patients • NCT02973087

20%

Gastroenteritis

20%

Arthralgia

10%

Increased appetite

10%

Oral herpes

10%

Pharyngitis

10%

Upper respiratory tract infection

10%

Urinary tract infection

10%

Rheumatoid arthritis

10%

Flatulence

10%

Diarrhoea

10%

Alanine aminotransferase increased

10%

Neck pain

10%

Rash pruritic

100%

80%

60%

40%

20%

Study treatment Arm

Switch Participants

Prior On-demand Participants

Trial Design

3Treatment groups

Experimental Treatment

Group I: On-demand TreatmentExperimental Treatment2 Interventions

Participants will receive recombinant von Willebrand factor (rVWF) treatment for non-surgical bleeding episodes over a 12 to 18-month period.

Group II: Emergency SurgeryExperimental Treatment2 Interventions

Within 3 hours prior to surgery. Minor surgery: infuse every 12-24 hours for at least 48 hours based on post-operative dosing. Oral Surgery: infuse at least once within first 8-12 hours post-surgery based on post-operative dosing. Major Surgery: infuse every 12-24 hours for at least first 96 hours post-surgery based on post-operative dosing.

Group III: Elective SurgeryExperimental Treatment2 Interventions

12-24 hours prior to surgery and within 3 hours of surgery. Minor surgery: infuse every 12-24 hours for at least 48 hours based on post-operative dosing. Oral Surgery: infuse at least once within first 8-12 hours post-surgery based on post-operative dosing. Major Surgery: infuse every 12-24 hours for at least first 96 hours post-surgery based on post-operative dosing.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Antihemophilic Factor (Recombinant)

2017

Completed Phase 3

~30

von Willebrand factor (Recombinant)

2017

Completed Phase 3

~30

0 / 4Eligibility criteria met

Find a Location

Who is running the clinical trial?

Baxalta now part of ShireLead Sponsor

110 Previous Clinical Trials

9,070 Total Patients Enrolled

Takeda Development Center Americas, Inc.Industry Sponsor

56 Previous Clinical Trials

12,000 Total Patients Enrolled

Study DirectorStudy DirectorTakeda

1,211 Previous Clinical Trials

489,475 Total Patients Enrolled

Media Library

Antihemophilic Factor (Recombinant) (Coagulation Factor) Clinical Trial Eligibility Overview. Trial Name: NCT02932618 — Phase 3

Von Willebrand Disease Research Study Groups: Elective Surgery, Emergency Surgery, On-demand Treatment

Von Willebrand Disease Clinical Trial 2023: Antihemophilic Factor (Recombinant) Highlights & Side Effects. Trial Name: NCT02932618 — Phase 3

Antihemophilic Factor (Recombinant) (Coagulation Factor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02932618 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Why is von Willebrand factor (Recombinant) commonly prescribed?

"von Willebrand factor (Recombinant) is most often used to help patients experiencing hemorrhaging. It can also be employed to treat von willebrand disease, bleeding episodes not related to trauma, and those brought on by injury that are associated with von willebrand disease."

Answered by AI

What other scientific tests have been done with this von Willebrand factor (Recombinant)?

"There are 9 ongoing clinical investigations into von Willebrand factor (Recombinant), 5 of which have reached Phase 3. While a few studies are taking place in Dallas, Texas, there are a total of 126 locations running trials for this medication."

Answered by AI

In how many different medical facilities is this research being conducted?

"There are 11 clinical sites running this trial, they are: Primary Children's Medical Center in Salt Lake City, Medical University of South carolina in Charleston, Nationwide Children's Hospital in Columbus. and 8 other locations."

Answered by AI

Are there any available vacancies for this clinical trial?

"Yes, as of 4/22/2022 this study is still recruiting patients that meet the eligibility criteria. The trial was initially posted on December 18th, 2017 and has been updated multiple times. There are currently 11 locations enrolling 34 participants total."

Answered by AI

Is this a new clinical trial?

"Nine active clinical trials are investigating the use of von Willebrand factor (Recombinant) across 50 cities in 19 countries. The very first study was completed in 2017 by pharmaceutical company Baxalta, which is now part of Shire. That initial trial had 34 participants and reached Phase 3 approval. In the four years since then, an additional 72 von Willebrand factor (Recombinant) studies have been completed."

Answered by AI

How many total people are allowed to participate in this clinical trial?

"The sponsor, Baxalta (now part of Shire), needs to enroll 34 patients that meet the eligibility criteria in order to move forward with the study. There are multiple planned trial sites, including Primary Children's Medical Center located in Salt Lake City, Utah and Medical University of South carolina situated in Charleston, South Carolina."

Answered by AI

Is there a risk for serious adverse reactions with von Willebrand factor (Recombinant)?

"There is some clinical data to support the efficacy of von Willebrand factor (Recombinant), and it has undergone multiple rounds of testing, so we've given it a safety score of 3."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD)

2017. "A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02932618.

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