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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

Must not have

Medical history of a thromboembolic event

Immunomodulatory drug treatment within 30 days prior to signing informed consent

Timeline

Screening 3 weeks

Treatment Varies

Follow Up throughout the study duration of approximately 6.5 years

Awards & highlights

Pivotal Trial

No Placebo-Only Group

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.

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.

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.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have had a blood clot in the past.

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I haven't taken any immune system modifying drugs in the last 30 days.

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I am HIV positive with a CD4 count below 200.

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I have been diagnosed with a serious liver or kidney disease.

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I have been diagnosed with a bleeding disorder.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ throughout the study duration of approximately 6.5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~throughout the study duration of approximately 6.5 years

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

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Hemostatic Efficacy

Secondary study objectives

Elective or Emergency Surgery: Assessment of Hemostatic Efficacy - Immediately After Surgery

Elective or Emergency Surgery: Overall Assessment of Hemostatic Efficacy 24 Hours After the Last Perioperative Infusion of rVWF

Hemostasis, Surgical

+2 more

Side effects data

From 2020 Phase 3 trial • 29 Patients • NCT02973087

31%

Headache

15%

Joint injury

15%

Ear infection

Nasopharyngitis

Fall

Urinary tract infection

Alanine aminotransferase increased

Injection site irritation

Multiple injuries

Supraventricular tachycardia

Ventricular extrasystoles

Herpes virus infection

Aspartate aminotransferase increased

Gout

Toothache

Iron deficiency

Vitamin B12 deficiency

Arthralgia

Seronegative arthritis

Sleep disorder

Purpura

100%

80%

60%

40%

20%

Study treatment Arm

Prior On-demand Participants

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Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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