rVWF +/− ADVATE for Von Willebrand Disease

Recruiting in Palo Alto (17 mi)

+27 other locations

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Recruiting

Sponsor: Baxalta now part of Shire

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?The main aim of the study is to check effectiveness, side effects, and tolerability of recombinant von Willebrand Factor (rVWF), with or without ADVATE, in the treatment and control of nonsurgical bleeding events in pediatric participants (less than (\<)18 years of age) with severe hereditary von Willebrand disease (VWD). The participants will be treated with rVWF for 12-18 months. Their von Willebrand Disease will be treated by their doctor according to their doctor's usual clinical practice. During the study, participants will be followed up at clinics or over telephone calls.

Eligibility Criteria

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.

Inclusion Criteria

I have been diagnosed with severe von Willebrand disease.

I've had a bleeding episode in the last year despite treatment with DDAVP.

I have never received VWF coagulation factor replacement therapy.

+4 more

Exclusion Criteria

I have had a blood clot in the past.

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

Pregnant or lactating female participants at the time of informed consent

+10 more

Participant Groups

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.

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.