rVWF for Von Willebrand Disease

Recruiting at 31 trial locations

Overseen By Shire Contact

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Baxalta now part of Shire

Must be taking: VWF products

Must not be taking: Immunomodulatory drugs

Disqualifiers: Type 2N VWD, Coagulation disorders, VWF inhibitor, Factor VIII inhibitor, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 12 months

38 Participants Needed

Trial Summary

What is the purpose of this trial?

The main aim of the study is to check effectiveness of rVWF (vonicog alfa) prophylaxis based on the annualized bleeding rate (ABR) of spontaneous (not related to trauma) bleeding episodes in pediatric and adult participants during the first 12 months on study treatment. The participants will be treated with rVWF for a maximum of 3 years. Their von Willebrand Disease will be treated according to Investigational product (IP) dosing directions.

Research Team

Study Director

Principal Investigator

Takeda

Eligibility Criteria

This trial is for pediatric and adult patients with severe Von Willebrand Disease (VWD), specifically types 1, 2A, 2B, 2M, or type 3. Participants must have a history of bleeding episodes requiring treatment and be willing to follow the study protocol. Women who can bear children must test negative for pregnancy and use effective birth control during the study.

Inclusion Criteria

I have records of my bleeding episodes and treatments for the last 12-24 months.

New participants (Cohort 4) who meet the above 2 and ALL the following additional criteria are eligible for this study:

Type 1 (VWF:RCo <20 IU/dL) or,

Exclusion Criteria

I have conditions like infections or abnormal cell growth in my cervix or uterus causing heavy or irregular bleeding.

The participant is pregnant or lactating at the time of enrollment.

I have had a blood clot in the past.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive rVWF (vonicog alfa) prophylaxis to assess effectiveness based on annualized bleeding rate (ABR) during the first 12 months

12 months

Extended Treatment

Participants continue to receive rVWF treatment for a maximum of 3 years to monitor long-term safety and efficacy

Up to 3 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• rFVIII
• rVWF

Trial Overview The trial tests rVWF (vonicog alfa) as a preventive treatment over three years to reduce spontaneous bleeding in people with severe VWD. The effectiveness will be measured by counting how many bleeds participants have in the first year on this treatment.

Participant Groups

Group I: Prophylaxis Experimental Treatment 2 Interventions

Participants will receive recombinant von Willebrand factor (rVWF).

Group II: On-Demand Experimental Treatment 2 Interventions

Participants will receive recombinant von Willebrand factor (rVWF) (with or without ADVATE).

Who Is Running the Clinical Trial?

Baxalta now part of Shire

Lead Sponsor

Trials

111

Recruited

9,100+

Flemming Ornskov

Baxalta now part of Shire

Chief Executive Officer since 2013

MD, MPH

Ludwig N. Hantson

Baxalta now part of Shire

Chief Medical Officer since 2015

PhD

Takeda Development Center Americas, Inc.

Industry Sponsor

Trials

58

Recruited

10,800+