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Coagulation Factor

Recombinant Von Willebrand Factor for Von Willebrand Disease

Phase 3

Recruiting

Research Sponsored by Takeda

Eligibility Criteria Checklist

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

Must have

Participant is <18 years of age at the time of screening

Participant has a body mass index (BMI) >=15 but <40 kg/m^2 (if >=12 years old) or BMI >=5th and <95th percentile (if >=2 to <12 years old)

Must not have

Participant has been diagnosed with significant liver disease or liver cirrhosis classified as Child-Pugh class B or C

Participant has a progressive fatal disease and/or life expectancy of less than 15 months

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 12 months

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial aims to see if using a specific type of von Willebrand factor called rVWF can prevent bleeding in children. Children who have previously been treated with VWF or pdVWF will

Who is the study for?

This trial is for children under 18 with severe Von Willebrand Disease (VWF:RCo <20 IU/dL) who have had previous VWF treatments. They must have a history of bleeding events controlled by VWF concentrate and be recommended for prophylactic treatment. Participants need reliable medical records of their condition, meet specific body weight criteria, and girls able to bear children must use effective birth control.

What is being tested?

The study tests the effectiveness of a new medication called recombinant von Willebrand factor (rVWF) over 12 months in children with severe vWD who were previously treated with VWF products. The goal is to see if rVWF can prevent bleeding episodes better than past treatments.

What are the potential side effects?

While the side effects are not explicitly listed here, common ones associated with similar treatments may include reactions at the injection site, headaches, dizziness, nausea or allergic reactions. Specific side effects will be monitored throughout the trial.

Eligibility Criteria

Inclusion Criteria

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

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I am under 18 years old.

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My BMI is within the required range for my age group.

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I am not pregnant and agree to use effective birth control.

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I have severe von Willebrand disease and need treatment to control bleeding.

Exclusion Criteria

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

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I have severe liver disease or cirrhosis.

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My doctor expects I have 15 months or less to live due to my illness.

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I am already scheduled for surgery during the study period.

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I understand the study and am willing to cooperate.

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I have been diagnosed with a bleeding disorder that is not von Willebrand disease.

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I have had a blood clot in the past.

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My kidney function is impaired with a creatinine level of 2.5 mg/dL or higher.

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I have had or currently have a VWF inhibitor.

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I have a history of FVIII inhibitor at a significant level.

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I have heavy or irregular bleeding due to a condition in my cervix or uterus.

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I have never received treatment with a VWF product before this study.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~12 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Bleeding time procedure

Secondary study objectives

Spontaneous abortion

Hemorrhage

Area Under the Level Versus Time Curve From 0 to 96 Hours (AUC0-96; ss) for FVIII:C

+37 more

Awards & Highlights

No Placebo-Only Group

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

Pivotal Trial

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

Trial Design

3Treatment groups

Experimental Treatment

Group I: Cohort 3: Participants With Age <6 yearsExperimental Treatment2 Interventions

Participants with age \<6 years who have received OD therapy of VWF product or prophylactic treatment with a pdVWF product will receive rVWF with an initial dose selected within the range of 40 to 60 IU/kg rVWF, intravenous infusions, twice-weekly for 12 months. Participants may receive rVWF with or without ADVATE intravenous infusions, when indicated (as deemed necessary for breakthrough bleeding episode treatment and perioperative bleeding management).

Group II: Cohort 2: Participants With Age >=6 to <12 yearsExperimental Treatment2 Interventions

Participants with age \>=6 to \<12 years who have received OD therapy of VWF product or prophylactic treatment with a pdVWF product will receive rVWF with an initial dose selected within the range of 40 to 60 IU/kg rVWF, intravenous infusions, twice-weekly for 12 months. Participants may receive rVWF with or without ADVATE intravenous infusions, when indicated (as deemed necessary for breakthrough bleeding episode treatment and perioperative bleeding management).

Group III: Cohort 1: Participants With Age >=12 to <18 yearsExperimental Treatment2 Interventions

Participants with age greater than or equal to (\>=) 12 to less than (\<) 18 years who have received on-demand (OD) therapy or prophylactic treatment with a pdVWF product will receive rVWF with an initial dose selected within the range of 40 to 60 international units per kilogram (IU/kg) rVWF, intravenous infusions, twice-weekly for 12 months. Participants may receive rVWF with or without ADVATE intravenous infusions, when indicated (as deemed necessary for breakthrough bleeding episode treatment and perioperative bleeding management).

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Recombinant von Willebrand Factor (rVWF)

2015

Completed Phase 3

~30

ADVATE

2022

Completed Phase 4

~1030