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Coagulation Factor

Wilate for Von Willebrand Disease

Phase 3

Waitlist Available

Research Sponsored by Octapharma

Eligibility Criteria Checklist

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

Must have

Type 3 (at least four patients), severe type 2 (except 2N) or severe type 1 VWD (any of which with VWF:RCo <20%) according to medical history, requiring substitution therapy with a VWF-containing product

Minimum body weight 12.5 kg at the time of screening

Must not have

Patients receiving, or scheduled to receive, immunosuppressant drugs (other than antiretroviral chemotherapy), such as prednisone (equivalent to >10 mg/day), or similar drugs

Medical history of a thromboembolic event

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 12 months of treatment

Awards & highlights

Pivotal Trial

No Placebo-Only Group

Summary

This trial is testing a new medication to see if it is safe and effective in treating von Willebrand Disease in children under the age of 6.

Who is the study for?

This trial is for children under 6 with severe Von Willebrand Disease (VWF:RCo <20%) and a minimum body weight of 12.5 kg. They must not have had thromboembolic events, other bleeding disorders, or be on immunosuppressants like high-dose prednisone. Children can't join if they've used certain drugs recently or are allergic to the study drug's components.

What is being tested?

The trial tests 'wilate' in young patients with severe VWD as routine prevention over one year. It aims to assess how effective it is, how the body processes it, whether it causes immune reactions, and its safety profile.

What are the potential side effects?

While specific side effects for wilate aren't listed here, treatments for VWD typically may include reactions at injection sites, headaches, dizziness, allergic reactions or rashes. Close monitoring will identify any potential side effects during the trial.

Eligibility Criteria

Inclusion Criteria

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

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I have a severe type of Von Willebrand Disease and need treatment with a specific product.

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I weigh at least 12.5 kg.

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I am under 6 years old.

Exclusion Criteria

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

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I am taking, or will take, drugs that lower my immune system, like high-dose prednisone.

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I have had a blood clot in the past.

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I haven't had DDAVP or VWF treatments in the last 3 days.

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I have a bleeding or clotting disorder.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 12 months of treatment

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 12 months of treatment

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

Treatment Details

Study Objectives

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

Secondary study objectives

Efficacy of wilate in the prevention and treatment of spontaneous and traumatic breakthrough BEs based on their rate and the proportion of spontaneous and traumatic BEs successfully treated with wilate

The overall efficacy of wilate in perioperative prophylaxis against excessive bleeding as assessed at the end of the postoperative period by the responsible treating investigator

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

1Treatment groups

Experimental Treatment

Group I: wilate treatmentExperimental Treatment1 Intervention

PK: Single dose of 80 IU/kg body weight (BW). Prophylactic treatment: 30-50 IU/kg BW administered 2-3 times per week at the recommended dose of over 12 months. Minor haemorrhage: loading dose 30-50 IU/kg BW followed by a maintenance dose of 30-40 IU/kg BW every 12-24 hours to achieve von Willebrand factor activity (VWF:Ac) and FVIII:C trough levels of \>30%. Major haemorrhage: loading dose 50-80 IU/kg BW followed by a maintenance dose of 30-50 IU/kg BW every 12-24 hours to achieve VWF:Ac and FVIII:C trough levels of \>50%. Minor surgery: loading dose of 40-60 IU/kg BW followed by a maintenance dose of 20-30 IU/kg BW every 12- 24 hours for up to 3 days, to achieve VWF:Ac peak levels of 50% after loading dose and trough levels \>30% during maintenance. Major surgery: loading dose of 60-80 IU/kg BW followed by a maintenance dose of 30-40 IU/kg BW every 12-24 hours for up to 6 days or longer, to achieve VWF:Ac peak levels of 100% after loading dose and trough levels \>50% during maintenance

0 / 7Eligibility criteria met