Wilate for Von Willebrand Disease

Recruiting in Palo Alto (17 mi)

+10 other locations

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Octapharma

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?

The WIL-33 study aims to determine the efficacy, pharmacokinetics, immunogenicity and safety of wilate as routine prophylaxis in up to 12 paediatric patients (eight evaluable) with severe von Willebrand Disease VWD (defined as screening von Willebrand factor ristocetin cofactor activity \[VWF:RCo\] \<20%) under the age of 6 years, over a period of 12 months.

Research Team

Eligibility Criteria

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.

Inclusion Criteria

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

Voluntarily given, fully informed written and signed consent obtained before any study-related procedures are conducted (obtained from the patient's parent(s)/ legal guardian(s))

I weigh at least 12.5 kg.

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Exclusion Criteria

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

I have had a blood clot in the past.

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

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Treatment Details

Interventions

Wilate (Coagulation Factor)

Trial OverviewThe 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.

Participant Groups

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