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New hope for toddlers with rare bleeding disorder: wilate shows promise

NCT ID NCT04953884

Disease control Sponsor: Octapharma Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested a medicine called wilate in 12 children under 6 years old with severe von Willebrand disease, a bleeding disorder. The goal was to see if regular infusions of wilate could prevent bleeding episodes over 12 months. Researchers measured how many bleeds occurred, how the drug moved through the body, and any side effects. The treatment aims to control the disease, not cure it.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

12 people

The number who actually took part.

Started

Sep 2021

Finished

Dec 2024

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

Up to 5 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Patients aged \<6 years at the time of screening 2. 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 3. Minimum BW 12.5 kg at the time of screening (for Moldova and Czech Republic, minimum BW 11.0 kg at the time of screening) 4. 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)) Exclusion Criteria: 1. History or current suspicion of VWF or FVIII inhibitors 2. Injection of 1-deamino-8-D-arginine vasopressin (DDAVP) or VWF-containing product within 72 hours prior to inclusion 3. Medical history of a thromboembolic event 4. Platelet count \<100,000/µL at screening (except for VWD type 2B) 5. Patients receiving, or scheduled to receive, immunosuppressant drugs (other than antiretroviral chemotherapy), such as prednisone (equivalent to \>10 mg/day), or similar drugs 6. Treatment with any investigational medicinal product (IMP) in another interventional clinical study currently or within four weeks before enrolment 7. Other coagulation disorders or bleeding disorders 8. Known hypersensitivity to any of the components of the study drug

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Conditions

The condition(s) this trial relates to.

hereditary von Willebrand disease von Willebrand disease (hereditary or acquired)

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Von Willebrand disease

Contacts and locations

Locations

  • Danylo Halytsky Lviv National Medical University, Communal Institution of Lviv Regional Council "Western Ukrainian Specialized Children's Medical Centre"

    Lviv, Ukraine

  • FSBI National Research Medical Center of Pediatric Hematology, Oncology and Immunology

    Moscow, Russia

  • Gerinnungszentrum Rhein-Ruhr Ambulanz und Fachlaboratorium für Gerinnungserkrankungen/Hämophilie

    Duisburg, Germany

  • IMSP Mother and Child Institute

    Chisinau, Moldova

  • Morozovskaya Children's Hospital

    Moscow, Russia

  • PHI University Clinic for Child Diseases

    Skopje, North Macedonia

  • Tulane University

    New Orleans, Louisiana, 70118, United States

  • University Hospital Motol, Department of Paediatric Haematology and Oncology

    Prague, Czechia

  • University Hospital Ostrava Department for Pediatric Medicine

    Ostrava, Czechia

More trials for these conditions

Other studies related to the condition(s) this trial covers.