New hope for toddlers with rare bleeding disorder: wilate shows promise
NCT ID NCT04953884
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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Danylo Halytsky Lviv National Medical University, Communal Institution of Lviv Regional Council "Western Ukrainian Specialized Children's Medical Centre"
Lviv, Ukraine
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FSBI National Research Medical Center of Pediatric Hematology, Oncology and Immunology
Moscow, Russia
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Gerinnungszentrum Rhein-Ruhr Ambulanz und Fachlaboratorium für Gerinnungserkrankungen/Hämophilie
Duisburg, Germany
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IMSP Mother and Child Institute
Chisinau, Moldova
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Morozovskaya Children's Hospital
Moscow, Russia
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PHI University Clinic for Child Diseases
Skopje, North Macedonia
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Tulane University
New Orleans, Louisiana, 70118, United States
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University Hospital Motol, Department of Paediatric Haematology and Oncology
Prague, Czechia
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University Hospital Ostrava Department for Pediatric Medicine
Ostrava, Czechia
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Tiny camera may spot hidden bleeding in rare blood disorder
- Trial for bleeding disorder drug in toddlers pulled before it started
- New hope for kids with rare bleeding disease: study tests rVWF therapy
- New study tracks joint replacement success in bleeding disorder patients
- Scientists dig into genetic roots of rare bleeding disorders
- New hope for rare bleeding disorder: Long-Term drug shows promise