New hope for tots with rare bleeding disorder

NCT ID NCT04953884

Summary

This study tested a drug called Wilate to see if it could safely prevent bleeding episodes in very young children with a severe form of von Willebrand disease (VWD), a rare bleeding disorder. Twelve children under 6 years old received regular doses of Wilate for a year to see how well it worked to control bleeds and how their bodies processed the medicine. The main goal was to measure the total number of bleeding episodes that occurred while on this preventive treatment.

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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

Conditions

Explore the condition pages connected to this study.