55
Participants
Start Date
February 4, 2021
Primary Completion Date
May 22, 2023
Study Completion Date
May 18, 2030
Emicizumab
"Initially, all participants will receive 4 loading doses of 3 milligrams per kilogram (mg/kg) emicizumab subcutaneously (SC) once every week (QW) for 4 weeks followed by the maintenance dosing regimen 3 mg/kg emicizumab SC once every 2 weeks (Q2W) for a total of 52 weeks. Starting from Week 17 of treatment, individual participants may have their dose up-titrated to 3 mg/kg SC QW if they experience suboptimal bleeding control.~At the Week 53 clinic visit following consultation with the treating physician, parents/caregivers may elect for their child to continue with the maintenance 3-mg/kg SC Q2W dosing regimen or to switch to the maintenance 1.5-mg/kg SC QW or 6-mg/kg SC once every 4 weeks (Q4W) dosing regimen for the subsequent 7-year long-term follow-up period.~During the study, participants will be treated with emicizumab until unacceptable toxicity, discontinuation from the study due to any cause, or other criteria specified in the protocol, whichever occurs first."
Medizinische Universität Wien, Vienna
Cliniques Universitaires St-Luc, Brussels
The Children's Hospital at Westmead, Westmead
Charlotte Maxeke Johannesburg Hospital, Johannesburg
UZ Leuven Gasthuisberg, Leuven
Royal Children's Hospital, Parkville
Perth Children's Hospital, Nedlands
Hospital Universitario la Paz, Madrid
Ege University, School of Medicine, Izmir
Hospital Universitario Virgen del Rocio, Seville
AOU di Parma, Parma
AOU Careggi, Florence
Universitätsklinikum Bonn, Bonn
Ondokuz Mayis Univ. Med. Fac., Samsun
Hämophilie-Zentrum Rhein Main GmbH, Mörfelden-Walldorf
Tulane University Health Sciences Center, New Orleans
Groupe Hospitalier Necker Enfants Malades, Paris
University of Colorado Denver, Children's Hospital, Aurora
AORN Santobono Pausilipon, Napoli
Phoenix Children's Hospital, Phoenix
Children's Hospital Los Angeles, Los Angeles
Seattle Children's Hospital, Seattle
Sheba Medical Center - National Hemophilia Center, Tel Litwinsky
University of Michigan, Ann Arbor
Hospital das Clínicas Faculdades Médicas de Ribeirão Preto, Ribeirão Preto
Children's Hospital of Eastern Ontario, Ottawa
The Hospital for Sick Children, Toronto
Hospital Sant Joan de Deu, Esplugues de Llobregat
Adana Acibadem Hospital, Adana
Hacettepe University Medical Faculty, Ankara
Queen Elizabeth University Hospital, Glasgow
Hoffmann-La Roche
INDUSTRY