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A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors
Sponsor: Hoffmann-La Roche
Summary
This is a multicenter, open-label, single-arm study designed to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of emicizumab in participants with mild or moderate hemophilia A without inhibitors against factor VIII (FVIII).
Official title: A Multicenter, Open-Label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Emicizumab in Patients With Mild or Moderate Hemophilia A Without FVIII Inhibitors
Key Details
Gender
All
Age Range
Any - Any
Study Type
INTERVENTIONAL
Enrollment
73
Start Date
2020-02-10
Completion Date
2025-12-19
Last Updated
2026-08-27
Healthy Volunteers
No
Conditions
Interventions
Emicizumab
Four loading doses of emicizumab 3 milligrams per kilogram of body weight (mg/kg) will be administered subcutaneously (SC) once a week (QW) for 4 weeks followed by participant's preference of one of the three following maintenance SC dose regimens: 1.5 mg/kg QW, 3 mg/kg once every 2 weeks (Q2W), or 6 mg/kg once every 4 weeks (Q4W).
Locations (22)
Childrens Hospital LA
Los Angeles, California, United States
Hemophilia of Georgia Center for Bleeding & Clotting Disorders
Atlanta, Georgia, United States
Indiana Hemophilia & Thrombosis center
Indianapolis, Indiana, United States
University of Michigan, C.S. Mott Children's Hospital
Ann Arbor, Michigan, United States
Washington Institute for Coagulation
Seattle, Washington, United States
Cliniques Universitaires St-Luc
Brussels, Belgium
UZ Leuven Gasthuisberg
Leuven, Belgium
Kaye Edmonton Clinic
Edmonton, Alberta, Canada
Eastern Health - General Hospital
St. John's, Newfoundland and Labrador, Canada
Hopital Cardio-vasculaire Louis Pradel
Bron, France
CH de Bicetre
Le Kremlin-Bicêtre, France
Groupe Hospitalier Necker Enfants Malades
Paris, France
Universitätsklinikum Bonn
Bonn, Germany
Klinikum der Universität München, Campus Innenstadt
München, Germany
Amsterdam UMC Location AMC
Amsterdam, Netherlands
Instytut Hematologii i Transfuzjologii
Warsaw, Poland
Charlotte Maxeke Johannesburg Hospital
Johannesburg, South Africa
Hospital Universitario la Paz
Madrid, Spain
Hospital Universitario Virgen del Rocio
Seville, Spain
Cardiff and Vale NHS Trust
Cardiff, United Kingdom
Royal Free Hospital
London, United Kingdom
Great Ormond street Hospital for Children NHS Foundation Trust
London, United Kingdom