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COMPLETED
NCT04158648
PHASE3

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

View on ClinicalTrials.gov

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

Interventions

DRUG

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