A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors

Completed · Phase 3

Conditions studied: Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor, Hemophilia A

In brief

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).

Key facts

Study ID

NCT04158648

Run by

Hoffmann-La Roche

People needed

73

Starts

2020-02-10

Expected to finish

2025-12-19

Last updated by the study team

2026-01-23

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Diagnosis of mild (FVIII level between >5% and <40%) or moderate (FVIII level between ≥1% and ≤5%) congenital Hemophilia A without FVIII inhibitors
• Weight ≥3 kilograms (kg)
• Need for prophylaxis based on investigator assessment
• A negative test for inhibitor (i.e., <0.6 Bethesda Units per milliliter [BU/mL]) within 8 weeks prior to enrollment
• No documented inhibitor (i.e., <0.6 BU/mL), FVIII half-life <6 hours, or FVIII recovery <66% in the last 5 years
• Documentation of the details of prophylactic or episodic FVIII treatment and of number of bleeding episodes for at least the last 24 weeks prior to enrollment
• Adequate hematologic, hepatic, and renal function
• For women of childbearing potential: agreement to remain abstinent or use contraception (as defined in the protocol) during the treatment period and for at least 24 weeks after the final dose of study drug

You may not qualify if…

• Inherited or acquired bleeding disorder other than mild or moderate congenital hemophilia A
• History of illicit drug or alcohol abuse within 48 weeks prior to screening, in the investigator's judgment
• Previous (within the last 12 months) or current treatment for thromboembolic disease or signs of thromboembolic disease
• Other conditions that may currently increase the risk of bleeding or thrombosis
• History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection
• Planned surgery during the emicizumab loading dose phase (surgeries in participants on emicizumab from Week 5 onwards are allowed)
• Known HIV infection with CD4 counts <200 cells per microlitre (/μL)
• Concomitant disease, condition, significant abnormality on screening evaluation or laboratory tests, or treatment that could interfere with the conduct of the study, or that would in the opinion of the investigator, pose an additional unacceptable risk in administering study drug to the participant
• Receipt of any of the following: An investigational drug to treat or reduce the risk of hemophilic bleeds within 5 half-lives of last drug administration with the exception of prior emicizumab prophylaxis; A non-hemophilia-related investigational drug within last 30 days or 5 half-lives, whichever is shorter; or Any other investigational drug currently being administered or planned to be administered
• Inability to comply with the study protocol in the opinion of the investigator
• Pregnant or breastfeeding, or intending to become pregnant during the study (women of childbearing potential must have a negative serum pregnancy test result within 7 days prior to initiation of study drug)

Where it is running

• 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

Full record on ClinicalTrials.gov

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