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

NCT ID: NCT04158648

Last Updated: 2026-01-23

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 73 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-02-10
• Study Completion Date: 2025-12-19

Brief 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).

Conditions

Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor; Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor; Hemophilia A

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Emicizumab

Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors will be enrolled to receive the emicizumab loading dose regimen followed by the participant's preference of one of 3 maintenance dose regimens.

Group Type: EXPERIMENTAL

Emicizumab

Intervention Type: DRUG

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).

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).

Intervention Type: DRUG

Other Intervention Names

Hemlibra; RO5534262; RG6013; ACE910

Eligibility Criteria

Inclusion Criteria

• 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

Exclusion Criteria

• 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)

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Hoffmann-La Roche

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Clinical Trials

Role: STUDY_DIRECTOR

Hoffmann-La Roche

Locations

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

Countries

United States; Belgium; Canada; France; Germany; Netherlands; Poland; South Africa; Spain; United Kingdom

References

Negrier C, Mahlangu J, Lehle M, Chowdary P, Catalani O, Bernardi RJ, Jimenez-Yuste V, Beckermann BM, Schmitt C, Ventriglia G, Windyga J, d'Oiron R, Moorehead P, Koparkar S, Teodoro V, Shapiro AD, Oldenburg J, Hermans C. Emicizumab in people with moderate or mild haemophilia A (HAVEN 6): a multicentre, open-label, single-arm, phase 3 study. Lancet Haematol. 2023 Mar;10(3):e168-e177. doi: 10.1016/S2352-3026(22)00377-5. Epub 2023 Jan 27.

Reference Type: DERIVED

PMID: 36716761 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

2019-002179-32

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

2023-506610-52-00

Identifier Type: REGISTRY

Identifier Source: secondary_id

BO41423

Identifier Type: -

Identifier Source: org_study_id