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NCT Number: NCT07416604

A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia A

The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of NXT007 prophylaxis compared with emicizumab prophylaxis in people age 12 years and older with severe or moderate congenital hemophilia A without factor VIII (FVIII) inhibitors or with hemophilia A of any severity (severe, moderate, and mild) with FVIII inhibitors.

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Key information

Conditions

Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Age range

12 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

The Chaim Sheba Medical Center - PPDS, Ramat Gan, Central District, Israel

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Diagnosis of severe (FVIII:C <1 International Unit per decilitre [IU/dL]) or moderate (FVIII:C between ≥1 IU/dL and ≤5 IU/dL) congenital hemophilia A with or without inhibitors against FVIII
  • Diagnosis of mild (FVIII:C between >5 IU/dL and <40 IU/dL) congenital hemophilia A with chronic FVIII inhibitors, defined as documented FVIII inhibitor ( ≥0.6 BU/mL or ≥1.0 BU/mL only for laboratories with a historical sensitivity cutoff for inhibitor detection of 1.0 BU/mL) and chronic reduction of endogenous baseline FVIII:C to <5 IU/dL for ≥12 months
  • Documented historical FVIII inhibitor assay results within the 12 months prior to enrollment
  • Documentation of the details of prophylactic and episodic FVIII treatment, bypassing agent (BPA) treatment, emicizumab prophylaxis treatment, and the number and type of bleeding episodes for at least the last 6 months prior to screening
  • For potential participants taking on-demand treatments prior to study entry: agreement to move to a prophylaxis treatment with either emicizumab or NXT007, according to assigned randomization

Exclusion criteria

  • Sensitivity to any of the study investigations, or components thereof, or drug or other allergy that, in the opinion of the investigator, contraindicates participation in the study
  • Use of systemic immunomodulators (e.g., interferon or rituximab) at the time of enrollment or planned use during the study, except for antiretroviral therapy to treat HIV
  • Refusal to accept plasma-derived and/or blood product transfusion support in an emergency scenario
  • Planned surgery (excluding minor procedures, such as non-molar tooth extraction or incision and drainage) during the study
  • History of ventricular dysrhythmias or risk factors for ventricular dysrhythmias such as structural heart disease (e.g., severe left ventricular systolic dysfunction, left ventricular hypertrophy), coronary heart disease (symptomatic or with ischemia demonstrated by diagnostic testing)
  • History or presence of an abnormal ECG that is deemed clinically significant, (e.g., complete left bundle branch block, second- or third-degree atrioventricular heart block) or evidence or clinical history of prior myocardial infarction

Treatment and study plan

NXT007

Combination Product

NXT007 will be administered subcutaneously (SC) using an integrated drug-device combination product.

Other names: Zemocimig, RO7589655, RG6512

Emicizumab

Drug

Emicizumab will be administered subcutaneously (SC) using vial and syringe.

Other names: Hemlibra, RO5534262, RG6013

Primary outcomes

  1. Annualized Bleed Rate (ABR) for Treated Bleeds Over the Main Study Treatment Period

    Time frame: From Month 2 until the clinical cutoff date (at least 7 months of study treatment)

Secondary outcomes

  1. ABR for All Bleeds Over the Main Study Treatment Period

    Time frame: From Month 2 until the clinical cutoff date (at least 7 months of study treatment)

  2. ABR for Treated Spontaneous Bleeds Over the Main Study Treatment Period

    Time frame: From Month 2 until the clinical cutoff date (at least 7 months of study treatment)

  3. ABR for Treated Joint Bleeds Over the Main Study Treatment Period

    Time frame: From Month 2 until the clinical cutoff date (at least 7 months of study treatment)

  4. Adjusted Mean Treatment Burden Domain Score in Comprehensive Assessment Tool of Challenges in Hemophilia (CATCH) Questionnaire - Adult Version at Month 8

    Time frame: Month 8

  5. ABR for Treated Target Joint Bleeds Over the Main Study Treatment Period

    Time frame: From Month 2 until the clinical cutoff date (at least 7 months of study treatment)

  6. Percentage of Participants with Zero Treated Bleeds Over the Main Study Treatment Period

    Time frame: From Month 2 until the clinical cutoff date (at least 7 months of study treatment)

  7. Number of Injections and Dose per Bleed of Coagulation Factors or Bypassing Agent Administered to Treat a Bleed Over the Main Study Treatment Period

    Time frame: From Month 2 until the clinical cutoff date (at least 7 months of study treatment)

  8. Annualized Injection Rate of FVIII or Bypassing Agent Over the Main Study Treatment Period

    Time frame: From Month 2 until the clinical cutoff date (at least 7 months of study treatment)

  9. Annualized Consumption Rate of FVIII or Bypassing Agent Over the Main Study Treatment Period

    Time frame: From Month 2 until the clinical cutoff date (at least 7 months of study treatment)

  10. Mean Treatment Burden Domain Score in CATCH Questionnaire - Adolescent Version at Month 8

    Time frame: Month 8

  11. Change From Baseline in Preoccupation Domain Score of the CATCH Questionnaire (Adult and Adolescent Versions)

    Time frame: At prespecified timepoints from Baseline until Study Completion (approximately 3.5 years)

  12. Change From Baseline in Social Activity Impact Domain Score of the CATCH Questionnaire (Adult and Adolescent Versions)

    Time frame: At prespecified timepoints from Baseline until Study Completion (approximately 3.5 years)

  13. Change From Baseline in Recreational Activity Impact Domain Score of the CATCH Questionnaire (Adult and Adolescent Versions)

    Time frame: At prespecified timepoints from Baseline until Study Completion (approximately 3.5 years)

  14. Physical Impact Domain Score of the Treatment Administration Satisfaction Questionnaire (TASQ) at Specified Timepoints

    Time frame: At prespecified timepoints from Baseline to Month 4

  15. Incidence and Severity of Adverse Events, With Severity Determined According To National Cancer Institute Common Terminology Criteria for Adverse Events, Version 5.0 (NCI CTCAE V5.0) Grading Scale

    Time frame: From Baseline until Study Completion (approximately 3.5 years)

  16. Incidence and Severity of Thromboembolic Events and Thrombotic Microangiopathy

    Time frame: From Baseline until Study Completion (approximately 3.5 years)

  17. Incidence and Severity of Injection-Site Reactions

    Time frame: From Baseline until Study Completion (approximately 3.5 years)

  18. Incidence of Adverse Events Leading to Discontinuation of Assigned Study Treatment

    Time frame: From Baseline until Study Completion (approximately 3.5 years)

  19. Incidence of Severe Hypersensitivity, Anaphylaxis, or Anaphylactoid Reactions

    Time frame: From Baseline until Study Completion (approximately 3.5 years)

  20. Plasma Concentration of NXT007

    Time frame: At prespecified timepoints from Baseline to Study Completion (approximately 3.5 years)

  21. Percentage of Participants With Anti-Drug Antibodies (ADAs) Against NXT007 at Baseline and During the Study

    Time frame: At prespecified timepoints from Baseline to Study Completion (approximately 3.5 years)

  22. Percentage of Participants With Neutralizing ADAs Against NXT007

    Time frame: At prespecified timepoints from Baseline to Study Completion (approximately 3.5 years)

Study contacts

Contact information is provided by the study sponsor or research team.

Fastest response: use the inquiry form. https://www.gene.com/contact-us/submit-medical-inquiry

CONTACT

Reference Study ID Number: BO45887 https://forpatients.roche.com/ No attachments to email below.

CONTACT

[email protected]

888-662-6728 (U.S. Only)

Sponsors and collaborators

Lead sponsor

Hoffmann-La Roche

Industry

Collaborators

  • Chugai Pharmaceutical

Registry information

Official study title

A Multicenter, Randomized, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of NXT007 Prophylaxis Versus Emicizumab Prophylaxis in People With Hemophilia A

Acronym: ZEBRHA 2

Important dates

Study start
2026
Primary completion
2028
Study completion
2032
First posted
Feb 18, 2026
Registry last updated
Jul 7, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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