A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia A (ZEBRHA 2)

September 3, 2026 updated by: Hoffmann-La Roche

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

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
360

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.
  • Name: Fastest response: use the inquiry form. https://www.gene.com/contact-us/submit-medical-inquiry

Study Contact Backup

Study Locations

  • Argentina
    • Mendoza Province
      • Godoy Cruz, Mendoza Province, Argentina, M5504FKD
        • Recruiting
        • Arbesu Hematologia
    • Santa Fe Province
      • Rosario, Santa Fe Province, Argentina, S2000CFK
        • Recruiting
        • Instituto de Hematología Y Medicina Clínica Dr. Rubén Dávoli
  • Belgium
      • Brussels, Belgium, 1200
        • Recruiting
        • Cliniques universitaires Saint-Luc
    • Antwerp
      • Leuven, Antwerp, Belgium, 3000
        • Recruiting
        • UZ Leuven
  • Brazil
    • São Paulo
      • Ribeirão Preto, São Paulo, Brazil, 14051-140
        • Recruiting
        • Hospital Das Clinicas da Faculdade de Medicina de Ribeirão Preto - USP
  • China
    • Guangdong
      • Guangzhou, Guangdong, China, 510515
        • Recruiting
        • Nanfang Hospital, Southern Medical University
    • Sichuan
      • Chengdu, Sichuan, China, 610073
        • Recruiting
        • Chengdu Women's and Children's Central Hospital
    • Tianjin Municipality
      • Tianjin, Tianjin Municipality, China, 301617
        • Suspended
        • Institute of Hematology and Blood Diseases Hospital Chinese Academy of Medical Sciences
  • Denmark
    • Capital
      • København Ø, Capital, Denmark, 2100
        • Recruiting
        • Rigshospitalet
  • France
      • Bron, France, 69500
        • Recruiting
        • Hospices Civils de Lyon
      • Lille, France, 59037
        • Recruiting
        • CHU de Lille - Institut Cœur Poumon
    • Val-de-Marne
      • Le Kremlin-Bicêtre, Val-de-Marne, France, 94270
        • Recruiting
        • AP-HP - Hôpital Bicêtre
  • Germany
    • North Rhine-Westphalia
      • Bonn, North Rhine-Westphalia, Germany, 53127
        • Recruiting
        • Universitatsklinikum Bonn
  • Hungary
    • Pest County
      • Debrecen, Pest County, Hungary, 4032
        • Recruiting
        • Debreceni Egyetem Klinikai Kozpont Nagyerdei Campus
  • Israel
    • Central District
      • Ramat Gan, Central District, Israel, 5262100
        • Recruiting
        • The Chaim Sheba Medical Center - PPDS
  • Italy
    • Lombardy
      • Milan, Lombardy, Italy, 20122
        • Recruiting
        • Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
      • Rozzano, Lombardy, Italy, 20089
        • Recruiting
        • IRCCS Istituto Clinico Humanitas
    • Tuscany
      • Florence, Tuscany, Italy, 50134
        • Recruiting
        • Azienda Ospedaliera Universitaria Careggi
  • Japan
    • Aiti
      • Nagoya, Aiti, Japan, 466-8560
        • Recruiting
        • Nagoya University Hospital
    • Gunma
      • Maebashi, Gunma, Japan, 371-8511
        • Recruiting
        • Gunma University Hospital
    • Nara
      • Kashihara-shi, Nara, Japan, 634-8522
        • Recruiting
        • Nara Medical University Hospital
    • Tokyo
      • Suginami-Ku, Tokyo, Japan, 167-0035
        • Recruiting
        • Ogikubo Hospital
  • Netherlands
      • Utrecht, Netherlands, 3584 CX
        • Recruiting
        • Universitair Medisch Centrum Utrecht Cancer Center - PPDS
  • New Zealand
      • Auckland, New Zealand, 1023
        • Recruiting
        • Auckland City Hospital
  • South Africa
    • Gauteng
      • Parktown, Gauteng, South Africa, 2193
        • Recruiting
        • Haemophilia Comprehensive Care Centre
  • South Korea
      • Daegu, South Korea, 41944
        • Recruiting
        • Kyungpook National University Hospital
  • Spain
      • Barcelona, Spain, 08041
        • Recruiting
        • Hospital de La Santa Creu i Sant Pau
      • Barcelona, Spain, 08035
        • Recruiting
        • Hospital Universitario Vall d'Hebron - PPDS
      • Madrid, Spain, 28046
        • Recruiting
        • Hospital Universitario La Paz - PPDS
      • Málaga, Spain, 29010
        • Recruiting
        • Hospital Regional Universitario de Malaga ? Hospital General
      • Seville, Spain, 41013
        • Recruiting
        • Hospital Universitario Virgen del Rocio - PPDS
      • Valencia, Spain, 46026
        • Recruiting
        • Hospital Universitari i Politecnic La Fe de Valencia
  • Taiwan
      • Taipei, Taiwan, 10002
        • Recruiting
        • National Taiwan University Hospital
  • United Kingdom
      • Cardiff, United Kingdom, CF14 4XW
        • Recruiting
        • University Hospital of Wales
      • London, United Kingdom, WC1N 3JH
        • Recruiting
        • Great Ormond Street Hospital
    • Middlesex
      • London, Middlesex, United Kingdom, E1 1FR
        • Recruiting
        • The Royal London Hospital
  • United States
    • California
      • Orange, California, United States, 92868
        • Recruiting
        • Center for Inherited Blood Disorders
    • Colorado
      • Aurora, Colorado, United States, 80045-7202
        • Recruiting
        • University of Colorado Hemophilia and Thrombosis Center
    • Florida
      • Tampa, Florida, United States, 33607-6307
        • Recruiting
        • St Joseph's Children's Hospital of Tampa
    • Indiana
      • Indianapolis, Indiana, United States, 46260
        • Recruiting
        • Innovative Hematology, Inc.
    • Iowa
      • Iowa City, Iowa, United States, 52242-1009
        • Recruiting
        • University of Iowa Hospitals and Clinics
    • Washington
      • Seattle, Washington, United States, 98101-3932
        • Recruiting
        • Washington Center for Bleeding Disorders

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Main Study Treatment Period: NXT007 Prophylaxis
Participants randomized to this arm will receive NXT007 prophylaxis for the main study treatment period.
Combination product: NXT007
NXT007 will be administered subcutaneously (SC) using an integrated drug-device combination product.
Other Names:
  • RO7589655
  • RG6512
  • Zemocimig
Active Comparator: Main Study Treatment Period: Emicizumab Prophylaxis
Participants randomized to this arm will receive emicizumab prophylaxis for the main study treatment period at 3 mg/kg once weekly (QW) for 4 weeks as loading doses, followed by maintenance dosing of either 1.5 mg/kg QW, 3 mg/kg once every 2 weeks (Q2W), or 6 mg/kg once every 4 weeks (Q4W). Loading doses are not required for participants who were taking emicizumab prior to study start.
Drug: Emicizumab
Emicizumab will be administered subcutaneously (SC) using vial and syringe.
Other Names:
  • Hemlibra
  • RO5534262
  • RG6013
Experimental: Open-Label Extension Period: NXT007 Prophylaxis
After the main study treatment period, participants in the NXT007 arm will be able to continue with NXT007 dosing, and participants in the Emicizumab arm will be able to switch to NXT007, in the open-label extension period.
Combination product: NXT007
NXT007 will be administered subcutaneously (SC) using an integrated drug-device combination product.
Other Names:
  • RO7589655
  • RG6512
  • Zemocimig

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
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)
From Month 2 until the clinical cutoff date (at least 7 months of study treatment)

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
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)
From Month 2 until the clinical cutoff date (at least 7 months of study treatment)
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)
From Month 2 until the clinical cutoff date (at least 7 months of study treatment)
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)
From Month 2 until the clinical cutoff date (at least 7 months of study treatment)
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
Month 8
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)
From Month 2 until the clinical cutoff date (at least 7 months of study treatment)
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)
From Month 2 until the clinical cutoff date (at least 7 months of study treatment)
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)
From Month 2 until the clinical cutoff date (at least 7 months of study treatment)
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)
From Month 2 until the clinical cutoff date (at least 7 months of study treatment)
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)
From Month 2 until the clinical cutoff date (at least 7 months of study treatment)
Mean Treatment Burden Domain Score in CATCH Questionnaire - Adolescent Version at Month 8
Time Frame: Month 8
Month 8
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)
At prespecified timepoints from Baseline until Study Completion (approximately 3.5 years)
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)
At prespecified timepoints from Baseline until Study Completion (approximately 3.5 years)
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)
At prespecified timepoints from Baseline until Study Completion (approximately 3.5 years)
Physical Impact Domain Score of the Treatment Administration Satisfaction Questionnaire (TASQ) at Specified Timepoints
Time Frame: At prespecified timepoints from Baseline to Month 4
At prespecified timepoints from Baseline to Month 4
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)
From Baseline until Study Completion (approximately 3.5 years)
Incidence and Severity of Thromboembolic Events and Thrombotic Microangiopathy
Time Frame: From Baseline until Study Completion (approximately 3.5 years)
From Baseline until Study Completion (approximately 3.5 years)
Incidence and Severity of Injection-Site Reactions
Time Frame: From Baseline until Study Completion (approximately 3.5 years)
From Baseline until Study Completion (approximately 3.5 years)
Incidence of Adverse Events Leading to Discontinuation of Assigned Study Treatment
Time Frame: From Baseline until Study Completion (approximately 3.5 years)
From Baseline until Study Completion (approximately 3.5 years)
Incidence of Severe Hypersensitivity, Anaphylaxis, or Anaphylactoid Reactions
Time Frame: From Baseline until Study Completion (approximately 3.5 years)
From Baseline until Study Completion (approximately 3.5 years)
Plasma Concentration of NXT007
Time Frame: At prespecified timepoints from Baseline to Study Completion (approximately 3.5 years)
At prespecified timepoints from Baseline to Study Completion (approximately 3.5 years)
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)
At prespecified timepoints from Baseline to Study Completion (approximately 3.5 years)
Percentage of Participants With Neutralizing ADAs Against NXT007
Time Frame: At prespecified timepoints from Baseline to Study Completion (approximately 3.5 years)
At prespecified timepoints from Baseline to Study Completion (approximately 3.5 years)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Hoffmann-La Roche

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Chugai Pharmaceutical

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Clinical Trials, Hoffmann-La Roche

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
April 27, 2026

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
February 29, 2028

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
January 29, 2032

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
February 11, 2026

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 11, 2026

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
February 18, 2026

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
September 4, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 3, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
September 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • BO45887
  • 2025 (U.S. NIH Grant/Contract: Faculty of Social Sciences Scientific Grant at the University of Gdańsk)
  • 2025-522435-33-00 (Ctis)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data_sharing

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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