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

NCT07416604 | Recruiting Phase 3 DMC FDA Drug

• 3 other identifiers: BO458872025-522435-33-002025
• Study Type: interventional
• Target: 360
• Locations: 19 countries
• Sites: 42

Brief Summary

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.

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (1)

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

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

Secondary Outcomes (22)

• ABR for All Bleeds Over the Main Study Treatment Period

  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

  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

  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

  Month 8

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

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

Study Arms (3)

Main Study Treatment Period: NXT007 Prophylaxis

EXPERIMENTAL

Participants randomized to this arm will receive NXT007 prophylaxis for the main study treatment period.

Combination Product: NXT007

Main Study Treatment Period: Emicizumab Prophylaxis

ACTIVE COMPARATOR

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

Open-Label Extension Period: NXT007 Prophylaxis

EXPERIMENTAL

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

Interventions

NXT007 COMBINATION_PRODUCT

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

Also known as: Zemocimig, RO7589655, RG6512

Main Study Treatment Period: NXT007 Prophylaxis; Open-Label Extension Period: NXT007 Prophylaxis

Emicizumab DRUG

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

Also known as: Hemlibra, RO5534262, RG6013

Main Study Treatment Period: Emicizumab Prophylaxis

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Hoffmann-La Roche: lead
• Chugai Pharmaceutical: collaborator

Study Sites (42)

Center for Inherited Blood Disorders

Orange, California, 92868, United States

RECRUITING

University of Colorado Hemophilia and Thrombosis Center

Aurora, Colorado, 80045-7202, United States

RECRUITING

St Joseph's Children's Hospital of Tampa

Tampa, Florida, 33607-6307, United States

RECRUITING

Innovative Hematology, Inc.

Indianapolis, Indiana, 46260, United States

RECRUITING

University Of Iowa Hospitals And Clinics

Iowa City, Iowa, 52242-1009, United States

RECRUITING

Washington Center for Bleeding Disorders

Seattle, Washington, 98101-3932, United States

RECRUITING

Arbesu Hematologia

Godoy Cruz, Mendoza Province, M5504FKD, Argentina

RECRUITING

Instituto de Hematología Y Medicina Clínica Dr. Rubén Dávoli

Rosario, Santa Fe Province, S2000CFK, Argentina

RECRUITING

UZ Leuven

Leuven, Antwerp, 3000, Belgium

RECRUITING

Cliniques Universitaires Saint-Luc

Brussels, 1200, Belgium

RECRUITING

Hospital Das Clinicas da Faculdade de Medicina de Ribeirão Preto - USP

Ribeirão Preto, São Paulo, 14051-140, Brazil

RECRUITING

Nanfang Hospital, Southern Medical University

Guangzhou, Guangdong, 510515, China

RECRUITING

Chengdu Women's and Children's Central Hospital

Chengdu, Sichuan, 610073, China

RECRUITING

Institute of Hematology and Blood Diseases Hospital Chinese Academy of Medical Sciences

Tianjin, Tianjin Municipality, 301617, China

SUSPENDED

Rigshospitalet

København Ø, Capital, 2100, Denmark

RECRUITING

AP-HP - Hôpital Bicêtre

Le Kremlin-Bicêtre, Val-de-Marne, 94270, France

RECRUITING

Hospices Civils de Lyon

Bron, 69500, France

RECRUITING

CHU de Lille - Institut Cœur Poumon

Lille, 59037, France

RECRUITING

Universitätsklinikum Bonn

Bonn, North Rhine-Westphalia, 53127, Germany

RECRUITING

Debreceni Egyetem Klinikai Kozpont Nagyerdei Campus

Debrecen, Pest County, 4032, Hungary

RECRUITING

The Chaim Sheba Medical Center - PPDS

Ramat Gan, Central District, 5262100, Israel

RECRUITING

Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico

Milan, Lombardy, 20122, Italy

RECRUITING

IRCCS Istituto Clinico Humanitas

Rozzano, Lombardy, 20089, Italy

RECRUITING

Azienda Ospedaliera Universitaria Careggi

Florence, Tuscany, 50134, Italy

RECRUITING

Nagoya University Hospital

Nagoya, Aiti, 466-8560, Japan

RECRUITING

Gunma University Hospital

Maebashi, Gunma, 371-8511, Japan

RECRUITING

Nara Medical University Hospital

Kashihara-shi, Nara, 634-8522, Japan

RECRUITING

Ogikubo Hospital

Suginami-Ku, Tokyo, 167-0035, Japan

RECRUITING

Universitair Medisch Centrum Utrecht Cancer Center - PPDS

Utrecht, 3584 CX, Netherlands

RECRUITING

Auckland City Hospital

Auckland, 1023, New Zealand

RECRUITING

Haemophilia Comprehensive Care Centre

Parktown, Gauteng, 2193, South Africa

RECRUITING

Kyungpook National University Hospital

Daegu, 41944, South Korea

RECRUITING

Hospital Universitario Vall d'Hebron - PPDS

Barcelona, 08035, Spain

RECRUITING

Hospital de la Santa Creu i Sant Pau

Barcelona, 08041, Spain

RECRUITING

Hospital Universitario La Paz - PPDS

Madrid, 28046, Spain

RECRUITING

Hospital Regional Universitario de Malaga ? Hospital General

Málaga, 29010, Spain

RECRUITING

Hospital Universitario Virgen del Rocio - PPDS

Seville, 41013, Spain

RECRUITING

Hospital Universitari i Politecnic La Fe de Valencia

Valencia, 46026, Spain

RECRUITING

National Taiwan University Hospital

Taipei, 10002, Taiwan

RECRUITING

The Royal London Hospital

London, Middlesex, E1 1FR, United Kingdom

RECRUITING

University Hospital of Wales

Cardiff, CF14 4XW, United Kingdom

RECRUITING

Great Ormond Street Hospital

London, WC1N 3JH, United Kingdom

RECRUITING

Related Links

• Please use this form to submit your questions for a faster response: https://www.gene.com/contact-us/submit-medical-inquiry. Do not include or attach any medical records when emailing or completing the form. A nurse will respond within 24 business hours.

MeSH Terms

Conditions

Hemophilia A

Interventions

emicizumab

Condition Hierarchy (Ancestors)

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

Study Officials

• Clinical Trials

  Hoffmann-La Roche

  STUDY DIRECTOR

Central Study Contacts

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

CONTACT

888-662-6728 (U.S. Only); global-roche-genentech-trials@gene.com

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

CONTACT

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 11, 2026
• First Posted: February 18, 2026
• Study Start: April 27, 2026
• Primary Completion (Estimated): February 29, 2028
• Study Completion (Estimated): January 29, 2032
• Last Updated: September 4, 2026

Record last verified: 2026-09

Data Sharing

• IPD Sharing: Will share

Locations

ES (6); DK (1); AR (2); ZA (1); BR (1); JP (4); US (6); NZ (1); CN (3); FR (3); HU (1); TW (1); BE (2); GB (3); NL (1); DE (1); IT (3); KR (1); IL (1)