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RECRUITING

NCT07416604

PHASE3

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

Sponsor: Hoffmann-La Roche

View on ClinicalTrials.gov

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.

Official 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

Key Details

Gender

All

Age Range

12 Years - Any

Study Type

INTERVENTIONAL

Enrollment

360

Start Date

2026-04-27

Completion Date

2032-01-29

Last Updated

2026-10-06

Healthy Volunteers

Conditions

Hemophilia A

Interventions

COMBINATION_PRODUCT

NXT007

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

DRUG

Emicizumab

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

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

Locations (59)

Center for Inherited Blood Disorders

Orange, California, United States

University of Colorado Hemophilia and Thrombosis Center

Aurora, Colorado, United States

University Of Miami Hospital

Miami, Florida, United States

St Joseph's Children's Hospital of Tampa

Tampa, Florida, United States

Innovative Hematology, Inc.

Indianapolis, Indiana, United States

University Of Iowa Hospitals And Clinics

Iowa City, Iowa, United States

University of Michigan - 1500 E Medical Center Dr

Ann Arbor, Michigan, United States

M Health Fairview - Pediatric Specialty Clinic-Discovery

Minneapolis, Minnesota, United States

The University of Texas Health Science Center at San Antonio

San Antonio, Texas, United States

Washington Center for Bleeding Disorders

Seattle, Washington, United States

Arbesu Hematologia

Godoy Cruz, Mendoza Province, Argentina

Instituto de Hematología Y Medicina Clínica Dr. Rubén Dávoli

Rosario, Santa Fe Province, Argentina

The Alfred Hospital

Melbourne, Victoria, Australia

Royal Children's Hospital Melbourne - PIN

Parkville, Victoria, Australia

UZ Leuven

Leuven, Antwerp, Belgium

Cliniques Universitaires Saint-Luc

Brussels, Belgium

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

Ribeirão Preto, São Paulo, Brazil

London Health Sciences Centre

London, Ontario, Canada

Peking Union Medical College Hospital - East Campus

Beijing, Beijing Municipality, China

Fujian Medical University Union Hospital

Fuzhou, Fujian, China

Nanfang Hospital, Southern Medical University

Guangzhou, Guangdong, China

Chengdu Women's and Children's Central Hospital

Chengdu, Sichuan, China

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

Tianjin, Tianjin Municipality, China

Rigshospitalet

København Ø, Capital, Denmark

AP-HP - Hôpital Bicêtre

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

Hospices Civils de Lyon

Bron, France

CHU de Lille - Institut Cœur Poumon

Lille, France

AP-HP - Hôpital universitaire Necker-Enfants malades

Paris, France

Universitätsklinikum Bonn

Bonn, North Rhine-Westphalia, Germany

Debreceni Egyetem Klinikai Kozpont Nagyerdei Campus

Debrecen, Pest County, Hungary

St James's Hospital

Dublin, Ireland

The Chaim Sheba Medical Center - PPDS

Ramat Gan, Central District, Israel

Azienda Ospedaliera Universitaria Federico II

Naples, Campania, Italy

Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico

Milan, Lombardy, Italy

IRCCS Istituto Clinico Humanitas

Rozzano, Lombardy, Italy

Azienda Ospedaliera Universitaria Careggi

Florence, Tuscany, Italy

Nagoya University Hospital

Nagoya, Aiti, Japan

Gunma University Hospital

Maebashi, Gunma, Japan

Nara Medical University Hospital

Kashihara-shi, Nara, Japan

Ogikubo Hospital

Suginami-Ku, Tokyo, Japan

Amsterdam UMC Location AMC- Meibergdreef 9

Amsterdam, North Holland, Netherlands

Universitair Medisch Centrum Groningen

Groningen, Netherlands

Universitair Medisch Centrum Utrecht Cancer Center - PPDS

Utrecht, Netherlands

Auckland City Hospital

Auckland, New Zealand

Wellington Hospital

Wellington, New Zealand

Haemophilia Comprehensive Care Centre

Parktown, Gauteng, South Africa

Kyungpook National University Hospital

Daegu, South Korea

Hospital Universitario Vall d'Hebron - PPDS

Barcelona, Spain

Hospital de la Santa Creu i Sant Pau

Barcelona, Spain

Hospital Universitario La Paz - PPDS

Madrid, Spain

Hospital Regional Universitario de Malaga ? Hospital General

Málaga, Spain

Hospital Universitario Virgen del Rocio - PPDS

Seville, Spain

Hospital Universitari i Politecnic La Fe de Valencia

Valencia, Spain

National Taiwan University Hospital

Taipei, Taiwan

Royal Free Hospital - PPDS

London, Camden, United Kingdom

St Thomas' Hospital

London, Greater London, United Kingdom

The Royal London Hospital

London, Middlesex, United Kingdom

University Hospital of Wales

Cardiff, United Kingdom

Great Ormond Street Hospital

London, United Kingdom