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Recruiting Phase 3

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

NCT07416604 · tracked via the Priya Life Science South Korea tracker
Phase
Phase 3
Started
2026-04-27
Last updated
2026-10-06

Condition(s) studied

Hemophilia A

Investigational drug(s) / intervention(s)

NXT007Emicizumab →

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

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

Study 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.

Eligibility

Sex
ALL
Min age
12 Years
Max age
—
Healthy volunteers
No
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

Primary outcome measure(s)

  • 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)

Trial sites (59)

FacilityCityRegionStatus
Center for Inherited Blood Disorders Orange California Recruiting
University of Colorado Hemophilia and Thrombosis Center Aurora Colorado Recruiting
University Of Miami Hospital Miami Florida Recruiting
St Joseph's Children's Hospital of Tampa Tampa Florida Recruiting
Innovative Hematology, Inc. Indianapolis Indiana Recruiting
University Of Iowa Hospitals And Clinics Iowa City Iowa Recruiting
University of Michigan - 1500 E Medical Center Dr Ann Arbor Michigan Recruiting
M Health Fairview - Pediatric Specialty Clinic-Discovery Minneapolis Minnesota Recruiting
The University of Texas Health Science Center at San Antonio San Antonio Texas Recruiting
Washington Center for Bleeding Disorders Seattle Washington Recruiting
Arbesu Hematologia Godoy Cruz Mendoza Province Recruiting
Instituto de Hematología Y Medicina Clínica Dr. Rubén Dávoli Rosario Santa Fe Province Recruiting
The Alfred Hospital Melbourne Victoria Recruiting
Royal Children's Hospital Melbourne - PIN Parkville Victoria Recruiting
UZ Leuven Leuven Antwerp Recruiting
Cliniques Universitaires Saint-Luc Brussels Belgium Recruiting
Hospital Das Clinicas da Faculdade de Medicina de Ribeirão Preto - USP Ribeirão Preto São Paulo Recruiting
London Health Sciences Centre London Ontario Recruiting
Peking Union Medical College Hospital - East Campus Beijing Beijing Municipality Recruiting
Fujian Medical University Union Hospital Fuzhou Fujian Suspended
Nanfang Hospital, Southern Medical University Guangzhou Guangdong Recruiting
Chengdu Women's and Children's Central Hospital Chengdu Sichuan Recruiting
Institute of Hematology and Blood Diseases Hospital Chinese Academy of Medical Sciences Tianjin Tianjin Municipality Recruiting
Rigshospitalet København Ø Capital Recruiting
AP-HP - Hôpital Bicêtre Le Kremlin-Bicêtre Val-de-Marne Recruiting
Hospices Civils de Lyon Bron France Recruiting
CHU de Lille - Institut Cœur Poumon Lille France Recruiting
AP-HP - Hôpital universitaire Necker-Enfants malades Paris France Recruiting
Universitätsklinikum Bonn Bonn North Rhine-Westphalia Recruiting
Debreceni Egyetem Klinikai Kozpont Nagyerdei Campus Debrecen Pest County Recruiting
St James's Hospital Dublin Ireland Recruiting
The Chaim Sheba Medical Center - PPDS Ramat Gan Central District Recruiting
Azienda Ospedaliera Universitaria Federico II Naples Campania Recruiting
Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico Milan Lombardy Recruiting
IRCCS Istituto Clinico Humanitas Rozzano Lombardy Recruiting
Azienda Ospedaliera Universitaria Careggi Florence Tuscany Recruiting
Nagoya University Hospital Nagoya Aiti Recruiting
Gunma University Hospital Maebashi Gunma Recruiting
Nara Medical University Hospital Kashihara-shi Nara Recruiting
Ogikubo Hospital Suginami-Ku Tokyo Recruiting

+ 19 more sites — see the full list on the official registry below.

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07416604 on ClinicalTrials.gov ↗ ← All trials in South Korea