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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

A CLINICAL STUDY TO EVALUATE THE EFFECTS OF NXT007 COMPARED TO EMICIZUMAB PROPHYLAXIS IN PEOPLE WITH HEMOPHILIA A

Daniel Hart

F. Hoffmann-La Roche Ltd.

1-1 NIHONBASHI-MUROMACHI 2-CHOME, CHUO-KU,Tokyo

clinical-trials@chugai-pharm.co.jp

Clinical trials information

Chugai Pharmaceutical Co., Ltd.

1-1 NIHONBASHI-MUROMACHI 2-CHOME, CHUO-KU,Tokyo

+81-120189706

clinical-trials@chugai-pharm.co.jp

Recruiting

April. 01, 2026

Interventional

randomized controlled trial

open(masking not used)

active control

parallel assignment

treatment purpose

Diagnosis of severe (FVIII:C < 1 IU/dL) or moderate (FVIII:C between >= 1 IU/dL and <= 5 IU/dL) congenital HA 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, 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

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 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
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)

Both

Hemophilia-A

NXT007:Subcutaneous injection, Protocol-specified dosage
Emicizumab:Subcutaneous injection, Protocol-specified dosage

Efficacy
Annualized number of treated bleeds (ABR) over the main study treatment period from the Month 2 visit until the Clinical cutoff date

Efficacy,Safety,Phamacokinetics,Phamacodynamics,other
ABR for All Bleeds Over the Main Study Treatment Period from Month 2 until the clinical cutoff date
ABR for Treated Spontaneous Bleeds Over the Main Study Treatment Period from Month 2 until the clinical cutoff date
ABR for Treated Joint Bleeds Over the Main Study Treatment Period from Month 2 until the clinical cutoff date
ABR for Treated Target Joint Bleeds Over the Main Study Treatment Period from Month 2 until the clinical cutoff date
Adjusted Mean Treatment Burden Domain Score in Comprehensive Assessment Tool of Challenges in Hemophilia (CATCH) Questionnaire - Adult Version at Month 8
Mean Treatment Burden Domain Score in CATCH Questionnaire - Adolescent Version at Month 8
Change From Baseline in Preoccupation Domain Score of the CATCH Questionnaire (Adult and Adolescent Versions)
Change From Baseline in Social Activity Impact Domain Score of the CATCH Questionnaire (Adult and Adolescent Versions)
Change From Baseline in Recreational Activity Impact Domain Score of the CATCH Questionnaire (Adult and Adolescent Versions)
Physical Impact Domain Score of the Treatment Administration Satisfaction Questionnaire (TASQ) at Specified Timepoints
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
Incidence and Severity of Thromboembolic Events and Thrombotic Microangiopathy
Incidence and Severity of Injection-Site Reactions
Incidence of Adverse Events Leading to Discontinuation of Assigned Study Treatment
Incidence of Severe Hypersensitivity, Anaphylaxis, or Anaphylactoid Reactions
Plasma Concentration of NXT007
Percentage of Participants With Anti-Drug Antibodies (ADAs) Against NXT007 at Baseline and During the Study
Percentage of Participants With Neutralizing ADAs Against NXT007

+81-744-22-3051

Jan. 28, 2026

+81-27-220-8740

Jan. 28, 2026

Yes

Qualified researchers may request access to individual patient level data through the clinical study data request platform. For further details on Chugai's Data Sharing Policy and how to request access to related clinical study documents, see here (www.chugai-pharm.co.jp/english/profile/rd/ctds_request.html).

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