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

Phase 3RecruitingNCT07416604

Hoffmann-La Roche360 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

360

Conditions

Hemophilia A

Interventions

NXT007COMBINATION_PRODUCT

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

EmicizumabDRUG

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

Eligibility

Sex: ALLMin age: 12 Years

Medical Language ↔ Plain English

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 (42)

Outcomes

Primary Outcomes

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)

Secondary Outcomes

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)

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)

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)

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

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)

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)

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)

Central 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

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.

Center for Inherited Blood Disorders

Orange, California, United States

RECRUITING

University of Colorado Hemophilia and Thrombosis Center

Aurora, Colorado, United States

RECRUITING

St Joseph's Children's Hospital of Tampa

Tampa, Florida, United States

RECRUITING

Innovative Hematology, Inc.

Indianapolis, Indiana, United States

RECRUITING

University Of Iowa Hospitals And Clinics

Iowa City, Iowa, United States

RECRUITING

Washington Center for Bleeding Disorders

Seattle, Washington, United States

RECRUITING

Arbesu Hematologia

Godoy Cruz, Mendoza Province, Argentina

RECRUITING

Instituto de Hematología Y Medicina Clínica Dr. Rubén Dávoli

Rosario, Santa Fe Province, Argentina

RECRUITING

UZ Leuven

Leuven, Antwerp, Belgium

RECRUITING

Cliniques Universitaires Saint-Luc

Brussels, Belgium

RECRUITING

...and 32 more locations

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)

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)

Mean Treatment Burden Domain Score in CATCH Questionnaire - Adolescent Version at Month 8

Time frame: 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)

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)

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)

Physical Impact Domain Score of the Treatment Administration Satisfaction Questionnaire (TASQ) at Specified Timepoints

Time frame: 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)

Incidence and Severity of Thromboembolic Events and Thrombotic Microangiopathy

Time frame: 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)

Incidence of Adverse Events Leading to Discontinuation of Assigned Study Treatment

Time frame: 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)

Plasma Concentration of NXT007

Time frame: 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)

Percentage of Participants With Neutralizing ADAs Against NXT007

Time frame: At prespecified timepoints from Baseline to Study Completion (approximately 3.5 years)