Recruiting
Phase 3

NXT007 vs. Emicizumab

Sponsor:

Hoffmann-La Roche

Code:

NCT07416604

Conditions

Hemophilia A

Eligibility Criteria

Sex: All

Age: 12+

Healthy Volunteers: Not accepted

Interventions

NXT007

Emicizumab

Study Details

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

Study ID

NCT07416604

Start date

Apr 27, 2026

Status verified date

Sep, 2026

Completion date

Jan 29, 2032

Anticipated

Primary completion date

Feb 29, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 12+

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

360 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Main Study Treatment Period: NXT007 Prophylaxis

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

active comparator: Main Study Treatment Period: Emicizumab Prophylaxis

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.

experimental: Open-Label Extension Period: NXT007 Prophylaxis

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.

Interventions

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.

Primary outcome measure

  • 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) ]

Central Contacts and Locations

Central contacts

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

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

Locations

Center for Inherited Blood Disorders

Recruiting

Orange, California, United States, 92868

University of Colorado Hemophilia and Thrombosis Center

Recruiting

Aurora, Colorado, United States, 80045-7202

St Joseph's Children's Hospital of Tampa

Recruiting

Tampa, Florida, United States, 33607-6307

Innovative Hematology, Inc.

Recruiting

Indianapolis, Indiana, United States, 46260

University Of Iowa Hospitals And Clinics

Recruiting

Iowa City, Iowa, United States, 52242-1009

Washington Center for Bleeding Disorders

Recruiting

Seattle, Washington, United States, 98101-3932

More Information

Sponsor

Hoffmann-La Roche

Last update posted

Sep 4, 2026

Last verified

Sep, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-05. This information was provided to ClinicalTrials.gov by Hoffmann-La Roche on 2026-09-04.