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

Purpose

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.

Condition

  • Hemophilia A

Eligibility

Eligible Ages
Over 12 Years
Eligible Sex
All
Accepts 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

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Main Study Treatment Period: NXT007 Prophylaxis 		Participants randomized to this arm will receive NXT007 prophylaxis for the main study treatment period.
  • Combination Product: NXT007
    NXT007 will be administered subcutaneously (SC) using an integrated drug-device combination product.
    Other names:
    • Zemocimig
    • RO7589655
    • RG6512
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.
  • Drug: Emicizumab
    Emicizumab will be administered subcutaneously (SC) using vial and syringe.
    Other names:
    • Hemlibra
    • RO5534262
    • RG6013
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.
  • Combination Product: NXT007
    NXT007 will be administered subcutaneously (SC) using an integrated drug-device combination product.
    Other names:
    • Zemocimig
    • RO7589655
    • RG6512

Recruiting Locations

Center for Inherited Blood Disorders
Orange, California 92868

Innovative Hematology, Inc.
Indianapolis, Indiana 46260

Washington Center for Bleeding Disorders
Seattle, Washington 98101-3932

More Details

Status
Recruiting
Sponsor
Hoffmann-La Roche

Study Contact

Reference Study ID Number: BO45887 https://forpatients.roche.com/
888-662-6728 (U.S. Only)
global-roche-genentech-trials@gene.com