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Purpose

The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of NXT007 prophylaxis compared with Factor VIII (FVIII) prophylaxis in participants with severe or moderate congenital hemophilia A without inhibitors. The study will include people aged ≥12 years old with severe or moderate congenital hemophilia A without inhibitors on previous FVIII prophylaxis treatment.

Condition

Eligibility

Eligible Ages
Over 12 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • Diagnosis of severe (FVIII:C <1 IU/dL [International Unit per decilitre]) or moderate (FVIII:C between ≥1 IU/dL and ≤5 IU/dL) congenital hemophilia A without inhibitors against FVIII - No documented inhibitor (i.e., <0.6 BU/mL [Bethesda unit per millilitre]), FVIII half-life ≥6 hours, or FVIII recovery >66% in the last 3 years prior to screening - Documented historical negative test for FVIII inhibitor (i.e., <0.6 BU/mL) within 12 months prior to enrollment - Documentation of the details of prophylactic and episodic FVIII treatment and of the number and type of bleeding episodes for at least the last 6 months prior to screening - Agreement to adhere to the contraception requirements (for potential participants with childbearing potential)

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 anti-retroviral therapy to treat HIV - Planned surgery (excluding minor procedures such as non-molar tooth extraction, 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 ECG evidence or clinical history of prior myocardial infarction - Refusal to accept plasma-derived and/or blood product transfusion support in an emergency scenario - 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)

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: FVIII SOC Prophylaxis 		Participants randomized to this arm will receive FVIII standard of care (SOC) prophylaxis for the main study treatment period.
  • Drug: Human Coagulation Factor VIII
    Factor VIII (FVIII) prophylaxis standard of care (SOC) will be administered at the dose and frequency as stated in the local labels and per local country practice.
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 FVIII 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

University of Colorado Hemophilia and Thrombosis Center
Aurora, Colorado 80045-7202

University of Iowa Hospitals and Clinics
Iowa City, Iowa 52242-1009

More Details

Status
Recruiting
Sponsor
Hoffmann-La Roche

Study Contact

Reference Study ID Number: WO45886 https://forpatients.roche.com/ No attachments to email below.
888-662-6728 (U.S. Only)
global-roche-genentech-trials@gene.com

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.