NXT007 versus emicizumab to prevent bleeds in people with Hemophilia A

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 3 interventional trial compares prophylactic NXT007 with standard emicizumab prophylaxis in people aged 12 years and older who have congenital Hemophilia A (severe or moderate without FVIII inhibitors, or any severity with chronic FVIII inhibitors). Participants receive either NXT007 or emicizumab prophylaxis and are followed prospectively for bleeding rates, safety events, and pharmacokinetic/pharmacodynamic measures. The study collects detailed historical bleeding and treatment data and monitors on-study bleeding episodes, adverse events, and drug levels to compare effectiveness and tolerability. Results will focus on how NXT007 performs relative to emicizumab for reducing bleeds and on its safety profile.

Why it matters

Who can join: the full rules

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

Where it's running

• Center for Inherited Blood Disorders
  Orange, California, United States Recruiting
• University of Colorado Hemophilia and Thrombosis Center
  Aurora, Colorado, United States Recruiting
• University Of Miami Hospital
  Miami, Florida, 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

+13 more sites. See ClinicalTrials.gov for the full list.

Study contacts

• Study coordinator: Reference Study ID Number: BO45887 https://forpatients.roche.com/ No attachments to email below.
• Email: global-roche-genentech-trials@gene.com
• Phone: 888-662-6728 (U.S. Only)

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Bone health in severe hemophilia A: emicizumab vs efanesoctocog alfa

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.