NXT007 versus emicizumab to prevent bleeds in people with Hemophilia A
A Multicenter, Randomized, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of NXT007 Prophylaxis Versus Emicizumab Prophylaxis in People With Hemophilia A
This will test whether NXT007 prevents bleeding better than emicizumab in people aged 12 and older with Hemophilia A, including those with or without factor VIII inhibitors.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 360 (estimated) |
| Ages | 12 Years and up |
| Sex | All |
| Sponsor | Hoffmann-La Roche Industry-sponsored |
| Drugs / interventions | emicizumab, rituximab |
| Locations | 6 sites (Orange, California and 5 other locations) |
| Trial ID | NCT07416604 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates are people aged 12 or older with documented congenital Hemophilia A who meet the specified factor VIII activity ranges and inhibitor status and who can provide recent inhibitor assay results and treatment/bleeding history.
Not a fit: People under age 12, those without confirmed Hemophilia A or without the required laboratory documentation, and those with different bleeding disorders are unlikely to benefit from this study.
Why it matters
Potential benefit: If successful, NXT007 could offer improved bleed protection or dosing advantages over emicizumab for people with Hemophilia A.
How similar studies have performed: Bispecific antibody approaches like emicizumab have already shown substantial clinical benefit, and NXT007 represents a newer agent using a similar concept that earlier-phase work has suggested may be promising.
Eligibility criteria
Show full inclusion / exclusion criteria
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 this trial is running
Orange, California and 5 other locations
- Center for Inherited Blood Disorders — Orange, California, United States (Recruiting)
- Innovative Hematology, Inc. — Indianapolis, Indiana, United States (Recruiting)
- Washington Center for Bleeding Disorders — Seattle, Washington, United States (Recruiting)
- Gunma University Hospital — Maebashi, Gunma, Japan (Recruiting)
- Nara Medical University Hospital — Kashihara-shi, Nara, Japan (Recruiting)
- Ogikubo Hospital — Suginami-Ku, Tokyo, Japan (Recruiting)
Study contacts
- Study coordinator: Reference Study ID Number: BO45887 https://forpatients.roche.com/
- Email: global-roche-genentech-trials@gene.com
- Phone: 888-662-6728 (U.S. Only)
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.