Comparing NXT007 injections with Factor VIII prophylaxis for people with moderate-to-severe hemophilia A

A Multicenter, Randomized, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of NXT007 Prophylaxis Versus Factor VIII Prophylaxis in People With Hemophilia A Without Inhibitors

This will see if regular NXT007 injections prevent bleeds better than standard Factor VIII prophylaxis in people aged 12 and older with moderate-to-severe hemophilia A who do not have FVIII inhibitors.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 126 (estimated)
• Ages: 12 Years and up
• Sex: All
• Sponsor: Hoffmann-La Roche (Industry-sponsored)
• Drugs / interventions: rituximab
• Locations: 2 sites (Kashihara-shi, Nara and 1 other locations)
• Trial ID: NCT07416526 on ClinicalTrials.gov

What this trial studies

This phase 3 interventional trial compares NXT007 prophylaxis head-to-head with standard Factor VIII (FVIII) prophylaxis in participants aged 12 years and older with severe or moderate congenital hemophilia A without inhibitors. The study measures efficacy (including bleeding rates), safety, and pharmacokinetics/pharmacodynamics while participants receive the assigned prophylactic regimen. Eligible participants must have documented negative FVIII inhibitor tests and a history of FVIII prophylaxis, and they will undergo regular clinic visits and blood sampling. Key outcomes include annualized bleeding rate, adverse events, and PK/PD parameters to determine how NXT007 performs versus FVIII.

Who should consider this trial

Good fit: People aged 12 years and older with severe (FVIII:C <1 IU/dL) or moderate (FVIII:C 1–5 IU/dL) congenital hemophilia A who have no documented FVIII inhibitors, a recent history of FVIII prophylaxis, required PK parameters, and who agree to the study contraception rules are the intended participants.

Not a fit: People with active or historical FVIII inhibitors, children under 12, or those with contraindicating allergies or other investigator-determined safety concerns are unlikely to benefit from participation.

Why it matters

Potential benefit: If successful, NXT007 could reduce bleeding frequency and/or treatment burden compared with standard FVIII prophylaxis for people with hemophilia A.

How similar studies have performed: Similar bispecific antibody prophylaxis (for example, emicizumab) has shown strong success in preventing bleeds in hemophilia A, so this therapeutic approach has prior positive precedent.

Eligibility criteria

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)

Where this trial is running

Kashihara-shi, Nara and 1 other locations

• Nara Medical University Hospital — Kashihara-shi, Nara, Japan (Recruiting)
• Tokyo Medical University Hospital — Shinjuku-Ku, Tokyo, Japan (Recruiting)

Study contacts

• Study coordinator: Reference Study ID Number: WO45886 https://forpatients.roche.com/

Conditions Hemophilia A