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 (NCT07416604)

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 (HA) without factor VIII (FVIII) inhibitors or with HA of any severity (severe, moderate, and mild) with FVIII inhibitors.

Trial phase

Phase 3

Trial participation type

This trial has been designed for the Clinical Research of a Drug.

Principal investigator

Prof Huyen Tran

Key inclusion data

INCLUSION: 1. 12 years and above 2. Body weight 40kg and above 3. Severe Haemophila A or Moderate HA with or without inhibitors 4. Agreement to take prophylaxis with either emicizumab or NXT007 according to assigned randomisation EXCLUSION: 1. Receipt of the following: An investigational drug to treat or reduce the risk of hemophilic bleeds within 5 half-lives of the last drug administration or persistently abnormal targeted parameter (e.g., anti-thrombin) outside the normal ranges, whichever is longer – Marstacimab, concizumab, or denecimig (Mim8) within 5 half-lives of the last drug administration prior to randomization – Altuviiio/Altuvoct [efanesoctocog alfa] within 7 days prior to randomization – Fitusiran (Qfitlia) within 5 half-lives of the last drug administration or persistently abnormal targeted parameter (anti-thrombin) outside the normal ranges, whichever is longer, prior to randomization – F8 gene therapy within the last five years, or plans to receive it – Any other investigational drug currently being administered or planned to be administered – Any ongoing doses of FVIII given for immune tolerance induction (ITI) or "FVIII tolerance-maintenance" regardless of dose or frequency 2. Planned surgery 3. Refusal to accept plasma-derived and/or blood product transfusion support in an emergency scenario 4. History or conditions, other than HA, which may indicate hypo- or hyper-coagulopathy risk including 5. Evidence of severe uncontrolled hypertension