A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A
Key facts
- Where
- Campinas and Ribeirão Preto, BrazilSee the 2 sites
- Phase
- Phase 3
Where in Brazil
- Hemocentro Da UNICAMPCampinas, BrazilTaking people
- Hospital Das Clinicas da Faculdade de Medicina de Ribeirão Preto - USP - PPDSRibeirão Preto, BrazilTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Hoffmann-La Roche
- Trial code
- NCT07416526EU 2025-522434-32-00
- Record updated
- 26 October 2026
- Original description
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 FVIII inhibitors. The study will include people aged ≥12 years old who have been on FVIII prophylaxis treatment prior to study entry.Read full original descriptionShow less
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
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 FVIII inhibitors. The study will include people aged ≥12 years old who have been on FVIII prophylaxis treatment prior to study entry.
Inclusion: 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: 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; 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; 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); Refusal to accept plasma-derived and/or blood product transfusion support in an emergency scenario.