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

Why it matters

Who can join: the full rules

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 it's running

• University of Colorado Hemophilia and Thrombosis Center
  Aurora, Colorado, United States Recruiting
• University of Iowa Hospitals and Clinics
  Iowa City, Iowa, United States Recruiting
• Arbesu Hematologia
  Godoy Cruz, Mendoza Province, Argentina Recruiting
• Instituto de Hematología Y Medicina Clínica Dr. Rubén Dávoli
  Rosario, Santa Fe Province, Argentina Recruiting
• Universitätsklinikum AKH Wien
  Vienna, Austria Recruiting
• Hemocentro Da UNICAMP
  Campinas, São Paulo, Brazil Recruiting

Study contacts

• Study coordinator: Reference Study ID Number: WO45886 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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• NXT007 versus emicizumab to prevent bleeds 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.

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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.