Evaluation of NXT007 Treatment Safety and Effects in Male Children and Adults with Moderate or Severe Hemophilia A

What this Trial Is About

Researchers are evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, immunogenicity, and efficacy of NXT007 in male participants with moderate or severe hemophilia A. This Phase III, open-label, non-randomized trial includes adult, adolescent, and pediatric male participants, with or without factor VIII inhibitors. The study aims to better understand how NXT007 works and its effects in these groups. Participants receive NXT007 through subcutaneous injections with a regimen of two loading doses every two weeks followed by maintenance doses once every four weeks. The trial has two parts Part 1 tests multiple ascending doses in adult and adolescent males, while Part 2 evaluates multiple doses in pediatric males. Dosing is carefully adjusted based on the schedule and participant age group. Throughout the study, participants undergo regular assessments including monitoring for adverse events, clinical laboratory tests, vital signs, and electrocardiograms over up to 7.5 years. Researchers track NXT007 blood levels, immune responses, and bleeding rates. Participants attend scheduled visits for evaluations and treatment, with the study lasting up to 7.5 years to gather long-term safety and effectiveness data.

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A