An Open-Label Study of Marstacimab Prophylaxis Compared to Historical Treatment in Pediatric Males With Severe Hemophilia A or Moderate to Severe Hemophilia B

What this Trial Is About

This research aims to learn about the safety and effects of marstacimab, a study medicine being evaluated for treating Hemophilia in pediatric patients. It focuses on children aged 1 to 17 years with severe Hemophilia A or moderate to severe Hemophilia B, including those with or without inhibitors. The study compares participant experiences on marstacimab with their past standard treatments to see if it helps prevent bleeding episodes common in Hemophilia. All participants will receive weekly marstacimab injections under the skin. The first dose is administered at the study site, and subsequent doses during the 12-month treatment period can be given at home or the study site. The study enrolls participants in age groups sequentially, starting with adolescents (12-17 years), followed by children 6-11 years, and then 1-5 years. The full participation lasts about 14 months, including screening, treatment, and follow-up periods. Participants will visit the study site at least 10 times and have 6 scheduled phone calls every two months. Researchers will monitor bleeding rates, adverse events, immune reactions, and joint health, along with quality of life changes. Safety assessments cover the entire study period, including follow-up, to evaluate marstacimab's effects and tolerability in children with Hemophilia.

A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B