A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
Status:
Not yet recruiting
Trial end date:
2028-09-10
Target enrollment:
Participant gender:
Summary
The purpose of this clinical trial is to learn about the safety and effects of the study
medicine (called marstacimab) for the potential treatment of hemophilia in pediatric
patients.
This study will enroll pediatric participants from ages 1 to 17 years in a sequential manner.
The study will open enrollment to adolescent participants aged 12 to 17 years first. Then
children aged 6 to 11 years will be permitted to enroll. Lastly, children aged 1 to 5 years
will be permitted to enroll.
This study will enroll participants who:
- have severe Hemophilia A or moderately severe to severe Hemophilia B (with or without
inhibitors)
- have accurate historical records documenting all factor VIII, factor IX, or bypass agent
infusions and hemophilia bleed events for at least 1 year prior to entering the study
- if a non-inhibitor patient, must be on a stable routine prophylaxis regimen with factor
VIII or factor IX replacement products for at least 12 months prior to study entry
- if an inhibitor patient, must be on an on-demand bypass treatment regimen during the 12
months prior to study entry
All participants in this study will receive marstacimab to use prophylactically. Marstacimab
will be given once a week as a subcutaneous (under the skin) shot. The first dose of
marstacimab will be given at the study site by the study site staff. During the 12-month
treatment period, weekly doses of marstacimab can be given at home, or if preferred, the
doses may be given by the study site staff.
To help us determine if the study medicine is safe and effective, we will compare participant
experiences when they are taking the study medicine to a historical period when they were
not. Researchers want to see if the study medicine works to prevent the bleeding episodes
commonly experienced by patients with Hemophilia.
Participants will be in this study for about 14 months (approximately 1 month in a Screening
period, 12 months receiving treatment, and 1 month in a follow-up period) during which they
will visit the study site at least 10 times. If preferred, and if local regulations allow it,
2 of the study visits can be completed at the participant's home instead of at the study
site. There will also be 6 scheduled telephone calls approximately every 2 months.