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NCT05611801
A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
Conditions: Hemophilia A, Hemophilia B
Sex: Male
Ages: 1 Year – 17 Years
Healthy volunteers: No
Phase: PHASE3
Enrollment: 100
Sponsor: Pfizer
Location: Intermountain - Primary Children's Hospital Salt Lake City Utah
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.
Eligibility Criteria
Inclusion Criteria:
* Male participants of appropriate age and required minimum weight
* Participants aged 12 to 17 years must be at least 25 kgs at time of consent.
* Participants aged 6 to 11 years must be at least 19 kgs at time of consent.
* Minimum weight requirement for participants aged 1 to 5 years is to be determined.
* Participants with a diagnosis of severe hemophilia A or moderately severe to severe hemophilia B
* Participants must have at least 1 year of diary and/or medical records available in which exogenous FVIII or FIX replacement or bypass agent infusions and hemophilic bleeding episodes were consistently documented over the 12 months prior to the time of consent.
Participants who are enrolled into the Non-Inhibitor Cohort must also meet the following criteria:
* No current detectable inhibitor and no documented history of inhibitors in the 5 years prior to consent
* Must have at least 50 exposure days to FVIII/FIX replacement products
* Must be at least 80% compliant with a stable and effective routine prophylaxis regimen with FVIII/FIX replacement products, for at least 12 months prior to consent
Participants who are enrolled into the Inhibitor Cohort must also meet the following criteria:
* Documentation of current high titer inhibitor (≥5 BU/mL); or current low titer inhibitor (\
Source: ClinicalTrials.gov (NCT05611801). StuddyBuddy aggregates publicly available trial information.