Study medicine for children with hemophilia A or B
Treatments studied
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial tests a study medicine (marstacimab) for children with severe hemophilia A or B, to see if it can help prevent or reduce bleeding episodes. It may be an option for children who do not have certain other health conditions.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Child must be male and within the age and weight ranges: 1–5 years old and at least 5 kg, 6–11 years old and at least 19 kg, or 12–17 years old and at least 25 kg.
- Child must have a diagnosis of severe hemophilia A or moderately severe to severe hemophilia B.
- For ages 6–17, there must be at least 1 year of medical records showing bleeding episodes and factor treatment; for ages 1–5, at least 3 months of records.
- For the non-inhibitor group: no history of inhibitors in the last 5 years, and (for ages 6–17) at least 50 days of prior factor treatment plus being on a stable prophylactic (preventive) regimen for at least 12 months (or 3 months for ages 1–5).
- For the inhibitor group: must have a high-titer inhibitor (≥5 BU/mL) or a low-titer inhibitor that does not respond to factor replacement, and be on on-demand treatment for bleeds (with a certain number of bleeds in the past year for ages 6–17, or in the past 3–6 months for ages 1–5).
- Child must not have other bleeding disorders, heart or blood-clotting conditions, active liver disease, or be HIV-positive (if under 6) or have low CD4 count (if 6–17).
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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