Trial of imatinib for people with RUNX1 deficiency
Treatments studied
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial tests if imatinib can help people with a genetic condition called RUNX1 deficiency, which causes bleeding problems. It aims to increase the activity of the RUNX1 gene to improve blood clotting.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed RUNX1 gene mutation that causes the condition.
- You must have a history of significant bleeding problems, such as needing platelet transfusions or medication for bleeding.
- You must be at least 18 years old and have adequate organ function (kidneys, liver, blood counts).
- You must not have certain cancer treatments or medications that affect platelets, and you must not be pregnant.
- You must agree to use effective birth control during the study and for 30 days after the last dose.
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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