Sirolimus to improve blood cell production in RUNX1 familial platelet disorder
Treatments studied
Part of Blood & lymphatic clinical trials.
This trial tests a low dose of sirolimus, a medicine that may help your bone marrow make more blood cells, especially platelets. It is for people with a specific genetic change (RUNX1) that affects blood cell production.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are 18 years or older.
- You have a confirmed RUNX1 gene change that is known to cause familial platelet disorder.
- You are willing to provide bone marrow samples before and after treatment.
- Your platelet count is at least 50,000 per microliter.
- Your kidney, liver, and heart function are within acceptable limits.
- You have not had certain blood cancers or conditions like myelodysplastic syndrome.
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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