Low-dose sirolimus for patients with RUNX1 platelet disorder
Treatments studied
Part of Blood & lymphatic, Cancer, Genetic & congenital clinical trials.
This trial tests whether a low dose of sirolimus, a drug that calms the immune system, can help improve blood counts and reduce bleeding risk in people with a genetic condition called RUNX1 familial platelet disorder. If it works, it might lower the chance of developing serious blood problems later.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be at least 18 years old.
- You must have a confirmed change in the RUNX1 gene that is known to cause the platelet disorder.
- Your platelet count must be at least 50,000 per microliter of blood.
- You need to be willing to provide bone marrow samples at the start and end of treatment.
- Your kidneys and liver need to be working well (based on routine blood tests).
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
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.
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This study follows people who have a known or suspected RUNX1 gene variant linked to FPDMM-like features. It may help improve understanding of the gene, and participants may contribute samples that researchers use for genetic testing and research.
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