Clinical Trial: Low-Dose Sirolimus for the Treatment of RUNX1 Familial Platelet Disorder

U.S., Sept. 29 -- ClinicalTrials.gov registry received information related to the study (NCT07844694) titled 'Low-Dose Sirolimus for the Treatment of RUNX1 Familial Platelet Disorder' on Sept. 21.

Brief Summary: This phase I trial studies the safety and side effects of low-dose sirolimus in treating patients with RUNX1 familial platelet disorder (FPD). RUNX1-FPD is a rare inherited disorder with symptoms such as mild to moderately low platelet count, abnormal platelet function, and an increased risk of developing cancers like myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). It is caused by changes (variants) in the RUNX1 gene that is passed down (inherited) from an affected parent. Sirolimus is typically given to help preve...