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Familial Platelet Disorder With Associated Myeloid Malignancy

Tundra lists 1 Familial Platelet Disorder With Associated Myeloid Malignancy clinical trial. Each listing includes eligibility criteria, study locations, and direct links to research sites in the Tundra directory.

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RECRUITING

NCT07844694

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

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 prevent organ rejection in patients receiving kidney transplants. However, sirolimus may also be able to prevent RUNX1-FPD from progressing to cancers like MDS and AML by targeting another protein called mTORC1. Sirolimus may be a safe treatment for patients with RUNX1-FPD.

Gender: All

Ages: 18 Years - Any

Updated: 2026-10-13

1 state

Familial Platelet Disorder With Associated Myeloid Malignancy