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Phase 1Recruiting

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

Low-Dose Sirolimus to Increase Hematopoietic Function in Patients With RUNX1 Familial Platelet Disorder - Part II

Sponsor
OHSU Knight Cancer Institute (Academic or other)
Enrolment
6 planned
Conditions
Familial Platelet Disorder With Associated Myeloid Malignancy
Interventions
Biospecimen Collection; Bone Marrow Aspiration; Bone Marrow Biopsy; Questionnaire Administration; Sirolimus
Ages
adults, older adults
Registry
NCT07844694

Full record on ClinicalTrials.gov

From the registry

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.

Primary outcomes

  • Incidence of grade 3+ treatment-related adverse events (TRAEs)

History

  1. Study completion expected

  2. Primary completion expected

  3. Last registry update

  4. Started

  5. Registered