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

StatusRecruiting
PhasePhase 1
Started

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Record History

  1. Phase changed: Early Phase 1 → Phase 1
    • Start date changed from estimate of Sep 30, 2026 to Oct 5, 2026
    • Site contact updated
  2. Study First Submitted
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
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.
Trial Details
NCT Number NCT07844694
Lead Sponsor OHSU Knight Cancer Institute
Collaborators: National Cancer Institute (NCI), Oregon Health and Science University, M.D. Anderson Cancer Center
Conditions
(from sponsor)		 Familial Platelet Disorder With Associated Myeloid Malignancy
Related terms Platelet Disorder, Familial, with Associated Myeloid Malignancy
Enrollment 6 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov