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Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency

NCT06090669

This study is recruiting at 1 US site in 1 state, according to its ClinicalTrials.gov record last updated .

Summary

Background: Runt-related transcription factor 1 (RUNX1) gene regulates the formation of blood cells. People with mutations of this gene may bleed or bruise easily; they are also at higher risk of getting cancers of the blood, bone marrow, and lymph nodes. Objective: The purpose of the study includes determining which dose of imatinib is best for people with pathogenic or likely pathogenic RUNX1 mutations without blood cancers, and to determine whether there are any changes in platelet function and inflammatory markers. Eligibility: Adults aged 18 and older with RUNX1 mutations.

Shortened from the sponsor's brief summary. View official record on ClinicalTrials.gov

Conditions studied

  • Inherited Bone Marrow Failure Syndrome
  • Familial Platelet Disorder With Predisposition to Myeloid Malignancies

Eligibility basics and study details

Study type
Interventional
Phase
Phase 1
Ages
18 to 120 years
Sex
All
Healthy volunteers
Accepted
Sponsor
National Cancer Institute (NCI)
Last updated on ClinicalTrials.gov
October 8, 2026

These are the basic criteria only. The full inclusion and exclusion criteria are on the official record, and the study team decides who can take part.

US recruiting sites

Maryland · 1

  • National Institutes of Health Clinical Center · Bethesda, MD

Source: ClinicalTrials.gov, data processed . Recruiting US sites filtered by distance and site status; summaries shortened.