Clinical Trial: Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency
Study Status: Completed
Recruit Status: RECRUITING
Condition: Inherited Bone Marrow Failure Syndrome
Study Type: INTERVENTIONAL
Official Title: Phase Ib Study of Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency
Brief 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:To test a drug (imatinib) in people with RUNX1 mutations that cause symptoms.Eligibility:Adults aged 18 and older with RUNX1 mutations.Healthy people without this mutation, including family members of affected participants, are also…
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