Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency

StatusRecruiting
PhasePhase 1
Started

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Trial flagged as At Risk
Primary endpoint reworded 1 of 2 changed
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Record History

The 50 most recent of 100 record versions are accounted for below. See every version on ClinicalTrials.gov ↗

  1. Primary endpoint(s) modified
    • Secondary endpoints revised (4)
  2. Enrollment target: 75 to 71
    • Study description expanded, about 42 words
  3. Re-verified, no change to tracked fields
  4. Re-verified, no change to tracked fields
  5. Re-verified, no change to tracked fields
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  1. Eligibility criteria expanded, about 16 words
    • Study description expanded, about 20 words
  2. 18 versions Re-verified since Apr 3, 2026, no change to tracked fieldsv77-v94
  3. Eligibility criteria revisedv76
  4. 12 versions Re-verified since Sep 20, 2025, no change to tracked fieldsv64-v75
  5. Enrollment target: 78 to 75v63
  6. Study Status, Study Description, Outcome Measuresv62
  7. Eligibility criteria shortened, about 47 wordsv61
  8. 6 versions Re-verified since Mar 7, 2025, no change to tracked fieldsv55-v60
  9. Eligibility criteria shortened, about 19 wordsv54
  10. 2 versions Re-verified since Dec 3, 2024, no change to tracked fieldsv52-v53
  11. Study Status, Eligibilityv51
  • 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.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
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. Healthy people without this mutation, including family members of affected participants, are also needed. Design: Participants with the RUNX1 mutation will be screened. They will have a physical exam with blood tests. They will have a test of their heart function. They may need a new bone marrow biopsy if they haven't had one in the past year. Imatinib is a tablet taken by mouth once a day, every day, at home. Affected participants in different parts of the study will take imatinib for either 42 days or up to 98 days. They will fill out questionnaires about how they are feeling. For the first part of the study, participants will have blood tests every 2 weeks, either at home or at the NIH, while they are taking the imatinib. They will have a follow up visit, at home or at the NIH, when they are done taking imatinib on Day 42. Participants on the second part of the study will come to NIH on days 1 and days 98. They will have blood tests every 2 weeks (at home or the NIH) while they are taking imatinib. They may opt to have a bone marrow biopsy repeated after they finish their course of imatinib. Participants will have a follow-up visit (at home or the NIH) 30 days after they stop taking imatinib. Participants who do not have the RUNX1 mutation will have 1 clinic visit. They will have blood tests. They will fill out questionnaires. They may opt to have a bone marrow biopsy.
Trial Details
NCT Number NCT06090669
Lead Sponsor National Cancer Institute (NCI)
Conditions
(from sponsor)		 Inherited Bone Marrow Failure Syndrome, Familial Platelet Disorder With Predisposition to Myeloid Malignancies
Related terms Congenital Bone Marrow Failure Syndromes
Enrollment 71 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov