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NCT06090669 · ClinicalTrials.gov registry record · Phase 1

Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency

A Phase 1 study of Inherited Bone Marrow Failure Syndrome and Familial Platelet Disorder With Predisposition to Myeloid Malignancies, sponsored by National Cancer Institute (NCI).

Recruiting
Registry status
Phase 1
Development phase
75
Enrollment target
1
Study location

NCT06090669 is a Phase 1 study of Inherited Bone Marrow Failure Syndrome and Familial Platelet Disorder With Predisposition to Myeloid Malignancies that is actively recruiting participants, run by National Cancer Institute (NCI). The registered enrollment target is 75 participants, below the 746-participant average among 3 other Inherited Bone Marrow Failure Syndrome trials with a reported enrollment target (90% lower). The trial reports 1 study location across 1 state.

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The verdict

NCT06090669, a Phase 1 study of Inherited Bone Marrow Failure Syndrome and Familial Platelet Disorder With Predisposition to Myeloid Malignancies, is actively recruiting participants, sponsored by National Cancer Institute (NCI).

RECRUITING
Registry status
Phase 1
Development phase
75 participants
Enrollment target
1
Study location

Study 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. 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 28 days or up to 84 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 28. Participants on the second part of the study will come to NIH on days 1 and days 84. 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.

Interventions

  • DRUG imatinib
  • DEVICE TruSight Oncology

Study Locations (1)

Maryland

  • National Institutes of Health Clinical Center - Bethesda

Trial Details

FieldValue
Enrollment Target 75 participants
Start Date 2023-12-19
Est. Completion 2027-10-30
Phase Phase 1

Sponsor

National Cancer Institute (NCI)

3,257 total trials

What the Registry Record Tells You About NCT06090669

The ClinicalTrials.gov registry entry for NCT06090669 describes a study currently listed as recruiting, categorized as Phase 1. The registered enrollment target is 75 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 746-participant average among 3 other Inherited Bone Marrow Failure Syndrome trials with a reported enrollment target (90% lower). The listed sponsor is National Cancer Institute (NCI), which has 3,257 total studies on file at ClinicalTrials.gov.

The record links to 2 conditions, with Inherited Bone Marrow Failure Syndrome appearing as the primary indexed condition, and to 2 interventions - of which imatinib is the first listed.

NCT06090669 reports 1 study location spanning 1 distinct geographic area - top geographies include Maryland.

Frequently Asked Questions

What is clinical trial NCT06090669 about?

NCT06090669 is a clinical study titled "Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency". 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 inclu...

What is the current status of trial NCT06090669?

This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 75 participants. The study started on 2023-12-19. Estimated completion is 2027-10-30.

What conditions does trial NCT06090669 study?

This clinical trial studies the following conditions: Inherited Bone Marrow Failure Syndrome, Familial Platelet Disorder With Predisposition to Myeloid Malignancies.

What interventions are being tested in trial NCT06090669?

The interventions under investigation include: imatinib (DRUG), TruSight Oncology (DEVICE).

Who is sponsoring clinical trial NCT06090669?

This trial is sponsored by National Cancer Institute (NCI), which has 3,257 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT06090669 being conducted?

This trial has 1 study location across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

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Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

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