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Tyrosine Kinase Inhibitor

Imatinib for Bone Marrow Failure Syndrome

Phase 1

Recruiting

Led By Lea C Cunningham, M.D.

Research Sponsored by National Cancer Institute (NCI)

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Affected participants must have a history of clinically significant bleeding as defined by history of abnormal ISTH-BAT score, use of anti-bleeding medications (e.g. amicar), or history of platelet transfusion.

--creatinine clearance >= 60 mL/min/1.73 m^2 for participants with creatinine levels above institutional normal.

Must not have

Participants who received prior hematologic malignancy directed therapy.

Participants receiving medication that would affect platelet number or function (e.g., aspirin and anti-platelet medications).

Timeline

Screening 3 weeks

Treatment Varies

Follow Up arm 1 for 1 month and arm 2 for 3 months

Awards & highlights

No Placebo-Only Group

Summary

This trial tests a drug (imatinib) to help people with RUNX1 mutations that cause bleeding/bruising & higher cancer risk. Adults with/without mutation needed. Participants take the drug, have tests & follow-up visits. Healthy participants have 1 visit with tests & questionnaires.

Who is the study for?

Adults over 18 with RUNX1 mutations causing symptoms like easy bleeding, and healthy relatives without the mutation. Participants must have certain blood cell counts, no significant organ dysfunction, and not be on conflicting medications or treatments for other conditions. Pregnant women and those unable to take oral medication are excluded.

What is being tested?

The trial is testing imatinib, a daily oral tablet taken at home for up to 84 days by people with RUNX1 gene mutations. It aims to see if it can improve their condition. The study includes regular clinic visits for monitoring through physical exams, blood tests, heart function tests, and optional bone marrow biopsies.

What are the potential side effects?

While specific side effects of imatinib in this trial context aren't listed here, common ones include nausea, muscle cramps, rash or swelling around the eyes. More serious but less common effects could involve liver problems or severe skin reactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have a history of significant bleeding issues.

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My kidneys are functioning well, with a creatinine clearance rate of 60 mL/min or higher.

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I have a confirmed genetic mutation in RUNX1.

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I can swallow pills and do not have major issues absorbing nutrients.

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I am 18 years old or older.

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I can take care of myself but might not be able to do active work.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have received treatment for blood cancer before.

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I am not on medications that affect blood clotting, like aspirin.

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My test shows a specific mutation in my cancer cells.

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I do not have serious heart conditions like heart failure, chest pain, or irregular heartbeat.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ arm 1 for 1 month and arm 2 for 3 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~arm 1 for 1 month and arm 2 for 3 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and arm 1 for 1 month and arm 2 for 3 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Determine the dose of imatinib for dose expansion in participants with pathogenic or likely pathogenic germline RUNX1 mutations during the dose escalation phase

Determine the safety of imatinib in participants with pathogenic or likely pathogenic germline RUNX1 mutations during the dose expansion phase

Secondary study objectives

Change in platelet aggregation score as compared to unaffected controls

Clinically meaningful change from baseline ISTH-BAT

Improvement in platelet dense granule structure by electron microscopy as compared to baseline

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