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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.

Timeline

Screening 3 weeks

Treatment Varies

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

Awards & highlights

Study 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.Check my eligibility

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.See study design

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.

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

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 outcome measures

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

+2 more

Trial Design

3Treatment groups

Experimental Treatment

Active Control

Group I: Dose ExpansionExperimental Treatment2 Interventions

Imatinib at the MTD

Group II: Dose EscalationExperimental Treatment2 Interventions

Escalating doses of imatinib to determine the MTD

Group III: No TreatmentActive Control1 Intervention

Collection of blood or marrow only. No treatment.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

imatinib

2009

Completed Phase 3

~2150

0 / 6Eligibility criteria met

Find a Location

Who is running the clinical trial?

National Cancer Institute (NCI)Lead Sponsor

13,670 Previous Clinical Trials

40,926,358 Total Patients Enrolled

Lea C Cunningham, M.D.Principal InvestigatorNational Cancer Institute (NCI)

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Does this experiment accept elderly persons for participation?

"Prerequisites for participating in this clinical trial are an age between 18 to 120 years old. Additionally, 352 trials have been created specifically for minors and 2394 for elderly patients."

Answered by AI

Is this experiment available for enrollment?

"This clinical trial is no longer recruiting, based on the information found on clinicaltrials.gov; it was initially posted in October 30th of 2023 and last updated on October 17th of that same year. However, there are currently 2610 other studies actively enrolling participants."

Answered by AI

Has Dose Escalation received regulatory authorization from the Food and Drug Administration?

"As this is a Phase 1 clinical trial, the safety rating of Dose Escalation was assessed as 1 due to limited available data concerning efficacy and protection."

Answered by AI

Would I be an appropriate candidate for this experimental procedure?

"The requirement to join this clinical trial is a diagnosis of bone marrow failure syndrome and an age between 18 and 120 years old. There are 78 slots available for the study in total."

Answered by AI

What are the key goals of this trial's design?

"This clinical study will evaluate the safety of imatinib on patients with pathogenic or likely pathogenic germline RUNX1 mutations over a 1 month and 3-month duration. The primary outcomes include clinically meaningful change from baseline ISTH-BAT, transformation in platelet aggregation score compared to healthy control groups, and improvement in platelet dense granule morphology as measured by electron microscopy."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency

2023. "Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06090669.

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