Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency

Recruiting Phase 1 Interventional Study
Inherited Bone Marrow Failure Syndrome Familial Platelet Disorder With Predisposition to Myeloid Malignancies
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Healthy Volunteers Welcome You do not need to have the condition being studied to take part.
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At a Glance
Age
18 – 120
Sex
Any
Trial phase
Phase 1
Study type
Interventional
Purpose
Treatment
Participants needed
75 (estimated)
Sponsor
National Cancer Institute (NCI) · NIH
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About This Trial
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…
Trial Locations
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Eligibility Criteria
* INCLUSION CRITERIA- AFFECTED PARTICIPANTS ONLY * Affected participants must have a confirmed pathogenic or likely pathogenic germline RUNX1 variant by history. ClinGen expert variant curation panel criteria for pathogenicity will be utilized. * Affected participants must have a history of clinical…
Contacts

Valentina Bolanos, R.N.

(240) 858-7666

valentina.bolanos@nih.gov

CONTACT

Lea C Cunningham, M.D.

(301) 642-1633

lea.cunningham@nih.gov

CONTACT