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.
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