Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency

PHASE1RecruitingINTERVENTIONAL
Enrollment

75

Participants

Timeline

Start Date

December 19, 2023

Primary Completion Date

October 30, 2026

Study Completion Date

October 30, 2027

Conditions
Inherited Bone Marrow Failure SyndromeFamilial Platelet Disorder With Predisposition to Myeloid Malignancies
Interventions
DRUG

imatinib

Imatinib at 300-400 mg PO QD based on arm assignment/dose level

DEVICE

TruSight Oncology

Assay sequencing platform to identify pathogenic genetic mutations in DNA and RNA

Trial Locations (1)

20892

RECRUITING

National Institutes of Health Clinical Center, Bethesda

All Listed Sponsors
lead

National Cancer Institute (NCI)

NIH