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3 recruiting studies matching your filters
Phase 1
Phase Ib Study of Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency
Background: Runt-related transcription factor 1 (RUNX1) gene regulates the formation of blood cells. People with mutations of this gene may bleed or bruise ea…
Inherited Bone Marrow Failure SyndromeFamilial Platelet Disorder With Predisposition to Myeloid Malignancies
National Cancer Institute (NCI)NCT06090669
Phase 1
Protocol Title: Safety and Feasibility of Autologous CD34+ Hematopoietic Stem Cells Mobilization and Apheresis in Participants With RUNX1 Familial Platelet Disorder
To evaluate the safety and feasibility of collecting hematopoietic stem cells (HSC) in participants with RUNX1-FPD.
RUNX1 Familial Platelet Disorder
M.D. Anderson Cancer CenterNCT06414889
Phase 2
Low-Dose Sirolimus to Increase Hematopoietic Function in Patients With RUNX1 Familial Platelet Disorder
To learn about the safety and effects of low-dose sirolimus in participants with RUNX1-FPD.
Familial Platelet DisorderHematopoietic
M.D. Anderson Cancer CenterNCT06261060