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5 recruiting studies matching your filters

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

Estimated

~$10 – $5,625

National Cancer Institute (NCI)NCT06090669

Phase 1

Low-Dose Sirolimus to Increase Hematopoietic Function in Patients With RUNX1 Familial Platelet Disorder - Part II

This phase I trial studies the safety and side effects of low-dose sirolimus in treating patients with RUNX1 familial platelet disorder (FPD). RUNX1-FPD is a r…

Familial Platelet Disorder With Associated Myeloid Malignancy

Estimated

~$10 – $7,500

OHSU Knight Cancer InstituteNCT07844694

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

Estimated

~$10 – $6,250

M.D. Anderson Cancer CenterNCT06261060

ATHN Transcends: A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment in People With Non-Neoplastic Hematologic Disorders

In parallel with the growth of ATHN's clinical studies, the number of new therapies for all blood disorders is increasing significantly. Some of the recently F…

Hematologic DisorderBleeding DisorderConnective Tissue Disorder+10 more

Estimated

~$10 – $1,125

American Thrombosis and Hemostasis NetworkNCT04398628

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

Estimated

~$10 – $6,250

M.D. Anderson Cancer CenterNCT06414889