{"disease":{"id":"familial-platelet-disorder","name":"familial platelet disorder"},"drugs":{"marketed":[],"pipeline":[],"offLabel":[],"totalMarketed":0,"totalPipeline":0},"trials":{"data":[{"nct_id":"NCT06090669","title":"Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency","phase":"PHASE1","overall_status":"RECRUITING","enrollment_count":75,"lead_sponsor_name":"National Cancer Institute (NCI)","has_results":false},{"nct_id":"NCT06261060","title":"Low-Dose Sirolimus to Increase Hematopoietic Function in Patients With RUNX1 Familial Platelet Disorder","phase":"PHASE2","overall_status":"RECRUITING","enrollment_count":6,"lead_sponsor_name":"M.D. Anderson Cancer Center","has_results":false},{"nct_id":"NCT06414889","title":"Protocol Title: Safety and Feasibility of Autologous CD34+ Hematopoietic Stem Cells Mobilization and Apheresis in Participants With RUNX1 Familial Platelet Disorder","phase":"PHASE1","overall_status":"RECRUITING","enrollment_count":4,"lead_sponsor_name":"M.D. Anderson Cancer Center","has_results":false}],"total":3},"guidelines":[],"source":"Drug Landscape verified database"}