NCT06414889

Protocol Title: Safety and Feasibility of Autologous CD34+ Hematopoietic Stem Cells Mobilization and Apheresis in Participants With RUNX1 Familial Platelet Disorder

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 4 (estimated)
• Sponsor: M.D. Anderson Cancer Center · Academic / Other
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Accepted

Summary

To evaluate the safety and feasibility of collecting hematopoietic stem cells (HSC) in participants with RUNX1-FPD.

Detailed description

Primary Objective: \- To evaluate the safety of harvesting HSCs in participants with RUNX1 FPD Secondary Objective \- To evaluate the feasibility and other relevant information of collecting HSCs from participants with RUNX1 FPD

Conditions

• RUNX1 Familial Platelet Disorder

Interventions

Type	Name	Description
DRUG	G-CSF (filgrastim or biosimilar)	Given by IV or SC
PROCEDURE	Apheresis	Given by procedure
DRUG	Plerixafor	Given by IV

Timeline

• Start date: 2024-05-20
• Primary completion: 2027-06-30
• Completion: 2027-06-30
• First posted: 2024-05-16
• Last updated: 2025-12-26

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study