Protocol Title: Safety and Feasibility of Autologous CD34+ Hematopoietic Stem Cells Mobilization and Apheresis in Participants With RUNX1 Familial Platelet Disorder
M.D. Anderson Cancer Center
Summary
To evaluate the safety and feasibility of collecting hematopoietic stem cells (HSC) in participants with RUNX1-FPD.
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
Eligibility
- Age range
- 18–75 years
- Sex
- All
- Healthy volunteers
- Yes
Inclusion Criteria: Participants who meet all of the following criteria are eligible to be included in the study: 1. Are aged ≥ 18 to 75 years a. Once a favorable review of safety has been completed by the SMC in 3 participants aged ≥ 18 years, the study will be opened to participants aged ≥ 12 years. 2. Are willing and able to provide informed consent, as appropriate (either directly or through a legally authorized representative \[LAR\]), as described in Appendix 1, Section 13.1 3. Have a confirmed diagnosis of RUNX1 FPD, verified by a Clinical Laboratory Improvement Amendments (CLIA)-…
Interventions
- DrugG-CSF (filgrastim or biosimilar)
Given by IV or SC
- ProcedureApheresis
Given by procedure
- DrugPlerixafor
Given by IV
Location
- MD Anderson Cancer CenterHouston, Texas