Protocol Title: Safety and Feasibility of Autologous CD34+ Hematopoietic Stem Cells Mobili
To evaluate the safety and feasibility of collecting hematopoietic stem cells (HSC) in participants with RUNX1-FPD.
| Condition(s) | RUNX1 Familial Platelet Disorder |
|---|---|
| Status | Recruiting |
| Phase | Phase 1 |
| Study type | Interventional |
| Summary | To evaluate the safety and feasibility of collecting hematopoietic stem cells (HSC) in participants with RUNX1-FPD. |
| Who can participate | 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)-certified genetic sequencing report. 4. Clearance by apheresis team to proceed 5. Have systolic blood pressure ≤ 170 mm Hg and diastolic blood pressure ≤ 95 mmHg 6. Are eligible for HSCT per institution req |
| Ages | 18 Years to 75 Years |
| Sex | All |
| Accepts healthy volunteers | Yes |
| Lead sponsor | M.D. Anderson Cancer Center |
| Locations | Houston, Texas, United States |
| Start date | 2024-05-20 |
| NCT ID | NCT06414889 |
| Official listing | https://clinicaltrials.gov/study/NCT06414889 |