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

NCT06414889•M.D. Anderson Cancer Center

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

Eligibility

Age

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)-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 requirements
•7. Have a Lansky (age \< 16 years)/Karnofsky performance status of ≥ 70 (see Appendix 2, Section 13.2).
•8. Are willing and able to comply with protocol-defined contraceptive requirements (see Appendix 3 Section 13.3)
+3 more criteria

Exclusion Criteria

•Participants who meet any of the following criteria are excluded from the study:
•1. Participants with cognitive impairments and/or any serious unstable pre-existing medical condition or psychiatric disorder that can interfere with safety or with obtaining informed consent or compliance with study procedures.
•2. Have uncontrolled bleeding
•3. Are using supplemental oxygen
•4. Have known severe splenomegaly (≥ 20 cm)
•5. Have a diagnosis of MDS or hematologic malignancies, as defined by WHO hematolymphoid tumor classification fifth edition (Khourey et al 2022) hematolymphoid tumor classification fifth edition (Khourey et al 2022)
•6. Have recent prior malignancies except resected basal cell carcinoma or treated cervical carcinoma in situ Note: Cancer treated with curative intent \< 5 years previously may be allowed following approval from the study investigator. Cancer treated with curative intent \> 5 years previously is allowed.
•7. Have any prior or current myeloproliferative or a significant coagulation or immunodeficiency disorder
•8. Have advanced liver disease, defined as any of the following:
•1. Persistent aspartate transaminase, alanine transaminase, or direct bilirubin value \> 5× the upper limit of normal (ULN) at screening
+15 more criteria

Locations (1)

MD Anderson Cancer Center

Houston, Texas, United States

Key Dates

Start Date

May 20, 2024

Primary Completion Date

June 30, 2027

Completion Date

June 30, 2027

Last Updated

December 26, 2025

Enrollment

ESTIMATED participants

Conditions

RUNX1 Familial Platelet Disorder

Interventions

G-CSF (filgrastim or biosimilar)

DRUG

Apheresis

PROCEDURE

Plerixafor

DRUG

Contacts

Chitra Hosing, MD

CONTACT

(713) 745-3219 cmhosing@mdanderson.org