RecruitingPhase 1

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

United States4 participantsStarted 2024-05-20

Plain-language summary

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

Age range18 Years – 75 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Are aged ≥ 18 to 75 years
✓. 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
✓. Have a confirmed diagnosis of RUNX1 FPD, verified by a Clinical Laboratory Improvement Amendments (CLIA)-certified genetic sequencing report.
✓. Clearance by apheresis team to proceed
✓. Have systolic blood pressure ≤ 170 mm Hg and diastolic blood pressure ≤ 95 mmHg
✓. Are eligible for HSCT per institution requirements
✓. Have a Lansky (age \< 16 years)/Karnofsky performance status of ≥ 70 (see Appendix 2, Section 13.2).
✓. Are willing and able to comply with protocol-defined contraceptive requirements (see Appendix 3 Section 13.3)

✕. 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.
✕. Have uncontrolled bleeding
✕. Are using supplemental oxygen
✕. Have known severe splenomegaly (≥ 20 cm)
✕. 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)
✕. 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.
✕. Have any prior or current myeloproliferative or a significant coagulation or immunodeficiency disorder
✕. Have advanced liver disease, defined as any of the following:

Safety and adverse events (AEs)

Timeframe: Through study completion; an average of 1 year.

NCT IDNCT06414889

SponsorM.D. Anderson Cancer Center

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2027-06-30

Chitra Hosing, MD

Who can participate

Age range18 Years – 75 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Are aged ≥ 18 to 75 years
✓. 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
✓. Have a confirmed diagnosis of RUNX1 FPD, verified by a Clinical Laboratory Improvement Amendments (CLIA)-certified genetic sequencing report.
✓. Clearance by apheresis team to proceed
✓. Have systolic blood pressure ≤ 170 mm Hg and diastolic blood pressure ≤ 95 mmHg
✓. Are eligible for HSCT per institution requirements
✓. Have a Lansky (age \< 16 years)/Karnofsky performance status of ≥ 70 (see Appendix 2, Section 13.2).
✓. Are willing and able to comply with protocol-defined contraceptive requirements (see Appendix 3 Section 13.3)

✕. 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.
✕. Have uncontrolled bleeding
✕. Are using supplemental oxygen
✕. Have known severe splenomegaly (≥ 20 cm)
✕. 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)
✕. 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.
✕. Have any prior or current myeloproliferative or a significant coagulation or immunodeficiency disorder
✕. Have advanced liver disease, defined as any of the following: