RECRUITING

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

Conditions

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

Official Title

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

Quick Facts

Study Start:2024-05-20

Study Completion:2027-06-30

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06414889

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years to 75 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:Yes

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
1. Are aged ≥ 18 to 75 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) 9. Have a platelet count ≥ 50,000/μL for initiation of apheresis, assessed within 24 hours prior to the procedure, or, if \< 50,000/μL are administered platelets on the day of the collection 10. Have hemoglobin ≥ 7.5 g/dL as assessed within 24 hours prior to the procedure	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 2. Screening prothrombin time (PT) or partial thromboplastin time (PTT) \> 1.5× ULN 9. Have had prior HSCT or gene therapy 10. Have history of concomitant sickle cell disease 11. Have been treated with an investigational drug within 30 days of screening or 5 half-lives (whichever is longer) 12. Have a positive test result for HIV, hepatitis B virus (HBV), or hepatitis C virus (HCV) at screening 1. Participants with positive hepatitis B core antibody (HbcAb) and/or hepatitis B-e antibody (HbeAb) are eligible provided viral load is negative by quantitative polymerase chain reaction (qPCR). 2. Participants who are positive for anti-hepatitis C antibody are eligible as long as they have a negative HCV viral load by qPCR. 13. Have a positive infectious disease panel at screening for human T-lymphotropic virus 1 or 2 (HTLV-1 and HTLV-2), or syphilis (rapid plasma 24 reagin \[RPR\]) 14. Have clinically significant and active bacterial, viral, fungal, or parasitic infection at screening 15. Have a white blood cell (WBC) count \< 2 × 109/L 16. Have a left ventricular ejection fraction \< 45% 17. Have a screening estimated glomerular filtration rate \< 60 mL/min/1.73 m2 18. Have a diagnosis of a significant psychiatric disorder that could seriously impede the ability to participate in the study 19. For women of childbearing potential: are pregnant or breastfeeding or lack adequate contraception 20. Are unable to comply with the study procedures, as assessed by the investigator

Inclusion Criteria

Exclusion Criteria

1. Are aged ≥ 18 to 75 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)
9. Have a platelet count ≥ 50,000/μL for initiation of apheresis, assessed within 24 hours prior to the procedure, or, if \< 50,000/μL are administered platelets on the day of the collection
10. Have hemoglobin ≥ 7.5 g/dL as assessed within 24 hours prior to the procedure

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
2. Screening prothrombin time (PT) or partial thromboplastin time (PTT) \> 1.5× ULN
9. Have had prior HSCT or gene therapy
10. Have history of concomitant sickle cell disease
11. Have been treated with an investigational drug within 30 days of screening or 5 half-lives (whichever is longer)
12. Have a positive test result for HIV, hepatitis B virus (HBV), or hepatitis C virus (HCV) at screening
1. Participants with positive hepatitis B core antibody (HbcAb) and/or hepatitis B-e antibody (HbeAb) are eligible provided viral load is negative by quantitative polymerase chain reaction (qPCR).
2. Participants who are positive for anti-hepatitis C antibody are eligible as long as they have a negative HCV viral load by qPCR.
13. Have a positive infectious disease panel at screening for human T-lymphotropic virus 1 or 2 (HTLV-1 and HTLV-2), or syphilis (rapid plasma 24 reagin \[RPR\])
14. Have clinically significant and active bacterial, viral, fungal, or parasitic infection at screening
15. Have a white blood cell (WBC) count \< 2 × 109/L
16. Have a left ventricular ejection fraction \< 45%
17. Have a screening estimated glomerular filtration rate \< 60 mL/min/1.73 m2
18. Have a diagnosis of a significant psychiatric disorder that could seriously impede the ability to participate in the study
19. For women of childbearing potential: are pregnant or breastfeeding or lack adequate contraception
20. Are unable to comply with the study procedures, as assessed by the investigator

Contacts and Locations

Study Contact

Chitra Hosing, MD

CONTACT

(713) 745-3219

cmhosing@mdanderson.org

Principal Investigator

Chitra Hosing

PRINCIPAL_INVESTIGATOR

M.D. Anderson Cancer Center

Study Locations (Sites)

MD Anderson Cancer Center

Houston, Texas, 77030

United States

Collaborators and Investigators

Sponsor: M.D. Anderson Cancer Center

Chitra Hosing, PRINCIPAL_INVESTIGATOR, M.D. Anderson Cancer Center

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-05-20

Study Completion Date2027-06-30

Study Record Updates

Study Start Date2024-05-20

Study Completion Date2027-06-30

Terms related to this study

Additional Relevant MeSH Terms

RUNX1 Familial Platelet Disorder