Protocol Title: Safety and Feasibility of Autologous CD34+ Hematopoietic Stem Cells Mobilization and Apheresis in Participants With RUNX1 Familial Platelet Disorder
NCT ID: NCT06414889
Last Updated: 2025-12-26
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1
4 participants
INTERVENTIONAL
2024-05-20
2027-06-30
Brief Summary
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Detailed Description
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\- 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
Conditions
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Study Design
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NA
SINGLE_GROUP
BASIC_SCIENCE
NONE
Study Groups
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Autologous CD34+ Hematopoietic Stem Cells Mobilization and Apheresis
On Days 1-5, participants will receive a Granulocyte colony-stimulating factor (G-CSF), such as filgrastim, as an injection or by vein over about 5 minutes.
If the study doctor thinks it is needed, participants will also receive plerixafor as an injection under the skin on Day 5 (and 6, if you have 2 days of apheresis).
G-CSF (filgrastim or biosimilar)
Given by IV or SC
Apheresis
Given by procedure
Plerixafor
Given by IV
Interventions
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G-CSF (filgrastim or biosimilar)
Given by IV or SC
Apheresis
Given by procedure
Plerixafor
Given by IV
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
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)
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
a. If the apheresis team decides that a central venous catheter (CVC) is to be placed, platelet count should be ≥ 50,000 prior to catheter placement.
10. Have hemoglobin ≥ 7.5 g/dL as assessed within 24 hours prior to the procedure
Exclusion Criteria
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
18 Years
75 Years
ALL
Yes
Sponsors
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RUNX1 Foundation
UNKNOWN
M.D. Anderson Cancer Center
OTHER
Responsible Party
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Principal Investigators
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Chitra Hosing
Role: PRINCIPAL_INVESTIGATOR
M.D. Anderson Cancer Center
Locations
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MD Anderson Cancer Center
Houston, Texas, United States
Countries
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Central Contacts
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Facility Contacts
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Chitra Hosing, MD
Role: primary
Related Links
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MD Anderson Cancer Center
Other Identifiers
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NCI-2024-04300
Identifier Type: OTHER
Identifier Source: secondary_id
2023-0799
Identifier Type: -
Identifier Source: org_study_id