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

NCT06414889 | Recruiting Phase 1 DMC FDA Drug

• 2 other identifiers: 2023-0799NCI-2024-04300
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

RUNX1 Familial Platelet Disorder

Outcome Measures

Primary Outcomes (1)

• Safety and adverse events (AEs)

  Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0

  Through study completion; an average of 1 year.

Study Arms (1)

Autologous CD34+ Hematopoietic Stem Cells Mobilization and Apheresis

EXPERIMENTAL

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).

Drug: G-CSF (filgrastim or biosimilar); Procedure: Apheresis; Drug: Plerixafor

Interventions

G-CSF (filgrastim or biosimilar) DRUG

Given by IV or SC

Also known as: FILGRASTIM SD/01

Autologous CD34+ Hematopoietic Stem Cells Mobilization and Apheresis

Apheresis PROCEDURE

Given by procedure

Autologous CD34+ Hematopoietic Stem Cells Mobilization and Apheresis

Plerixafor DRUG

Given by IV

Autologous CD34+ Hematopoietic Stem Cells Mobilization and Apheresis

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Participants who meet all of the following criteria are eligible to be included in the study:
• 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.
• 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)
• 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.
• Have hemoglobin ≥ 7.5 g/dL as assessed within 24 hours prior to the procedure

You may not qualify if:

• Participants who meet any of the following criteria are excluded from the study:
• 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:
• Persistent aspartate transaminase, alanine transaminase, or direct bilirubin value \> 5× the upper limit of normal (ULN) at screening
• Screening prothrombin time (PT) or partial thromboplastin time (PTT) \> 1.5× ULN
• Have had prior HSCT or gene therapy
• Have history of concomitant sickle cell disease
• Have been treated with an investigational drug within 30 days of screening or 5 half-lives (whichever is longer)
• Have a positive test result for HIV, hepatitis B virus (HBV), or hepatitis C virus (HCV) at screening

Sponsors & Collaborators

• M.D. Anderson Cancer Center: lead
• RUNX1 Foundation: collaborator

Study Sites (1)

MD Anderson Cancer Center

Houston, Texas, 77030, United States

RECRUITING

Related Links

• MD Anderson Cancer Center

MeSH Terms

Interventions

Granulocyte Colony-Stimulating Factor; Filgrastim; Biosimilar Pharmaceuticals; pegfilgrastim; Blood Component Removal; plerixafor

Intervention Hierarchy (Ancestors)

Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors; Biological Products; Complex Mixtures; Therapeutics

Study Officials

• Chitra Hosing

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Chitra Hosing, MD

CONTACT

(713) 745-3219; cmhosing@mdanderson.org

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: BASIC SCIENCE
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 10, 2024
• First Posted: May 16, 2024
• Study Start: May 20, 2024
• Primary Completion (Estimated): June 30, 2027
• Study Completion (Estimated): June 30, 2027
• Last Updated: December 26, 2025

Record last verified: 2025-12

Locations

US (1)