Actively Recruiting

Phase 1
Age: 18Years - 75Years
All Genders
Healthy Volunteers
NCT06414889

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

Led by M.D. Anderson Cancer Center · Updated on 2025-12-26

4

Participants Needed

1

Research Sites

162 weeks

Total Duration

On this page

Sponsors

M

M.D. Anderson Cancer Center

Lead Sponsor

R

RUNX1 Foundation

Collaborating Sponsor

AI-Summary

What this Trial Is About

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

CONDITIONS

Official Title

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

Who Can Participate

Age: 18Years - 75Years
All Genders
Healthy Volunteers

Eligibility Criteria

Eligible

You may qualify if you...

  • Aged 18 to 75 years
  • Willing and able to provide informed consent
  • Confirmed diagnosis of RUNX1 Familial Platelet Disorder by genetic testing
  • Cleared by the apheresis team to proceed
  • Systolic blood pressure ≤ 170 mm Hg and diastolic blood pressure ≤ 95 mmHg
  • Eligible for hematopoietic stem cell transplant as per institution requirements
  • Lansky (for under 16 years) or Karnofsky performance status of 70 or higher
  • Willing and able to comply with contraceptive requirements
  • Platelet count ≥ 50,000/µL for apheresis initiation, or will receive platelets on collection day
  • Hemoglobin level ≥ 7.5 g/dL within 24 hours prior to procedure
Not Eligible

You will not qualify if you...

  • Cognitive impairments or serious unstable medical or psychiatric conditions interfering with safety or consent
  • Uncontrolled bleeding
  • Use of supplemental oxygen
  • Known severe splenomegaly (20 cm or larger)
  • Diagnosis of myelodysplastic syndrome or hematologic malignancies
  • Recent malignancies except certain treated cancers with investigator approval
  • Prior or current myeloproliferative, significant coagulation, or immunodeficiency disorders
  • Advanced liver disease defined by specific abnormal lab values
  • Previous hematopoietic stem cell transplant or gene therapy
  • History of sickle cell disease
  • Treatment with investigational drug within 30 days or 5 half-lives
  • Positive tests for HIV, hepatitis B or C with active viral load
  • Positive infectious disease panel for HTLV-1, HTLV-2, or syphilis
  • Active significant infections
  • White blood cell count less than 2 x 10^9/L
  • Left ventricular ejection fraction below 45%
  • Estimated glomerular filtration rate under 60 mL/min/1.73 m2
  • Significant psychiatric disorder preventing study participation
  • Pregnant or breastfeeding women or those not using adequate contraception
  • Unable to comply with study procedures as assessed by investigator

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

MD Anderson Cancer Center

Houston, Texas, United States, 77030

Actively Recruiting

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Research Team

C

Chitra Hosing, MD

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

BASIC_SCIENCE

Number of Arms

1

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