Houston, TXNCT06414889Now EnrollingIRB Ready

RUNX1 Familial Platelet Disorder Clinical Trial in Houston, TX

Access cutting-edge runx1 familial platelet disorder treatment through this clinical trial at a research site in Houston. Study-provided care at no cost to qualified participants.

Sponsored by M.D. Anderson Cancer Center

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Expert Care in Houston

Access runx1 familial platelet disorder specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related runx1 familial platelet disorder treatment provided free

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Check if you qualify for this runx1 familial platelet disorder clinical trial in Houston, TX

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Why Participate?

  • No-Cost Study Care

  • Local to Houston

    Convenient for TX residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Houston site if eligible
  4. 4Begin participation

About This RUNX1 Familial Platelet Disorder Study in Houston

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

Sponsor: M.D. Anderson Cancer Center

Who Can Participate

Inclusion Criteria

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

Exclusion Criteria

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
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).
Participants who are positive for anti-hepatitis C antibody are eligible as long as they have a negative HCV viral load by qPCR.
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 \[RPR\])
Have clinically significant and active bacterial, viral, fungal, or parasitic infection at screening
Have a white blood cell (WBC) count \< 2 × 109/L
Have a left ventricular ejection fraction \< 45%
Have a screening estimated glomerular filtration rate \< 60 mL/min/1.73 m2
Have a diagnosis of a significant psychiatric disorder that could seriously impede the ability to participate in the study
For women of childbearing potential: are pregnant or breastfeeding or lack adequate contraception
Are unable to comply with the study procedures, as assessed by the investigator

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Houston?

Yes, this clinical trial (NCT06414889) has an active research site in Houston, TX that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

RUNX1 Familial Platelet Disorder Treatment Options in Houston, TX

If you're searching for runx1 familial platelet disorder treatment options in Houston, TX, this clinical trial (NCT06414889) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Houston research site is actively enrolling participants for this clinical trial. You'll receive care from experienced runx1 familial platelet disorder specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all runx1 familial platelet disorder clinical trials near you to find additional studies recruiting in your area.

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