Houston, TXNCT06261060Now EnrollingIRB Ready

Familial Platelet Disorder Clinical Trial in Houston, TX

Access cutting-edge familial platelet disorder treatment through this phase 2 clinical trial at a research site in Houston. Study-provided care at no cost to qualified participants.

Sponsored by M.D. Anderson Cancer Center

Quick Self-Assessment

See if you qualify for this Houston location

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Expert Care in Houston

Access familial platelet disorder specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related familial platelet disorder treatment provided free

Apply for This Houston Location

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

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Your information is protected and will only be shared with the research team.

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 Familial Platelet Disorder Study in Houston

To learn about the safety and effects of low-dose sirolimus in participants with RUNX1-FPD.

Sponsor: M.D. Anderson Cancer Center

Who Can Participate

Inclusion Criteria

Participants has provided signed, informed consent before initiation of any study specific procedures
Aged ≥18 years at the time of signing the informed consent
Confirmed P/LP germline RUNX1 variant per ClinGen Myeloid Malignancy Variant Curation Expert Panel (MM-VCEP) RUNX1-specific variant curation rules80
Participants must be willing to provide bone marrow sample at time of screening and at the end of treatment with sirolimus
Platelet count of ≥50,000/µL
Adequate renal function: estimated glomerular filtration rate based on Modification of Diet in Renal Disease (MDRD) calculation, \>30 mL/min/1.73m2
Adequate hepatic function: aspartate aminotransferase (AST) and alanine aminotransferase (ALT) \<3 × upper limit of normal (ULN) and total bilirubin \<1.5 × ULN
Adequate cardiac function: left ventricular ejection fraction \>50%

Exclusion Criteria

Known allergy to sirolimus
History of lymphoma or other hematologic malignancies
Uncontrolled bleeding
Any prior diagnosis of myelodysplastic syndrome or other hematologic malignancy using International Working Group criteria
Prior treatment with sirolimus or a rapalog, mTOR inhibitor, or B-cell-depleting therapy within 28 days before study day 1
Treatment with strong inhibitors of cytochrome P450 3A4 (CYP3A4; eg, ketoconazole, voriconazole, itraconazole, erythromycin, telithromycin, and clarithromycin), strong inducers of CYP3A4 (eg, rifampin and rifabutin), other drugs that could increase sirolimus blood concentrations (eg, bromocriptine, cimetidine, cisapride, clotrimazole, danazol, diltiazem, fluconazole, letermovir, protease inhibitors \[eg, ritonavir, indinavir, boceprevir, and telaprevir\], metoclopramide, nicardipine, troleandomycin, and verapamil), other drugs that could decrease sirolimus blood concentrations (eg, carbamazepine, phenobarbital, phenytoin, rifapentine, St. John's Wort \[Hypericum perforatum\]), or drugs with blood concentrations that could increase (eg, verapamil) within 7 days before study day 1
Use of cannabidiol, which can increase blood levels of sirolimus, within 7 days before study day 1
Myocardial infarction within 6 months before study day 1, congestive heart failure (New York Heart Association \> class II)
Total cholesterol \>300 mg/dL or triglyceride \>400 mg/dL
Arterial thrombosis (eg, stroke or transient ischemic attack) within 6 months before study day 1
Infection requiring intravenous anti-infective treatment within 1 week of study day 1
Live vaccines (eg, measles, mumps, rubella, oral polio, BCG, yellow fever, varicella, and TY21a typhoid) within 28 days before study day 1
Known diagnosis of chronic viral infection (eg, hepatitis B or C or HIV, and Epstein-Barr) or tuberculosis
Women who are pregnant, may become pregnant, or who are breastfeeding

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 (NCT06261060) 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.

Familial Platelet Disorder Treatment Options in Houston, TX

If you're searching for familial platelet disorder treatment options in Houston, TX, this clinical trial (NCT06261060) 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 phase 2 clinical trial. You'll receive care from experienced 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 familial platelet disorder clinical trials near you to find additional studies recruiting in your area.

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