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Low-Dose Sirolimus for RUNX1 Familial Platelet Disorder

This study is looking at a medication called sirolimus to see if it can help people with RUNX1 Familial Platelet Disorder (RUNX1-FPD). This condition affects how your body makes blood cells, especially platelets. Researchers want to understand if a low dose of sirolimus is safe and how well people tolerate it. They will also check if it helps increase platelet counts and improves bleeding. You may be able to join if you are 18 or older and have a confirmed genetic change in the RUNX1 gene. The study is currently unclear about its recruitment status and plans to enroll 6 participants.

Study design
This is an interventional study, meaning participants will receive a treatment. It plans to enroll 6 participants.
What's involved
You will need to provide signed informed consent and be willing to provide a bone marrow sample.
Compensation
Not stated in the trial record.
Follow-up
Safety and side effects will be measured throughout the study, for an average of 1 year.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT06261060

Low-Dose Sirolimus to Increase Hematopoietic Function in Patients With RUNX1 Familial Platelet Disorder

Active, Not Recruiting
PHASE2Ages 18+InterventionalTreatment
M.D. Anderson Cancer Center
~6 participants
Updated 2026-06-12 on ClinicalTrials.gov
What's tested:Sirolimus

At a glance

Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Safety and adverse events (AEs)
Measured over Through study completion; an average of 1 year

Conditions

Familial Platelet Disorder
Hematopoietic

Explore related trials

Familial Platelet DisorderRUNX1 Gene

Where it's being run

1 sites across 1 states
Texas1

Study leadership

  • Courtney DiNardo, MD · PRINCIPAL_INVESTIGATOR · M.D. Anderson Cancer Center

Who to contact

This trial hasn't published a contact. View it on ClinicalTrials.gov

Do you actually qualify for this trial?

Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.

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Eligibility criteria

Inclusion

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

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

What this trial measures

  • Safety and adverse events (AEs)Through study completion; an average of 1 year

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