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Low-Dose Sirolimus to Increase Hematopoietic Function in Patients With RUNX1 Familial Platelet Disorder | Clinical Trials | Clareo Health

RECRUITINGPHASE2

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

NCT06261060•M.D. Anderson Cancer Center

View on ClinicalTrials.gov

Summary

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

Detailed Description

Primary Objective: • Evaluate the safety and tolerability of low-dose sirolimus in participants with RUNX1 familial platelet disorder (RUNX1-FPD) Secondary Objectives: * Evaluate increases in platelet counts during and after treatment with low-dose sirolimus * Evaluate changes in somatic mutation variant allele frequency (VAF) * Monitor the rate of somatic mutation acquisition (ie, mutation burden) * Assess change in platelet aggregation score * Measure the change from baseline in bleeding score (ISTH-BAT) * Evaluate change in mTORC1 downstream signaling (pS6/EBP) Exploratory Objectives: * Measure rescue of elevated cytokine profiles * Evaluate reversal of myeloid skewing using flow cytometry * Determine changes in bone marrow (eg, megakaryocytic atypia and cellularity) * Assess changes in patient-reported outcomes measures (eg, EORTC and PRO-CTCAE) * Describe the pharmacokinetics of sirolimus in patients with RUNX1-FPD * Determine the correlation between sirolimus trough levels and each endpoint

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

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
+4 more criteria

Locations (1)

MD Anderson Cancer Center

Houston, Texas, United States

Key Dates

Start Date

June 20, 2024

Primary Completion Date

June 11, 2026

Completion Date

June 11, 2028

Last Updated

October 24, 2025

Enrollment

ESTIMATED participants

Conditions

Familial Platelet DisorderHematopoietic

Interventions

Sirolimus

DRUG

Contacts

Courtney DiNardo, MD

CONTACT

(713) 794-1141 cdinardo@mdanderson.org

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: October 24, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

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

Inclusion Criteria

Exclusion Criteria

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