Recruiting
Phase 2

Sirolimus

Sponsor:

M.D. Anderson Cancer Center

Code:

NCT06261060

Conditions

Familial Platelet Disorder

Hematopoietic

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Sirolimus

Study Details

Brief summary:

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

Conditions

Familial Platelet Disorder

Hematopoietic

Study ID

NCT06261060

Start date

Jun 20, 2024

Status verified date

Sep, 2026

Completion date

Jun 11, 2028

Anticipated

Primary completion date

Jun 11, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

10 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Arm 1

Participants will visit the study clinic 2 times during Week 1, one (1) time during Weeks 2-4, and then 1 time every 2 weeks after that (Weeks 6, 8, 10, and so on) until Week 22 (Month 6). Then participants will have a follow-up visit at Week 24 and again at Week 52 (Month 12). Participants will take sirolimus by mouth every day, at about the same time each day. Swallow the tablet(s) whole with a full glass of water (about 1 cup). Do not crush or chew the tablet(s). Participants may take sirolimus with or without food.

Interventions

Sirolimus

Given by PO

Primary outcome measure

  • Safety and adverse events (AEs) [ Time Frame: Through study completion; an average of 1 year ]

Central Contacts and Locations

Central contacts

Courtney DiNardo, MD

(713) 794-1141cdinardo@mdanderson.org

Locations

MD Anderson Cancer Center

Recruiting

Houston, Texas, United States, 77030

Contacts

Courtney DiNardo, MD

713-794-1141cdinardo@mdanderson.org

Principal Investigator:

Courtney DiNardo, MD

More Information

Sponsor

M.D. Anderson Cancer Center

Last update posted

Sep 10, 2026

Last verified

Sep, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-11. This information was provided to ClinicalTrials.gov by M.D. Anderson Cancer Center on 2026-09-10.