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RecruitingPhase 2NCT06261060

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

Sponsor

M.D. Anderson Cancer Center

Enrollment

6 participants

Start Date

Jun 20, 2024

Study Type

INTERVENTIONAL

Conditions

Familial Platelet DisorderHematopoietic

Summary

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

Eligibility

Min Age: 18 Years

Inclusion Criteria8

  • 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 Criteria14

  • 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
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Interventions

DRUGSirolimus

Given by PO

Locations(1)

MD Anderson Cancer Center

Houston, Texas, United States

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NCT06261060