RECRUITING

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

Conditions

Familial Platelet Disorder Hematopoietic

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

Official Title

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

Quick Facts

Study Start:2024-06-20

Study Completion:2028-06-11

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06261060

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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%	* 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

Inclusion Criteria

Exclusion 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%

* 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

Contacts and Locations

Study Contact

Courtney DiNardo, MD

CONTACT

(713) 794-1141

cdinardo@mdanderson.org

Principal Investigator

Courtney DiNardo, MD

PRINCIPAL_INVESTIGATOR

M.D. Anderson Cancer Center

Study Locations (Sites)

MD Anderson Cancer Center

Houston, Texas, 77030

United States

Collaborators and Investigators

Sponsor: M.D. Anderson Cancer Center

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

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-06-20

Study Completion Date2028-06-11

Study Record Updates

Study Start Date2024-06-20

Study Completion Date2028-06-11

Terms related to this study

Additional Relevant MeSH Terms

Familial Platelet Disorder
Hematopoietic