Actively Recruiting

Phase 2
Age: 18Years +
All Genders
NCT06261060

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

Led by M.D. Anderson Cancer Center · Updated on 2026-04-15

6

Participants Needed

1

Research Sites

207 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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

CONDITIONS

Official Title

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

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Provided signed, informed consent before starting any study procedures
  • Aged 18 years or older at the time of signing informed consent
  • Confirmed pathogenic or likely pathogenic germline RUNX1 variant as determined by expert panel
  • Willing to provide bone marrow samples at screening and after sirolimus treatment
  • Platelet count of 50,000 per microliter or higher
  • Adequate kidney function with estimated glomerular filtration rate above 30 mL/min/1.73m2
  • Adequate liver function with AST and ALT less than three times the upper limit of normal and total bilirubin less than 1.5 times upper limit of normal
  • Adequate heart function with left ventricular ejection fraction above 50%
Not Eligible

You will not qualify if you...

  • Known allergy to sirolimus
  • History of lymphoma or other blood cancers
  • Uncontrolled bleeding
  • Prior diagnosis of myelodysplastic syndrome or other blood malignancy
  • Treatment with sirolimus, related drugs, or certain other medications within 28 days before study start
  • Use of strong CYP3A4 inhibitors or inducers or other drugs affecting sirolimus levels within 7 days before study start
  • Use of cannabidiol within 7 days before study start
  • Heart attack within 6 months before study start or congestive heart failure above class II
  • Total cholesterol above 300 mg/dL or triglycerides above 400 mg/dL
  • Arterial thrombosis such as stroke within 6 months before study start
  • Infection requiring intravenous antibiotics within 1 week before study start
  • Live vaccinations within 28 days before study start
  • Known chronic viral infections like hepatitis B or C, HIV, Epstein-Barr, or tuberculosis
  • Women who are pregnant, may become pregnant, or breastfeeding

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

MD Anderson Cancer Center

Houston, Texas, United States, 77030

Actively Recruiting

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Research Team

C

Courtney DiNardo, MD

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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