Low-Dose Sirolimus to Increase Hematopoietic Function in Patients With RUNX1 Familial Platelet Disorder
NCT06261060 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 6
Last updated 2026-04-15
Summary
To learn about the safety and effects of low-dose sirolimus in participants with RUNX1-FPD.
Conditions
- Familial Platelet Disorder
- Hematopoietic
Interventions
- DRUG
-
Sirolimus
Given by PO
Sponsors & Collaborators
-
M.D. Anderson Cancer Center
lead OTHER
Principal Investigators
-
Courtney DiNardo, MD · M.D. Anderson Cancer Center
Study Design
- Allocation
- NA
- Purpose
- TREATMENT
- Masking
- NONE
- Model
- SINGLE_GROUP
Eligibility
- Min Age
- 18 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2024-06-20
- Primary Completion
- 2026-06-11
- Completion
- 2028-06-11
- FDA Drug
- Yes
Countries
- United States
Study Locations
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