Could a low-dose drug boost blood health in rare platelet disorder?
NCT ID NCT06261060
First seen Nov 01, 2025 · Last updated Jun 06, 2026 · Updated 30 times
Summary
This study tests whether low-dose sirolimus can safely improve blood cell function in adults with RUNX1 familial platelet disorder, a genetic condition that raises bleeding and leukemia risks. Six participants will take sirolimus and be monitored for side effects and changes in blood counts. The goal is to see if the drug can help control the disease, not cure it.
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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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MD Anderson Cancer Center
RECRUITINGHouston, Texas, 77030, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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