RUNX1 familial platelet disorder
Clinical trials for RUNX1 familial platelet disorder explained in plain language.
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Could a low-dose drug boost blood health in rare platelet disorder?
Disease control Recruiting nowThis 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 receive the drug and are monitored for side effects and changes in blo…
Matched conditions: RUNX1 FAMILIAL PLATELET DISORDER
Phase 2 • Sponsor: M.D. Anderson Cancer Center • Aim: Disease control
Last updated Sep 12, 2026 00:00 UTC
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First step toward a cure? stem cell harvesting trial launches for rare platelet disorder
Knowledge-focused Recruiting nowThis phase 1 trial at M.D. Anderson Cancer Center is testing whether it is safe to collect stem cells from people with RUNX1 familial platelet disorder, a rare inherited condition that raises the risk of bleeding and leukemia. Four participants will receive drugs to mobilize stem…
Matched conditions: RUNX1 FAMILIAL PLATELET DISORDER
Phase 1 • Sponsor: M.D. Anderson Cancer Center • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:06 UTC