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NCT06261060

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

Recruiting now Phase 2 Last updated 15 April 2026
What this trial tests

Phase 2 trial testing Sirolimus in Familial Platelet Disorder in 6 participants. Currently enrolling.

Timeline
20 June 2024
Primary endpoint
11 June 2026
11 June 2028

Quick facts

Lead sponsorM.D. Anderson Cancer Center
PhasePhase 2
StatusRecruiting now
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment6
Start date20 June 2024
Primary completion11 June 2026
Estimated completion11 June 2028
Sites1 location across United States

Drugs / interventions tested

Conditions studied

Sponsor

M.D. Anderson Cancer Center — full company profile →

Who can join

18 and older, any sex, with Familial Platelet Disorder or Hematopoietic. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

  1. Clonal hematopoiesis in myeloid malignancies and solid tumors.
    Cai X, Bowman RL, Trowbridge JJ. · · 2025 · cited 3× · PMID 40681796 · DOI 10.1038/s43018-025-01014-0

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Other trials of Sirolimus

Trials testing the same drug.

Other M.D. Anderson Cancer Center trials

Trials by the same sponsor.

Verify against primary sources

Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT06261060.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing