CI

At a glance

ClinicalIndex Comparison Record
Phase 2Active· 6 enrolled / 6 target
Drug / intervention
Sirolimusdrug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT06261060
NCT06261060Phase 2ActiveOn Track (0.3/mo)
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Low-Dose Sirolimus to Increase Hematopoietic Function in Patients With RUNX1 Familial Platelet Disorder

M.D. Anderson Cancer Center·interventional·Posted Feb 15, 2024·Updated Jun 12, 2026

In Brief

A Phase 2 clinical trial evaluating Sirolimus for Familial Platelet Disorder and Hematopoietic. Active but no longer recruiting, targeting 6 participants across 1 site.

Detailed Summary

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

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsFamilial Platelet Disorder, Hematopoietic
CountriesUnited States
Collaborators--

Timeline

Phase 2Active
2025202620272028
First PostedFeb 15, 2024
Enrollment StartJun 20, 2024
Primary CompletionJun 11, 2028
TodayJul 1, 2026
Enrollment to primary: 4.0 yearsPosted 2.4 years agoPrimary completion in 1.9 years

Arms & Interventions

Arm 1experimental

Participants will visit the study clinic 2 times during Week 1, one (1) time during Weeks 2-4, and then 1 time every 2 weeks after that (Weeks 6, 8, 10, and so on) until Week 22 (Month 6). Then participants will have a follow-up visit at Week 24 and again at Week 52 (Month 12). Participants will take sirolimus by mouth every day, at about the same time each day. Swallow the tablet(s) whole with a full glass of water (about 1 cup). Do not crush or chew the tablet(s). Participants may take sirolimus with or without food.

Drug: Sirolimus

Interventions

Sirolimusdrug

Given by PO