Low-Dose Sirolimus for the Treatment of RUNX1 Familial Platelet Disorder
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This page only displays public information reproduced from the official ClinicalTrials.gov record NCT07844694. It adds no medical advice, opinions, or eligibility judgments. The official record remains the source of truth for eligibility criteria, enrollment, and contact information.
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Official title
Low-Dose Sirolimus to Increase Hematopoietic Function in Patients With RUNX1 Familial Platelet Disorder - Part II
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
This phase I trial studies the safety and side effects of low-dose sirolimus in treating patients with RUNX1 familial platelet disorder (FPD). RUNX1-FPD is a rare inherited disorder with symptoms such as mild to moderately low platelet count, abnormal platelet function, and an increased risk of developing cancers like myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). It is caused by changes (variants) in the RUNX1 gene that is passed down (inherited) from an affected parent. Sirolimus is typically given to help prevent organ rejection in patients receiving kidney transplants. However, sirolimus may also be able to prevent RUNX1-FPD from progressing to cancers like MDS and AML by targeting another protein called mTORC1. Sirolimus may be a safe treatment for patients with RUNX1-FPD.
Study identification
- NCT ID
- NCT07844694
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 1
- Enrollment
- 6 participants
Conditions and interventions
Interventions
- Biospecimen Collection Procedure
- Bone Marrow Aspiration Procedure
- Bone Marrow Biopsy Procedure
- Questionnaire Administration Other
- Sirolimus Drug
Procedure · Other · Drug
Eligibility (public fields only)
- Age range
- 18 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Oct 5, 2026 (Actual)
- Primary completion
- Jun 30, 2030 (Estimated)
- Completion
- Dec 31, 2030 (Estimated)
- Last update posted
- Oct 13, 2026
2026 – 2030
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| OHSU Knight Cancer Institute | Portland | Oregon | 97239 | Recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT07844694, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Oct 13, 2026 · Synced Nov 14, 2026
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