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

Conditions

• Familial Platelet Disorder
• Hematopoietic

Eligibility Criteria

Sex

ALL

Age

18 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• Sirolimus — DRUG
  Given by PO

Study Details

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

Key Dates

Start date

Jun 20, 2024

Status verified

Apr 2026

Primary completion

Jun 11, 2026

Completion

Jun 11, 2028

Study Design

Enrollment

6 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: Arm 1
  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.

Primary Outcome Measure

Safety and adverse events (AEs) [ Time Frame: Through study completion; an average of 1 year ]

Central Contacts

• Courtney DiNardo, MD
  (713) 794-1141
  [email protected]

Locations (1)

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