Protocol Title: Safety and Feasibility of Autologous CD34+ Hematopoietic Stem Cells Mobilization and Apheresis in Participants With RUNX1 Familial Platelet Disorder

Conditions

• RUNX1 Familial Platelet Disorder

Eligibility Criteria

Sex

ALL

Age

18 Years - 75 Years

Healthy Volunteers

Accepted

Interventions

• G-CSF (filgrastim or biosimilar) — DRUG
  Given by IV or SC
• Apheresis — PROCEDURE
  Given by procedure
• Plerixafor — DRUG
  Given by IV

Study Details

To evaluate the safety and feasibility of collecting hematopoietic stem cells (HSC) in participants with RUNX1-FPD.

Key Dates

Start date

May 20, 2024

Status verified

Jun 2026

Primary completion

Jun 30, 2027

Completion

Jun 30, 2027

Study Design

Enrollment

4 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

BASIC_SCIENCE

Arms

• Experimental: Autologous CD34+ Hematopoietic Stem Cells Mobilization and Apheresis
  On Days 1-5, participants will receive a Granulocyte colony-stimulating factor (G-CSF), such as filgrastim, as an injection or by vein over about 5 minutes. If the study doctor thinks it is needed, participants will also receive plerixafor as an injection under the skin on Day 5 (and 6, if you have 2 days of apheresis).

Primary Outcome Measure

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

Central Contacts

• Chitra Hosing, MD
  (713) 745-3219
  [email protected]

Locations (1)

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