First step toward a cure? stem cell harvesting trial launches for rare platelet disorder
NCT ID NCT06414889
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase 1 trial at M.D. Anderson Cancer Center is testing whether it is safe to collect stem cells from people with RUNX1 familial platelet disorder, a rare inherited condition that raises the risk of bleeding and leukemia. Four participants will receive drugs to mobilize stem cells, which are then collected through a procedure called apheresis. The goal is simply to see if the collection process is safe and feasible, not yet to treat the disorder.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- G-CSF (filgrastim) and plerixafor
- What this could lead to
- If successful, this could enable future gene therapy or stem cell treatments for this rare bleeding disorder.
- What could go wrong
- This is a very early, small trial with only 4 participants, so results may not apply broadly. The stem cell collection process carries risks like infection or bleeding.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
MD Anderson Cancer Center
RECRUITINGHouston, Texas, 77030, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.