Stem cell harvest trial aims to unlock gene therapy for rare blood disorders
NCT ID NCT07585136
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 2 times
Summary
This early-phase study at St. Jude Children's Research Hospital will test whether it is safe and possible to collect blood stem cells from 12 young adults with inherited bone marrow failure syndromes. Participants will receive two drugs (filgrastim and plerixafor) to move stem cells into the bloodstream, then undergo a procedure called leukapheresis to collect them. The goal is not to treat the disease directly, but to advance research into gene therapies that could one day correct the underlying genetic cause.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Filgrastim and plerixafor (drugs to mobilize stem cells), followed by leukapheresis (procedure to collect stem cells)
What this could lead to
If successful, this study could show that collecting stem cells from patients with bone marrow failure is safe and feasible, paving the way for future gene therapies.
What could go wrong
This is a very small, early-phase trial (12 participants) focused on safety and feasibility, not treatment. The approach may not work or could cause side effects like low blood counts or infection.
Disclaimer
Read more
Show less
This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for BONE MARROW FAILURE SYNDROME are added.
By submitting, you agree to our Terms of use
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
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
Saint Jude Children's Research Hospital
Memphis, Tennessee, 38105-2794, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••