First step toward gene therapy: stem cell collection tested in rare blood disorders
NCT ID NCT07585136
First seen May 13, 2026 · Last updated May 15, 2026 · Updated 1 time
Summary
This early-phase study tests whether it is safe and possible to collect blood stem cells from 12 people aged 18-25 with bone marrow failure syndromes. Participants receive two drugs (Filgrastim and plerixafor) to move stem cells into the bloodstream for collection. The goal is to learn how to gather enough healthy cells to enable future gene therapy, not to treat the disease directly.
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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.
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Saint Jude Children's Research Hospital
Memphis, Tennessee, 38105-2794, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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