First step toward gene therapy for rare anemia: can stem cells be harvested?
NCT ID NCT07186179
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study checks if people with Diamond Blackfan Anemia (ages 3-30) can produce enough blood stem cells for future gene therapy. Participants receive two drugs to move stem cells from bone marrow into the blood, then researchers measure the cell count. No actual stem cell collection is done—this is a feasibility test to see if enough cells can be mobilized.
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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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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.
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Study contacts
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Contact
Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Cohen Children's Medical Center
RECRUITINGNew Hyde Park, New York, 11040, United States
Contact
Contact Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••