Gene therapy hope for sickle cell patients, but study pulled before start
NCT ID NCT05951205
First seen Mar 26, 2026 · Last updated May 25, 2026 · Updated 6 times
Summary
This study aimed to test a single dose of exa-cel (CTX001) in adolescents and adults with a severe form of sickle cell disease called HbSC. The treatment was designed to increase fetal hemoglobin to reduce pain crises and other complications. However, the study was withdrawn before enrolling any participants, so no results are available.
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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.
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