New gene therapy aims to transform sickle cell treatment
NCT ID NCT04819841
First seen Nov 01, 2025 · Last updated Apr 25, 2026 · Updated 21 times
Summary
This study tests a new gene therapy called nula-cel for people with severe sickle cell disease. The treatment modifies a patient's own blood stem cells to produce healthy hemoglobin instead of sickle hemoglobin. About 15 participants aged 12 to 40 will receive the therapy to see if it is safe and works. The goal is to reduce or eliminate the severe pain crises and other complications of sickle cell disease.
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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Children's Hospital Los Angeles
RECRUITINGLos Angeles, California, 90027, United States
Contact
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Lucile Packard Children's Hospital
RECRUITINGPalo Alto, California, 94304, United States
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Nationwide Children's Hospital
RECRUITINGColumbus, Ohio, 43205, United States
Contact Email: •••••@•••••
Contact Email: •••••@•••••
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Washington University
RECRUITINGSt Louis, Missouri, 63110, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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