CRISPR gene therapy aims to free patients from lifelong transfusions
NCT ID NCT05477563
First seen Nov 06, 2025 · Last updated May 22, 2026 · Updated 34 times
Summary
This study tests a single dose of a CRISPR-based gene therapy (CTX001) in 26 adults with transfusion-dependent beta-thalassemia or severe sickle cell disease. The treatment uses the patient's own blood stem cells, which are edited to produce more fetal hemoglobin, potentially reducing or eliminating the need for regular blood transfusions. Participants will be monitored for safety and effectiveness over time.
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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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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica
RECRUITINGRome, Italy
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King Faisal Specialist Hospital & Research Centre - Riyadh - Hematology
RECRUITINGAl Mathar Ash Shamali, Saudi Arabia
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Levine Children's Hospital - Hematology
RECRUITINGCharlotte, North Carolina, 28203, United States
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New York Presbyterian Hospital - Morgan Stanley Children's Hospital
RECRUITINGNew York, New York, 10032, United States
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TriStar Medical Group Children's Specialists - Pediatric Oncology
RECRUITINGNashville, Tennessee, 37203, United States
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University Hospital Dusseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology
RECRUITINGDüsseldorf, Germany
Conditions
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