CRISPR gene therapy offers hope for kids with sickle cell disease
NCT ID NCT05329649
First seen Nov 01, 2025 · Last updated May 15, 2026 · Updated 25 times
Summary
This study tests a single dose of a gene-edited stem cell treatment (CTX001) in 13 children with severe sickle cell disease who cannot take or did not benefit from hydroxyurea. The goal is to see if the treatment can prevent severe pain crises and hospital stays for at least a year. Because this is an early-phase trial, long-term safety and effectiveness are still being evaluated.
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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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Locations
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IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica
Rome, Italy
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Levine Children's Hospital - Hematology
Charlotte, North Carolina, 28203, United States
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St. Jude Children's Research Hospital
Memphis, Tennessee, 38105, United States
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St.Mary's Hospital - Haematology Dept
London, United Kingdom
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The Children's Hospital of Philadelphia - Hematology
Philadelphia, Pennsylvania, 19104, United States
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TriStar Medical Group Children's Specialists - Pediatric Oncology
Nashville, Tennessee, 37203, United States
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University Hospital Duesseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology
Düsseldorf, Germany
Conditions
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