CRISPR gene therapy could free kids with thalassemia from lifelong blood transfusions
NCT ID NCT05356195
First seen Oct 31, 2025 · Last updated Jun 09, 2026 · Updated 30 times
Summary
This study tests a one-time gene therapy (CTX001) in 16 children with transfusion-dependent beta-thalassemia, a severe blood disorder requiring regular transfusions. The treatment uses CRISPR to modify the child's own blood stem cells so they produce healthy red blood cells. The main goal is to see if children can go without transfusions for at least 12 months. While not a cure (lifelong monitoring is needed), it aims to greatly reduce or eliminate the need for transfusions.
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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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Great Ormond Street Hospital for Children
London, United Kingdom
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Hospital for Sick Children - Hematology
Toronto, Canada
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IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica
Rome, Italy
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St.Mary's Hospital - Children's Clinical Research Facility
London, United Kingdom
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TriStar Medical Group Children's Specialists - Pediatric Oncology
Nashville, Tennessee, 37203, United States
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University Hospital Dusseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology
Düsseldorf, Germany
Conditions
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