CRISPR hope for kids with sickle cell: One-Time treatment may stop pain crises
NCT ID NCT05329649
First seen Nov 01, 2025 · Last updated May 22, 2026 · Updated 28 times
Summary
This study tests a one-time gene-editing treatment (CTX001) in children with severe sickle cell disease who cannot take hydroxyurea. The treatment uses the child's own blood stem cells, modified with CRISPR-Cas9, to produce healthy red blood cells. The goal is to prevent severe pain crises and hospital stays for at least 12 months. The study is in Phase 3 and involves 13 participants.
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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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