CRISPR stem cell therapy shows promise for kids with sickle cell
NCT ID NCT05329649
First seen Nov 01, 2025 · Last updated Jun 20, 2026 · Updated 31 times
Summary
This phase 3 trial tests a single dose of CTX001, a CRISPR-edited stem cell therapy, in 13 children with severe sickle cell disease who have not responded to standard treatments. The goal is to see if it can prevent severe pain crises for at least a year. Participants receive their own modified stem cells after chemotherapy to prepare the body.
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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.
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
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
CTX001 (CRISPR-edited stem cells)
What this could lead to
If it works, this could offer children with severe sickle cell disease a long-term reduction in painful crises and hospital stays.
What could go wrong
This is a small, early-phase study (13 kids) and requires intensive chemotherapy before the infusion, which carries serious risks. Long-term effects are unknown.
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.