CRISPR gene therapy aims to free kids from lifelong blood transfusions
NCT ID NCT05356195
First seen Oct 31, 2025 · Last updated Jun 19, 2026 · Updated 32 times
Summary
This phase 3 trial tests a single dose of CTX001, a CRISPR gene therapy made from the child's own blood stem cells, for children with transfusion-dependent beta-thalassemia. The goal is to help them become transfusion-free for at least 12 months. Sixteen children will receive the treatment after chemotherapy to prepare their bone marrow.
Disclaimer
Read more
Show less
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for HEMOGLOBINOPATHIES are added.
Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
Genom att skicka in godkänner du våra Användarvillkor
Locations
-
Great Ormond Street Hospital for Children
London, United Kingdom
-
Hospital for Sick Children - Hematology
Toronto, Canada
-
IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica
Rome, Italy
-
St.Mary's Hospital - Children's Clinical Research Facility
London, United Kingdom
-
TriStar Medical Group Children's Specialists - Pediatric Oncology
Nashville, Tennessee, 37203, United States
-
University Hospital Dusseldorf - 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 (Exagamglogene autotemcel) - a CRISPR gene therapy made from the patient's own blood stem cells
What this could lead to
If it works, this could allow children with severe beta-thalassemia to stop needing regular blood transfusions for at least a year or more.
What could go wrong
This is an early-phase study with only 16 participants, so results may not apply to everyone. The treatment requires strong chemotherapy beforehand, which carries serious risks like infection and infertility.
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.