CRISPR gene therapy aims to free kids from lifelong blood transfusions
NCT ID NCT05356195
First seen Jun 27, 2026 · Last updated Jul 02, 2026 · Updated 2 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.
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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for BETA-THALASSEMIA are added.
By submitting, you agree to our Terms of use
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.
Contacts and locations
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
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Could muscle loss predict blood cancer outcomes?
- Can a safer dose of an old drug shield vulnerable children from deadly fungal infections?
- Could a simple exercise routine strengthen bones in thalassemia?
- Gene therapy aims to free patients from lifelong blood transfusions
- Double-Pronged immune cells take aim at stubborn blood cancers
- Triple therapy aims to overcome antibody barrier in Half-Matched stem cell transplants