CRISPR gene therapy: a Long-Term safety check for blood disease patients
NCT ID NCT04208529
First seen Jun 27, 2026 · Last updated Aug 12, 2026 · Updated 2 times
Summary
This study follows 160 children and adults with beta-thalassemia or sickle cell disease who received a one-time treatment of their own CRISPR-edited stem cells (CTX001). The goal is to monitor long-term safety, including any new cancers or blood disorders, and to see how well the therapy works over time. Participants join after completing earlier studies, and there are no new exclusion criteria.
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
-
Ann & Robert H. Lurie Children's Hospital of Chicago - Hematology
Chicago, Illinois, 60611, United States
-
Center for Pediatric Clinical Studies (CPCS)
Klinik Für Kinder- Und Jugendmedizin, Germany
-
Great Ormond Street Hospital for Children
London, United Kingdom
-
Hammersmith Hospital - Haematology Dept
London, United Kingdom
-
Herbert Irving Pavilion - Hematology
New York, New York, 10032, United States
-
Hopital Universitaire des Enfants Reine Fabiola (HUDERF) - Hematology
Brussels, Belgium
-
Hospital for Sick Children - Hematology
Toronto, Canada
-
IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica
Rome, Italy
-
Levine Children's Hospital - Hematology
Charlotte, North Carolina, 28203, United States
-
Lucile Packard Children's Hospital
Palo Alto, California, 94304, United States
-
Methodist Healthcare System of San Antonio, Methodist Hospital, Methodist Children's Hospital
San Antonio, Texas, 78229, United States
-
New York Presbyterian Hospital - Morgan Stanley Children's Hospital
New York, New York, 10032, United States
-
Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medicine
Regensburg, Germany
-
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105, United States
-
St. Paul's Hospital - Hematology
Vancouver, Canada
-
The Children's Hospital of Philadelphia - Hematology
Philadelphia, Pennsylvania, 19104, United States
-
Toronto General Hospital - Hematology
Toronto, Canada
-
TriStar Medical Group Children's Specialists - Pediatric Oncology
Nashville, Tennessee, 37203, United States
-
University College London Hospital NHS Foundation - Main
London, United Kingdom
-
University Hospital Duesseldorf - 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 Co-Designed support help parents embrace sickle cell care?
- Can community hospitals in zambia master severe disease care?
- A Pocket-Sized coach for sickle cell: can an app turn Pill-Taking into a habit?
- Can a phone app keep teens on track with sickle cell treatment?
- A common amino acid may calm sickle cell pain crises — a trial puts it to the test