CRISPR gene therapy: a lifelong watch begins for blood disease patients
NCT ID NCT04208529
First seen Nov 03, 2025 · Last updated May 25, 2026 · Updated 28 times
Summary
This study follows 160 children and adults with beta-thalassemia or sickle cell disease who already received an experimental CRISPR gene therapy called CTX001. Researchers will track participants for years to check for long-term side effects, new cancers, and how well the treatment keeps working. The goal is to make sure the therapy is safe and continues to control these serious blood disorders over time.
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
-
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
Conditions
Explore the condition pages connected to this study.