CRISPR gene therapy patients monitored for years in groundbreaking study
NCT ID NCT04208529
Summary
This study tracks the long-term safety and effectiveness of a one-time CRISPR gene therapy called CTX001 in people with sickle cell disease or beta-thalassemia. It follows 160 children and adults who have already received the therapy in earlier studies. The main goal is to monitor for any long-term side effects and see if the benefits, like reducing or eliminating the need for blood transfusions or painful crises, are sustained over many years.
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Contacts and locations
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Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago - Hematology
Chicago, Illinois, 60611, United States
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Center for Pediatric Clinical Studies (CPCS)
Klinik Für Kinder- Und Jugendmedizin, Germany
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Great Ormond Street Hospital for Children
London, United Kingdom
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Hammersmith Hospital - Haematology Dept
London, United Kingdom
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Herbert Irving Pavilion - Hematology
New York, New York, 10032, United States
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Hopital Universitaire des Enfants Reine Fabiola (HUDERF) - Hematology
Brussels, Belgium
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Hospital for Sick Children - Hematology
Toronto, Canada
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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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Lucile Packard Children's Hospital
Palo Alto, California, 94304, United States
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Methodist Healthcare System of San Antonio, Methodist Hospital, Methodist Children's Hospital
San Antonio, Texas, 78229, United States
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New York Presbyterian Hospital - Morgan Stanley Children's Hospital
New York, New York, 10032, United States
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Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medicine
Regensburg, Germany
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St. Jude Children's Research Hospital
Memphis, Tennessee, 38105, United States
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St. Paul's Hospital - Hematology
Vancouver, Canada
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The Children's Hospital of Philadelphia - Hematology
Philadelphia, Pennsylvania, 19104, United States
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Toronto General Hospital - Hematology
Toronto, Canada
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TriStar Medical Group Children's Specialists - Pediatric Oncology
Nashville, Tennessee, 37203, United States
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University College London Hospital NHS Foundation - Main
London, United Kingdom
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University Hospital Duesseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology
Düsseldorf, Germany
Conditions
Explore the condition pages connected to this study.