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A Long-term Follow-up Study in Participants Who Received CTX001
Sponsor: Vertex Pharmaceuticals Incorporated
Summary
This is a multi-site, open- label rollover study to evaluate the long-term safety and efficacy of CTX001 in pediatric and adult participants who received CTX001 in parent studies 111 (NCT03655678) 141 (NCT05356195) or 161 (NCT05477563) (transfusion-dependent β-thalassemia \[TDT\] studies) or Study 121 (NCT03745287) or 151 (NCT05329649) or 161(NCT05477563) (severe sickle cell disease \[SCD\] studies).
Official title: A Long-term Follow-up Study of Subjects With β-thalassemia or Sickle Cell Disease Treated With Autologous CRISPR-Cas9 Modified Hematopoietic Stem Cells (CTX001)
Key Details
Gender
All
Age Range
2 Years - Any
Study Type
INTERVENTIONAL
Enrollment
160
Start Date
2021-01-20
Completion Date
2039-09-30
Last Updated
2026-03-25
Healthy Volunteers
No
Conditions
Interventions
CTX001
CTX001 infusion.
Locations (20)
Lucile Packard Children's Hospital
Palo Alto, California, United States
Ann & Robert H. Lurie Children's Hospital of Chicago - Hematology
Chicago, Illinois, United States
Herbert Irving Pavilion - Hematology
New York, New York, United States
New York Presbyterian Hospital - Morgan Stanley Children's Hospital
New York, New York, United States
Levine Children's Hospital - Hematology
Charlotte, North Carolina, United States
The Children's Hospital of Philadelphia - Hematology
Philadelphia, Pennsylvania, United States
St. Jude Children's Research Hospital
Memphis, Tennessee, United States
TriStar Medical Group Children's Specialists - Pediatric Oncology
Nashville, Tennessee, United States
Methodist Healthcare System of San Antonio, Methodist Hospital, Methodist Children's Hospital
San Antonio, Texas, United States
Hopital Universitaire des Enfants Reine Fabiola (HUDERF) - Hematology
Brussels, Belgium
Hospital for Sick Children - Hematology
Toronto, Canada
Toronto General Hospital - Hematology
Toronto, Canada
St. Paul's Hospital - Hematology
Vancouver, Canada
University Hospital Duesseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology
Düsseldorf, Germany
Center for Pediatric Clinical Studies (CPCS)
Klinik Für Kinder- Und Jugendmedizin, Germany
Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medicine
Regensburg, Germany
IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica
Rome, Italy
Great Ormond Street Hospital for Children
London, United Kingdom
Hammersmith Hospital - Haematology Dept
London, United Kingdom
University College London Hospital NHS Foundation - Main
London, United Kingdom