63
Participants
Start Date
November 27, 2018
Primary Completion Date
July 7, 2025
Study Completion Date
July 7, 2025
CTX001
Administered by IV infusion following myeloablative conditioning with busulfan.
Columbia University Medical Center (21+ years), New York
Columbia University Medical Center (≤21 years), New York
Children's Hospital of Philadelphia, Philadelphia
The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers, Nashville
St. Jude Children's Research Hospital, Memphis
Ann & Robert Lurie Children's Hospital of Chicago, Chicago
University of Illinois at Chicago Hospitals and Health Systems, Chicago
Methodist Children's Hospital/Texas Transplant Institute, San Antonio
Lucile Packard Children's Hospital of Stanford University, Palo Alto
Hopital Universitaire des Enfants Reine Fabiola (HUDERF), Brussels
The Hospital for Sick Children, Toronto
Hopital Necker Enfants Malades, Paris
University Hospital Duesseldorf, Düsseldorf
Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medicine, Paediatric Haemotology, Oncology and Stem Cell Transplantation, Regensburg
Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica Ospedale Pediatrico Bambino Gesu - IRCCS, Rome
Imperial College Healthcare NHS Trust, Hammersmith Hospital, London
Royal London and St Bartholomew's Hospital, Pathology and Pharmacy Building, London
Collaborators (1)
CRISPR Therapeutics
INDUSTRY
Vertex Pharmaceuticals Incorporated
INDUSTRY