59
Participants
Start Date
September 14, 2018
Primary Completion Date
December 8, 2025
Study Completion Date
December 8, 2025
CTX001
Administered by IV infusion following myeloablative conditioning with busulfan
Columbia University Medical Center (21+ years), New York
Columbia University Medical Center, New York
Children's Hospital of Philadelphia, Philadelphia
The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers, Nashville
Ann & Robert Lurie Children's Hospital of Chicago, Chicago
Lucile Packard Children's Hospital, Palo Alto
The Hospital for Sick Children, Toronto
British Columbia Children's Hospital, Vancouver
Universitätsklinikum Düsseldorf Hospital Duesseldorf, Düsseldorf
Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medicine, Regensburg
University Hospital Tübingen, Tübingen
Ospedale Pediatrico Bambino Gesù, IRCCS, Rome
Imperial College Healthcare NHS Trust, Hammersmith Hospital, London
University College London Hospitals NHS Foundation Trust, London
Collaborators (1)
CRISPR Therapeutics
INDUSTRY
Vertex Pharmaceuticals Incorporated
INDUSTRY