Clinical Research Directory
Browse clinical research sites, groups, and studies.
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
Sponsor: Vertex Pharmaceuticals Incorporated
Summary
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participants with severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.
Official title: A Phase 1/2/3 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease
Key Details
Gender
All
Age Range
12 Years - 35 Years
Study Type
INTERVENTIONAL
Enrollment
63
Start Date
2018-11-27
Completion Date
2025-07-07
Last Updated
2026-09-16
Healthy Volunteers
No
Interventions
Exa-cel
Administered by IV infusion following myeloablative conditioning with busulfan.
Locations (17)
Lucile Packard Children's Hospital of Stanford University
Palo Alto, California, United States
Ann & Robert Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
University of Illinois at Chicago Hospitals and Health Systems
Chicago, Illinois, United States
Columbia University Medical Center (21+ years)
New York, New York, United States
Columbia University Medical Center (≤21 years)
New York, New York, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
St. Jude Children's Research Hospital
Memphis, Tennessee, United States
The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers
Nashville, Tennessee, United States
Methodist Children's Hospital/Texas Transplant Institute
San Antonio, Texas, United States
Hopital Universitaire des Enfants Reine Fabiola (HUDERF)
Brussels, Belgium
The Hospital for Sick Children
Toronto, Canada
Hopital Necker Enfants Malades
Paris, France
University Hospital Duesseldorf
Düsseldorf, Germany
Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medicine, Paediatric Haemotology, Oncology and Stem Cell Transplantation
Regensburg, Germany
Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica Ospedale Pediatrico Bambino Gesu - IRCCS
Rome, Italy
Imperial College Healthcare NHS Trust, Hammersmith Hospital
London, United Kingdom
Royal London and St Bartholomew's Hospital, Pathology and Pharmacy Building
London, United Kingdom