A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
Completed · Phase 2/Phase 3
Conditions studied: Sickle Cell Disease, Hematological Diseases, Hemoglobinopathies
In brief
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in subjects 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.
Key facts
- Study ID
- NCT03745287
- Run by
- Vertex Pharmaceuticals Incorporated
- People needed
- 63
- Starts
- 2018-11-27
- Expected to finish
- 2025-07-07
- Last updated by the study team
- 2025-08-11
Who can join
Age: 12 and older, up to 35. Sex: any. Healthy volunteers: not accepted.
Where it is running
- 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
Full record on ClinicalTrials.gov
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