A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia
Completed · Phase 2/Phase 3
Conditions studied: Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies
In brief
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participans with transfusion-dependent β-thalassemia (TDT). 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
- NCT03655678
- Run by
- Vertex Pharmaceuticals Incorporated
- People needed
- 59
- Starts
- 2018-09-14
- Expected to finish
- 2025-11-13
- Last updated by the study team
- 2025-12-17
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 — Palo Alto, California, United States
- Ann & Robert Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
- Columbia University Medical Center (21+ years) — New York, New York, United States
- Columbia University Medical Center — New York, New York, United States
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
- The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers — Nashville, Tennessee, United States
- The Hospital for Sick Children — Toronto, Canada
- British Columbia Children's Hospital — Vancouver, Canada
- Universitätsklinikum Düsseldorf Hospital Duesseldorf — Düsseldorf, Germany
- Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medicine — Regensburg, Germany
- University Hospital Tübingen — Tübingen, Germany
- Ospedale Pediatrico Bambino Gesù, IRCCS — Rome, Italy
- Imperial College Healthcare NHS Trust, Hammersmith Hospital — London, United Kingdom
- University College London Hospitals NHS Foundation Trust — London, United Kingdom
Full record on ClinicalTrials.gov
Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.