Study Summary
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.
Want to learn more about this trial?
Request More InfoInterventions
CTX001BIOLOGICAL
Administered by IV infusion following myeloablative conditioning with busulfan
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| 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 |