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.
Interested in this trial?
Submit an InquiryInterventions
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 |