Table 1.
Currently active gene therapy trials for β-thalassemias
| Protocol no. (study name) and status | Phase | Gene | Vector | Location | Sponsor | Condition | Conditioning regimen | Intervention | Start date |
|---|---|---|---|---|---|---|---|---|---|
| NCT02453477 (TIGET-BTHAL) recruiting | 1/2 | β-globin | GLOBE | Italy | IRCCS San Raffaele | β-TM | Myeloablative | Transplantation of HSCs transduced ex vivo with an LV (intrabone injection) | May 2015 |
| NCT01745120 (HGB-204) active, not recruiting | 1/2 | βA-T87Q-globin | BB305 | United States, Thailand, Australia | Bluebird Bio | β-TM | Myeloablative | Transplantation of HSCs transduced ex vivo with an LV | August 2013 |
| NCT01745120 (HGB-205) active, not recruiting | 1/2 | βA-T87Q-globin | BB305 | France | Bluebird Bio | β-TM and severe sickle cell anemia | Myeloablative | Transplantation of HSCs transduced ex vivo with an LV | July 2013 |
| NCT02906202 recruiting | 3 | βA-T87Q-globin | BB305 | France, Germany, Greece, Italy, Thailand, United States | Bluebird Bio | Myeloablative | Transplantation of HSCs transduced ex vivo with an LV | August 2016 | |
| NCT01639690 active, not recruiting | 1 | β-globin | TNS9.3.55 | United States | Memorial Sloan-Kettering Cancer Center | β-TM | Partial cytoreduction (Bu 8 mg/kg) for 3 patients, myeloablative (Bu 14 mg/kg) for 1 patient | Transplantation of HSCs transduced ex vivo with an LV | July 2012 |
Bu, busulfan; HSC, hematopoietic stem cell; LV, lentiviral vector; TM, thalassemia major.