Study Summary
This is a single-arm, multi-site, single-dose, Phase 3 study in approximately 18 participants less than or equal to (\<=) 50 years of age with transfusion-dependent β-thalassemia (TDT), who have a β0/β0, β0/IVS-I-110, or IVS-I-110/IVS-I-110 genotype. The study will evaluate the efficacy and safety of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin BB305 Drug Product.
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LentiGlobin BB305 Drug ProductGENETIC
LentiGlobin BB305 Drug Product is administered by IV infusion following myeloablative conditioning with busulfan.
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| UCSF Benioff Children's Hospital Oakland | Oakland | California | United States |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | United States |
| Hopital d'enfants de la Timone | Marseille | France | |
| Hannover Medical School | Hanover | Germany | |
| University of Heidelberg | Heidelberg | Germany | |
| General Hospital of Thessaloniki 'G.Papanikolaou' | Thessaloniki | Greece | |
| IRCCS Ospedale Pediatrico Babino Gesu | Rome | Italy | |
| University College London Hospital | London | United Kingdom |