A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia

NCTID	NCT03207009 (View at clinicaltrials.gov)
Description	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. (Show More)
Development Status	Approved
Indication	Beta-Thalassemia Major
Disease Ontology Term	DOID:0080771
Compound Name	ZYNTEGLO
Compound Alias	betibeglogene autotemcel
Sponsor	bluebird bio
Funder Type	Industry
Recruitment Status	Completed
Enrollment Count	19
Results Posted	View Results

Target Gene/Variant	HBB
Therapy Type	Gene transfer
Therapy Route	Ex-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Autologous cells
Target Tissue/Cell	CD34+ cells
Delivery System	Viral transduction
Vector Type	BB305 LV
Editor Type	none
Dose 1	Minimum dose: 5.0E6 CD34+ cells/kg
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	9
Locations	Greece,United States,Italy,United Kingdom,France,Germany

Has US IND	True
FDA Designations	Breakthrough Therapy, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates	FDA approval granted 8/17/22, $2.8M estimated cost/treatment