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

Completed · Phase 3 · NCT03207009

Conditions: Beta-Thalassemia

At a glance

Research payments reported, 2025
$24,220
U.S. investigators named
1
Sites paid
1
Planned or actual enrollment
19

About the trial

Sponsor
Genetix Biotherapeutics Inc.
Tested
LentiGlobin BB305 Drug Product (genetic)
Dates
Started 2017-06-08; completion 2022-11-15
Results posted
Yes

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.

From ClinicalTrials.gov, last updated 2024-03-07. Full record, eligibility and contacts (opens in a new tab)

Industry funding

Genetix Biotherapeutics Inc reported $24,220 in research payments for this study in 2025 (2 payments).

Products named: Zynteglo

Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.

U.S. investigators named on this study

Clinicians the company named as principal investigators in its Open Payments reports.

Sites paid to run it

Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.