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for people ages 12–50 (full criteria)
at Oakland, California and other locations
study started
estimated completion:



This is a single-arm, multi-site, single-dose, Phase 3 study in approximately 15 subjects ≥12 and ≤50 years of age with transfusion-dependent β-thalassemia (TDT), who have a β0 mutation at both alleles of the β-globin (HBB) gene (i.e. β0/β0). The study will evaluate the efficacy and safety of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin BB305 Drug Product.

Official Title

A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects With Transfusion-dependent β-Thalassemia, Who Have a β0/β0 Genotype, by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With a Lentiviral βA-T87Q-Globin Vector in Subjects ≥12 and ≤50 Years of Age


Beta-Thalassemia Thalassemia


You can join if…

Open to people ages 12–50

  • Subjects between 12 and 50 years of age, inclusive, at the time of consent or assent(as applicable), and able to provide written consent or assent.
  • Diagnosis of TDT with a history of at least 100 mL/kg/year of pRBCs or ≥8 transfusions of pRBCs per year for the prior 2 years.
  • Clinically stable and eligible to undergo HSCT.
  • Treated and followed for at least the past 2 years in a specialized center that maintained detailed medical records, including transfusion history.

You CAN'T join if...

  • Presence of a mutation characterized as other then β0 (e.g., β+, βE, βC) on at least one HBB allele.
  • Positive for presence of human immunodeficiency virus type 1 or 2 (HIV-1 and HIV-2),hepatitis B virus (HBV), or hepatitis C (HCV).
  • A white blood cell (WBC) count <3×109/L, and/or platelet count <100×109/L not related to hypersplenism.

  • Uncorrected bleeding disorder.
  • Any prior or current malignancy.
  • Prior HSCT.
  • Advanced liver disease.
  • A cardiac T2* <10 ms by magnetic resonance imaging (MRI).
  • Any other evidence of severe iron overload that, in the investigator's opinion,warrants exclusion.
  • Participation in another clinical study with an investigational drug within 30 days of Screening.
  • Any other condition that would render the subject ineligible for HSCT, as determined by the attending transplant physician or investigator.
  • Prior receipt of gene therapy.
  • Pregnancy or breastfeeding in a postpartum female or absence of adequate contraception for fertile subjects.
  • A known and available HLA-matched family donor.


  • UCSF Benioff Children's Hospital Oakland accepting new patients
    Oakland, California, 94609, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago accepting new patients
    Chicago, Illinois, 60611, United States
  • University College London Hospital accepting new patients
    London, United Kingdom
  • IRCCS Ospedale Pediatrico Babino Gesu accepting new patients
    Rome, Italy


accepting new patients
Start Date
Completion Date
bluebird bio
Phase 3
Study Type
Last Updated
December 12, 2017
I’m interested in this study!