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for people ages up to 50 years (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 ≤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 ≤50 Years of Age


Beta-Thalassemia Thalassemia


You can join if…

Open to people ages up to 50 years

  • Subjects ≤50 years of age at the time of consent or assent (as applicable), and able to provide written consent (adults, or legal guardians, as applicable) or assent(adolescents or children). Provided that the DMC has approved enrolling subjects younger than 5 years of age, subjects younger than 5 years of age may be enrolled if they weigh a minimum of 6 kg and are reasonably anticipated to be able to provide at least the minimum number of cells required to initiate the manufacturing process.
  • Diagnosis of TDT with a history of at least 100 mL/kg/year of packed red blood cells(pRBCs) in the 2 years preceding enrollment (all subjects), or be managed under standard thalassemia guidelines with ≥8 transfusions of pRBCs per year in the 2 years preceding enrollment (subjects ≥12 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.
  • Any contraindications to the use of G-CSF and plerixafor during the mobilization of hematopoietic stem cells and any contraindications to the use of busulfan and any other medicinal products required during the myeloablative conditioning, including hypersensitivity to the active substances or to any of the excipients.


  • 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
  • Hannover Medical School not yet accepting patients
    Hannover, 30625, Germany
  • Hopital d'enfants de la Timone not yet accepting patients
    Marseille, 13385, France
  • IRCCS Ospedale Pediatrico Babino Gesu accepting new patients
    Rome, Italy
  • General Hospital of Thessaloniki 'G.Papanikolaou' accepting new patients
    Thessaloníki, Greece


accepting new patients
Start Date
Completion Date
bluebird bio
Phase 3
Study Type
Last Updated
February 6, 2018
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