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Your journey
1What's a trial
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More info
You can access this
clinical trial
if you have
β-thalassemia Major
and you are
between 12 and 35
years old
This is a medium phase trial determining
the safety and dosage of the new treatment, as well as its efficacy.
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The purpose

This is a non-randomized, open label, multi-site, single-dose, phase 1/2 study in up to 18 participants (including at least 3 adolescents between 12 and 17 years of age, inclusive) with β-thalassemia major. The study will evaluate the safety and efficacy of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin BB305 Drug Product [autologous CD34+ hematopoietic stem cells transduced with LentiGlobin BB305 lentiviral vector encoding the human βA-T87Q-globin gene].

Provided treatments

  • Genetic: LentiGlobin BB305 Drug Product

Locations near you

Unfortunately, there are no recruiting locations near you. Please check the list with all locations below.
Tris trial is registered with FDA with number: NCT01745120. The sponsor of the trial is bluebird bio and it is looking for 19 volunteers for the current phase.
Official trial title:
A Phase 1/2 Open Label Study Evaluating the Safety and Efficacy of Gene Therapy in Subjects With β-Thalassemia Major by Transplantation of Autologous CD34+ Cells Transduced Ex Vivo With a Lentiviral βA-T87Q-Globin Vector (LentiGlobin® BB305 Drug Product)