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Clinical Trial Summary

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].


Clinical Trial Description

Subject participation for this study will be 2 years. Subjects who enroll in this study will be asked to participate in a subsequent long-term follow up study that will monitor the safety and efficacy of the treatment they receive for up to 13 years post-transplant. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT01745120
Study type Interventional
Source bluebird bio
Contact
Status Completed
Phase Phase 1/Phase 2
Start date August 2013
Completion date February 21, 2018

See also
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Terminated NCT04205435 - β-globin Restored Autologous HSC in β-thalassemia Major Patients Phase 1/Phase 2
Not yet recruiting NCT04592458 - Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients Phase 1
Withdrawn NCT01724138 - An Open Label Study to Evaluate the Pharmacokinetics, Safety, Tolerability and Efficacy of Deferasirox Administered to Chinese Patients With β-thalassemia Major Aged From 2 to Less Than 6 Years Old Phase 4
Recruiting NCT05776173 - Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent β-thalassemia N/A