This study is a multi-center, long-term safety and efficacy follow-up study for subjects with transfusion-dependent β-thalassemia (TDT) who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored clinical studies.
The study aims to assess the long-term safety and efficacy of the drug product in TDT subjects.
Transduction in the phase 3 studies used a refined manufacturing process compared to the phase 1/2 studies. LTF-303 assessments include Hb, peripheral blood vector copy number (PB VCN), assessment of…
Author notes - American Society of Hematology
After 2 y of follow-up, pts could enroll in a long-term follow-up study ( LTF-303 [NCT02633943 ]) for up to an additional 13 y. We report results from LTF - 303 in pts with…
Abstract - American Society of Hematology
This research study is evaluating the long-term effects of an investigational gene transfer approach to treat beta thalassemia major, also referred to as transfusion-dependent β–thalassemia, and severe sickle cell disease…
LTF-303 - Health Research Authority