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)
Latest Information Update: 30 Jan 2024
At a glance
- Drugs Betibeglogene-autotemcel (Primary)
- Indications Beta-thalassaemia
- Focus Adverse reactions; Therapeutic Use
- Acronyms NorthStar
- Sponsors bluebird bio
- 12 Dec 2023 Results of an analysis reporting iron management outcomes in patients who completed either a phase 1/2 (NCT01745120, NCT02151526) or phase 3 beti-cel parent studies (NCT02906202 and NCT03207009) and subsequently enrolled in the long-term follow-up study (NCT02633943) up to 9 years of follow up presented at the 65th American Society of Hematology Annual Meeting and Exposition
- 12 Dec 2023 Results of an analysis reporting report efficacy, safety, and quality of life (QOL) data from following clinical studies: NCT01745120, NCT02151526, NCT02906202, NCT03207009 and NCT02633943 presented at the 65th American Society of Hematology Annual Meeting and Exposition
- 10 Dec 2023 According to bluebird bio media release, abstract containing data from this study presented at the 65th American Society of Hematology (ASH) Annual Meeting & Exposition