Trial Profile
Long-term Follow-up of Subjects with Hemoglobinopathies Treated with Ex Vivo Gene Therapy using Autologous Hematopoietic Stem Cells Transduced with a Lentiviral Vector
Status:
Active, no longer recruiting
Phase of Trial:
Phase III
Latest Information Update: 28 Mar 2024
Price :
$35
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At a glance
- Drugs Betibeglogene-autotemcel (Primary) ; Busulfan
- Indications Beta-thalassaemia; Haemoglobinopathies; Sickle cell anaemia
- Focus Adverse reactions; Pharmacodynamics; Registrational; Therapeutic Use
- 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 Results published in the bluebird bio Media Release