LentiGlobin BB305 study in subjects with Sickle Cell Disease

• Research type

  Research Study

• Full title

  A Phase 3 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with the LentiGlobin BB305 Lentiviral Vector in Subjects with Sickle Cell Disease.

• IRAS ID

  266755

• Contact name

  Jesus de la Fuente Pereda

• Contact email

  josu.delafuente@nhs.net

• Sponsor organisation

  bluebird bio, Inc

• Eudract number

  2019-000331-63

• Duration of Study in the UK

  2 years, 7 months, 27 days

• Research summary

  This is a Phase 3 study to evaluate the safety and efficacy of the LentiGlobin BB305 drug product in adult and paediatric subjects (≤2 and ≥50) with Sickle Cell Disease. Participation lasts for around 27 months and is split into 4 stages. Stage 1 - Screening and Eligibility assessment; Stage 2 - Stem cell collection, LentiGlobin BB305 manufacture and preparation for clinical use. During this stage, subjects will undergo a transfusion regimen, for 60 prior to stem cell collection, to reach a target level of Haemoglobin; Stage 3 - Myeloaeblative conditioning and infusion of LentiGlobin BB305. This stage involves administration of Busulfan to reduce the activity of bone marrow cells, before teatment with the LentiGlobin BB305; Stage 4 - 24 month follow up following drug product infusion.

• REC name

  London - West London & GTAC Research Ethics Committee

• REC reference

  20/LO/1093

• Date of REC Opinion

  12 Feb 2021

• REC opinion

  Further Information Favourable Opinion