Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I)

• Research type

  Research Study

• Full title

  Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I): Phase I/II clinical study to evaluate the safety and efficacy of the infusion of autologous hematopoietic stem cells transduced with a lentiviral vector encoding the ITGB2 gene

• IRAS ID

  1006163

• Contact name

  Johnathan Schwartz

• Contact email

  js@rocketpharma.com

• Sponsor organisation

  Rocket Pharmaceuticals Inc.

• Research summary

  This is a long term follow up study (RP-L201-0121-LTFU) for Gene Therapy of Leukocyte adhesion deficiency-I (LAD-I). To take part in this study, patients had to be previously treated with RP-L201 (CD34+ hematopoietic stem cells modified with a lentiviral vector containing the ITGB2 gene, encoding for the human CD18 receptor) in the Phase 1/2 RP-L201-0318 study.
  This long-term follow-up study does not include administration of any treatment and will periodically monitor RP-L201 treatment effects in patients for a total of 15 years after the infusion occured in the RP-L201-0318 parent study. A total of 9 pediatric patients (6 from the US, 2 from the UK and 1 from Spain) will take part in this study.

• REC name

  South Central - Oxford A Research Ethics Committee

• REC reference

  23/SC/0137

• Date of REC Opinion

  31 Aug 2023

• REC opinion

  Further Information Favourable Opinion