Gene therapy Long Term Follow Up Study

• Research type

  Research Study

• Full title

  Long Term Follow-up for Patients with Inborn Errors of Immunity Treated with Autologous Ex Vivo Gene Modified CD34+ Advanced Therapies at Great Ormond Street Hospital.

• IRAS ID

  1009819

• Contact name

  Hannah Badham

• Contact email

  hannah.badham@gosh.nhs.uk

• Sponsor organisation

  Great Ormond Street Hospital NHS Trust

• ISRCTN Number

  ISRCTN91900773

• Research summary

  GOSH is currently the sponsor of a number of clinical trials investigating the safety and efficacy of novel gene therapies for the treatment of genetic disorders of immunity such as Chronic Granulomatous disease and Severe Combined Immunodeficiency. The investigational treatments for these diseases involve harvesting the patient’s own blood stem cells, genetically modifying them with viral vectors to correct the disorder, and then infusing these corrected stem cells back into the patient to rebuild their immune system.
  
  After receiving a gene therapy on a clinical trial at GOSH, participants are usually followed up for 24-36 months. Following this, participants need to be enrolled onto a long-term follow-up study to meet the legal requirement for gene therapy developers to provide long-term safety and efficacy follow up for 15 years. This long-term follow up is required to enable detection of delayed adverse events such as insertional oncogenesis. This long-term observational safety study will fulfil these obligations and assure the long-term safety of GOSH patients who have received an autologous ex vivo gene modified CD34+ advanced therapeutic investigational medicinal product at GOSH whilst recruited on a GOSH sponsored trial, via compassionate use pathway, or where a commercial long-term follow up study has been discontinued. This study would primarily focus on the detection of potential gene therapy-related adverse events. Patients will continue to be assessed by GOSH clinicians as per standard of care. There will also be the option to collect efficacy data regarding the patient’s gene therapy by retrospective review of patients standard of care medical notes.

• REC name

  London - West London & GTAC Research Ethics Committee

• REC reference

  25/LO/0188

• Date of REC Opinion

  14 May 2025

• REC opinion

  Further Information Favourable Opinion