ICTU is involved in two trials evaluating gene therapy medicinal products:
- SERCA-LVAD trial: A randomised, placebo-controlled trial of AAV1/SERCA2A in patients with advanced heart failure and a left ventricular assist device (LVAD) implanted. The trial will take place in 2 UK sites and plans to recruit 24 patients
- Repeated application of gene therapy in Cystic Fibrosis patients: A randomised, placebo-controlled trial to assess whether repeated doses of gene therapy administered to the lungs of CF patients can lead to clinical improvement. The trial recruited 130 patients from two UK sites.