Good morning!

All the talk about placebos, appropriate controls and FDA rules, made me look back into my folders about a gene therapy clinical trial I was familiar with. It was gene therapy for haemophilia by providing factor VIII using a gutted-adenoviral vector. It was in 2002 and was one of the first few gene therapy trials with adenoviral vectors. This FDA and RAC approved clinaical trial protocol had no placebo controls to compare with. It was assyaing for factor VIII before and after treatment . It was a Phase 1, and the end point was detectable factor VIII and of course the safety issue that is associated with using adenoviral vector. My involvement in this trial was minimal, and do not have many required details and thats why I hesitated to post it here. But it might be useful info to know that FDA did approve a gene therapy protocol without an appropriate, untreated/vector alone group. It all depended on how the protocol was written and justified to the FDA.
Thanks
Girija