Clinical Programme
We started our clinical programme in July 2006
Single Dose Trial (Pilot Study)
Multi Dose Clinical Trial
Our ultimate aim is to conduct a
clinical trial over a long enough time period that we could look for and
hope to see clinical benefit. The exact design of this study is not
certain yet, and will be influenced by the findings in our single dose
pilot study. However, we are planning to include over 100 patients from
the Run-in study, who will be asked to proceed based on the results of
their measurements. Unlike the pilot study, this will be a blinded,
placebo-controlled study, meaning that each participant will have a
50:50 chance of receiving gene therapy or ‘dummy’ treatment, with
neither patients nor investigators knowing which.
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