As a recap of the year so far, our team at the University of Alberta has been hard at work preparing for our upcoming choroideremia gene therapy clinical trial. The study details, including the criteria for participation can be found here. We will begin recruiting participants in Fall 2014 when we receive final approval from Health Canada. Until we have that final approval we cannot recruit participants. At present, we have preliminary approval from Health Canada but need to provide some more information on the vector that we will be using in our clinical trial. We expect the information from the company providing the vector shortly.
Since our clinical trial will be a small Phase 1 study, we will only be enrolling 12 participants over the course of a year. We have put in place a formal process for selecting participants. Selection will be made by an external board of Canadian ophthalmologists. We need participants whose clinical symptoms are at the specific stage to give us the best possible results in the clinical trial. We also need to select participants who will stick to the follow-up schedule. The most important parts of the trial are the follow-up visits where we collect the results. Approval for any further, larger clinical trials will be based on the results of this trial.
We need to be prepared so that we can recruit as soon as we receive final Health Canada approval. Therefore, we are looking at anonymous records in the Foundation Fighting Blindness Canada Patient Registry to later identify individuals who might meet our selection criteria and also going over the clinical records of Dr. MacDonald’s patients. As mentioned in the previous blog post, we have also been fine-tuning the methods and equipment we will eventually be using to assess vision in the clinical trial. Vision is the main outcome measure to assess the results of the clinical trial.
We want to make clear that the clinical data we will be using to assess eligibility does not need to come from our centre in Edmonton. We want to encourage interested people with choroideremia to register with the FFB Canada Patient Registry as we have full access to this data. This is also beneficial because other gene therapy research teams may also apply for access to this registry data.