Please login to post a response.

• XLRS Human Study
• Posted: 2009-10-28 15:29:31 By Sarah E
• The Applied Genetic Technologies Corporation(AGTC) website has a news release regarding FFB-funded clinical trial that is planned for retinoschisis gene therapy:
  http://www.agtc.com/news/news_agtc_063009_collaborations.htm
  
  I emailed AGTC to ask them questions about when the study is scheduled, what ages would be included, finding participants, etc. They sent me back this information:
  
  (9/30/09)
  (BEGINNING of email response)
  1.The clinical trial will not begin until at least 3 years from now. We still need to do a variety of testing in animals (part of which is funded by the FFB), scale up the production process so we can make enough for a clinical trial, and complete other activities required by the FDA before we can begin a clinical trial.
  
  2. We haven’t decide for sure what the ages of study participants will be. In general, we begin studies in individuals >18 years of age and then later, after we have shown that the treatment is safe in older individuals, study the treatment in younger individuals.
  
  3. We will identify possible participants by a number of methods – from the practices of the doctors who will be investigators in the clinical trial, by announcements on the FFB website and other websites, and by sending letters to patients on a list maintained by the FFB.
  
  4. XLRS will have to be genetically verified to participate in the clinical trial. However, most people with XLRS have a typical clinical presentation, and those with a clinical diagnosis who are interested in being considered for the clinical trial can be genetically tested through one of the clinical trial sites.
  
  5. Participants will have to travel to one of the clinical trial sites to participate in the study – but we have not yet identified where those sites will be. The sites that were listed in the news release are the sites where the testing in animals that is funded by the FFB will be done.
  
  Thank you for your interest in our program. We are enthusiastic about this project. We recognize that everything takes longer than we would hope, but we need to make sure that we do everything correctly so that people who enroll in the trial have the best chance for a successful outcome.
  
  (END of email response)
  
  It is exciting to see that human studies are in the works, though it is hard to be patient and wait at least three years. I hope that the amazing success from the recent LCA studies will speed things up.
  
  Thanks,
  Sarah E.
  

• WOW!
• Posted: 2009-11-24 11:15:29 By Waleska S
• Sarah.
  
  This is great. I will continue to pray for the study to happen. We will wait patiently for more good news.
  
  Thak you
  Johanna