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The Foundation’s 5 Most Asked Questions About Clinical Trials

Image of BeakerAt the Foundation, we get asked tons of questions – so many, in fact, that, on occasion, I’m going to address the “most asked” for a specific subject. This time around, it’s one that’s on many minds: the clinical trials necessary to obtain FDA approval of a drug or treatment.

How do I enroll in, and then keep apprised of, clinical trials?
To stay abreast of clinical trials, visit this Foundation web page, where you’ll need to be registered, if you aren’t already. But that’s quick and painless, and it gets you on the list to receive informative publications and news alerts on retinal research.

Our site is easy to navigate and up-to-date, providing links to informative articles on the emerging treatments entering human studies. Each listing on the site has a link to www.clinicaltrials.gov, the federal government’s website, where you can navigate for additional information. In addition, on clinicaltrials.gov, you can search on your retinal degeneration and get a list of clinical trials. And a new website, NIH Clinical Research Trials and You, explains clinical trials in detail, offering, among other things, basic information, explanations from researchers and first-hand experiences from trial participants.

I’m on the Foundation’s National Registry, but nobody ever calls me. So how does it work?
Researchers often come to us when they’re looking for clinical trial participants. That’s where the Registry comes in. If the treatment is for Stargardt, for example, we’ll search the Registry and let those patients know about recruitment.  We provide you with the researcher’s contact information, and you reach out. This maintains your confidentiality.

If you’re not listed on the Registry, you can go to this page to access an application and FAQ. With all the new clinical trials coming to fruition, the sooner you get registered, the better.

What are the criteria for clinical trials? How do I know if I can participate?
Criteria for participation in a clinical trial vary from trial to trial. With an early safety study, for example, researchers may only want people with advanced disease, those with less vision to lose, in case the treatment has an adverse effect on vision. In some trials that are further along, and known to be safe, young children are treated because of a disease’s early onset.

If you have complications such as glaucoma or cataracts, diabetes, heart disease or cancer, that can exclude you. The only way to know is to check the trial listing first and then talk with the appropriate person at a clinical trial site. Participation in one clinical trial might exclude you from a future study, but that depends on the actual clinical trial, so it is not a hard-and-fast rule.

What’s expected once you’ve made yourself available for a clinical trial?
While everyone with a retinal degeneration wants a treatment or cure as soon as possible, that isn’t the best reason to go into a clinical trial. Clinical trials are human experiments. The main reason to participate is to help advance the science. Even if a treatment doesn’t make it through the trial (i.e.,  the intervention does not have the expected positive result), researchers learn about the treatment and/or the potential of possible alternatives. And while researchers collaborate with the FDA to ensure safety, there are still risks. You should be aware of this before signing on.

Clinical trials often require several or many visits, sometimes over years, to a research center for follow-up. Some visits may last several hours, as you undergo several tests, some of which can be grueling. This can be especially tough if you don’t live near the center. And investigators want you to finish what you started. It’s critical that you comply with the treatment and schedule until the trial’s done. If people drop out or don’t comply, the trial will likely fail.

What could happen to me during or after a clinical trial?
Before you participate in a clinical trial, you will have to read what has become a long consent form thoroughly and weigh your personal along with scientific considerations. Really, the bottom line is: Are you willing to help the field move forward, not only for yourself but for others while understanding that there could be some risk involved?

Here are a couple other things to consider: If a trial is for a drug, you might be given a placebo instead of the active medication; and even if the treatment requires an implant or surgical procedure, you might receive a sham implant or incision. The ability to determine if a potential treatment has an effect requires this comparison.

 


11 Responses to 'The Foundation’s 5 Most Asked Questions About Clinical Trials'

  1. Edvaldo F de Castro says:

    I hope that Dr. Stephen Rose will comment on the StarGen Clinical Trials. Only he will be able to shed new light on them.

  2. I have retinitis pigmentosa and I would like to enter your list of patient to try the latest trial treatment on my ailment. I am almost losing all my eyesight except for a very narrow tunnel left.
    Please kindly help me.
    I had been to some retina specialist in the Mass eye ear & throat infirmary institute and the specialist just told me at the present time theris no cure. I will be willing to take any experimental trial so please include me in any trial the foundation is involved.
    Thank you very much !!!!
    Respectfully yours;
    Alfonso C. salido, MD.

  3. Maria G. Mendoza says:

    I have retinitis pigmentosa also and I would like to enter your list of participates in your trials. I have been seen by may doctors and all have told me there is no cure for what I have. I am willing to be part of your research and also my family is willing to be of your research projects. I have a appointment to be seen by a Dr. in a clinic in Arizona that is doing experimental stem cell implantation. Please contact me as soon as possible i am very interested in being part of our trails.

    Thank you for your consideration,

    Maria G. Mendoza

    • EyeOnTheCure says:

      Hi Maria, thanks for posting. I will pass along your information to put you on the list. Mitsy, who provides patient resources, will contact you shortly by email. Thanks again

  4. Thanks for the blog. Our staff should find the info really interesting

  5. sanjay sharma says:

    I have a daughter (avani sharma 11yrs) diagonosised with LCA with flat erg .can she be treated?I am from jodhpur rajasthan. india .my cell phone=+919784994400.please help me. Thank you

    • EyeOnTheCure says:

      Dear Sanjay, If you have not done so already, you should try to identify your daughter’s disease gene/mutation. There is currently a clinical trial being conducted for LCA caused by mutations in the RPE-65 gene. If there are clinical trials being conducted on your daughter’s gene, she should have a high likelihood of qualifying. For information on genetic testing, please see the following web link to download a PDF document: http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf
      You should also consider enrolling your daughter in FFB’s “My Retina Tracker”, a free registry that can help you find out about clinical trials that are recruiting for your specific disease. For more information on “My Retina Tracker” please see the following web link: https://www.myretinatracker.org/
      Finally, it may also be helpful to periodically check the website: http://WWW.CLINICALTRIALS.GOV which is maintained by the National Institutes of Health and contains a searchable list of clinical trials for most known diseases. Each clinical trial listing will provide you with information on what the study is about, the requirements for participating and contact information.

  6. Dennis McGowan says:

    I would like to be placed on list for trials, I have RP, as does my mother and my daughter. I would like to help any way I can.

    • Dr. Tim Schoen says:

      Dear Dennis,

      You should consider genetic testing to try and identify the mutant gene responsible for causing the disease. If the gene is identified, medical databases such as PubMed (http://www.ncbi.nlm.nih.gov/pubmed/) can be searched to identify any research that is being conducted. With a molecular diagnosis, one may also qualify for gene therapy trials that are taking place. For information on genetic testing, please see the following web link to download a PDF document:
      http://www.blindness.org/sites/default/files/pages/pdfs/Genetic-Testing-Booklet-V5.2-20151023.pdf
      You should also consider participating in FFB’s “My Retina Tracker”, a free registry that monitors clinical trials that are recruiting for various retinal diseases. For more information on “My Retina Tracker” please see the following web link:
      https://www.myretinatracker.org/
      Finally, you may find it helpful to periodically check the website: http://WWW.CLINICALTRIALS.GOV which is maintained by the National Institutes of Health and contains a searchable list of clinical trials for most known diseases. Each clinical trial listing will provide you with information on what the study is about, the requirements for participating and contact information. Thank you for your support that is helping to accelerate the development of new safe and effective treatments for inherited retinal disease.

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