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Need-to-Know Information about Clinical Trials

clinical trial patientWith about 15 clinical trials underway for inherited retinal diseases, and several more poised to begin in a few years, patients are eager to sign up for access to potential vision-saving therapies.

However, a clinical trial isn’t a promised land. It’s an experiment with risks, and a person’s motivation for participating in the study should be to help advance the research. Though investigators take extensive precautions to protect the patients, they continue to learn about a therapy’s safety and effect on vision and the body, especially during early stages of the study.

Also, participants often make a big commitment to visiting research centers, perhaps several times a year, for many hours of grueling tests. Sometimes, they travel long distances to get to the center. Some drop out of studies, because the commitment is too much. Unfortunately, losing patients can jeopardize the study and the future of the treatment.

It’s also important to understand that, in some trials, participants might not actually be treated. In masked, randomized studies, some will get a placebo, and won’t know whether they received the placebo or the therapy until the study concludes. Those who get the placebo are still helping to advance the trial. Hopefully, the emerging therapy performs better than the placebo.

Phase I
A participant’s experience also depends on what phase of the study he or she has enrolled in. For example, in a Phase I study, investigators are focused on safety, ensuring that the potential treatment does no harm. In some cases, they may recruit healthy volunteers to evaluate the systemic effects of a treatment.

For a therapy that’s applied directly to the eye — for example, a gene or stem-cell therapy — they may enroll patients with very little remaining vision; so there’s little vision to lose if something goes wrong. Phase I studies are usually of short duration, from a few months to a year, and involve about a dozen patients.

Investigators may also evaluate increasing doses of a treatment in different patient groups, often referred to as dose escalation, to identify the maximum tolerable level.

Phase II
In a Phase II study for an inherited retinal disease, researchers are still monitoring safety, but also begin evaluating efficacy. Is the treatment saving or restoring vision? Depending on the therapy and the prevalence of the diseases included in the trial, the number of participants could range from two dozen to a few dozen. A Phase II is likely to last from two to four years.

Phase III
A Phase III study is the final, or “pivotal,” stage before seeking marketing approval from the U.S. Food and Drug Administration or the European Medicines Agency. In this phase, investigators are looking for strong evidence of efficacy. The therapy may be studied at multiple clinical sites to determine if investigators can achieve the same results independently.

For a blockbuster drug, for example, a new therapy for high blood pressure, thousands of patients may be enrolled in a Phase III. But in the world of rare, inherited retinal conditions, researchers may only be able to enroll a few dozen participants. A Phase III usually lasts from two to four years.

Combined Phases
For many emerging therapies for rare conditions, clinical phases are combined. For example, Phase I and II can be combined as a Phase I/II, or Phase II and III as a Phase II/III. This is done to save time and money and accommodate the limited number of participants available for the clinical trial.

For instance, the gene therapy clinical trial for Leber congenital amaurosis (RPE65 mutations) at the Children’s Hospital of Philadelphia was organized in two phases: a 12-patient Phase I/II and a 24-patient Phase III. The University of Iowa was added as a site for the Phase III. This study began in late 2007 and is scheduled to conclude later this year.

Qualifying for a Study
Finally, it’s important to understand that clinical trials have several inclusion and exclusion criteria for participants. One’s age, genetic profile, level of vision and medical history are all factors in determining eligibility for a study.

Help Drive the Research
I hope my cautionary comments do not dissuade anyone from considering a clinical trial. We need participants, because without them we would have no trials or treatments. But at the same time, it’s important for anyone considering participating to have realistic expectations.

To learn more about clinical trials underway for inherited retinal diseases, visit www.clinicaltrials.gov.


69 Responses to 'Need-to-Know Information about Clinical Trials'

  1. Stephen meyn says:

    I’ve got rp
    I’m 39
    Male
    I have patches of tunnel vision
    Hard to see good at night.
    My centre vision is better then normal
    I’ll be interested in looking into a trial

    • Mubashar says:

      Hi! Its great news that your centre vision is better than normal is really great. I am also one of that and i have lot of problems throughout vision .. If you will get anything good for it please let me inform email
      mubashar_pk2@hotmail.com

  2. tracy says:

    My 2 sons have retinitis pigmentosa there 15 and 8

    • EyeOnTheCure says:

      You may want to try to identify the disease gene/mutation for your sons. A molecular diagnosis is the most accurate diagnosis possible. With the genetic information you can check the medical databases to see what, if any research is being done. You also may be able to 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/genetic_testing_booklet_201311rev.pdf

      Whether or not you identify your son’s disease gene, you should consider enrolling them 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/

      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.

  3. Russ Myers says:

    I have rp am 51 and interested in trial

    • EyeOnTheCure says:

      There are three inherited forms of RP: recessive, dominant and X-linked. If you are not sure which type you have, you should ask your ophthalmologist. For information on inheritance types, please see the following web link to download a PDF document on inheritance:
      http://www.blindness.org/sites/default/files/inheritance_of_retinal_degeneration_-_july_2012.compressed.pdf

      You may also want to try to identify the disease gene/mutation. A molecular diagnosis is the most accurate diagnosis possible. With the genetic information you can check the medical databases to see what, if any research is being done. You also may be able to 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/genetic_testing_booklet_201311rev.pdf

      Whether you identify your disease gene or not, you should consider participating 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/

      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.

  4. Valerie Sandler says:

    I am one of the rare females in the world who, through lyonization, is affected severely by choroideremia and would like to know if there are any studies being done that include the female. I have been studied by the Berman Gund Labs at MEE in Boston since I was 28 and diagnosed at the age of 14. There have been 9 members of my family who have gone blind from choroideremia, including my Father. I am 70 years old, have lost complete sight in my left eye and much of my right although I still retain a few “windows” for reference purposes. I currently also have psudoexfoliation, severe dry eyes that have led to Fooks dystrophy and will undergo cataract surgery in my right eye on Tuesday April 21. I live in the Charlotte area of North Carolina and would travel to participate in clinical trials. Hope to hear from someone. Valerie Sandler

  5. Margaret says:

    My whole family; father, siblings, cousins & now nieces & nephews have RP.

    • EyeOnTheCure says:

      There are three inherited types of RP: recessive, dominant and X-linked. If you are not sure which type you have, you should ask your ophthalmologist. For information on inheritance types, please see the following web link to download a PDF document on inheritance:
      http://www.blindness.org/sites/default/files/inheritance_of_retinal_degeneration_-_july_2012.compressed.pdf

      You may also want to try to identify the disease gene/mutation. A molecular diagnosis is the most accurate diagnosis possible. With the genetic information you can check the medical databases to see what, if any research is being done. You also may be able to 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/genetic_testing_booklet_201311rev.pdf

      Whether you identify your disease gene or not, you should consider participating 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/

      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.

      I hope you find this information helpful. Please let me know if there is anything else I can help you with.

  6. mateen says:

    I am intrested in clinical trial. I have stargardts.
    I am from india

  7. Deborah vestal says:

    I am blind in my right eye as a result of glocoma from a cornea transplant I have a long story of multiple eye problems and surgeries. I would like to receive emails about your progress.

  8. Shaukataytrr Ali says:

    I am 35 year old live in NYC me and my sibling have RP I want to participate clinical trial pls let me know how can I participate

    • EyeOnTheCure says:

      You should know that there are three major types of RP: recessive, dominant and X-linked. If you are not sure which type you have, you should ask your ophthalmologist. For information on inheritance types, please see the following web link to download a PDF document on inheritance:
      http://www.blindness.org/sites/default/files/inheritance_of_retinal_degeneration_-_july_2012.compressed.pdf
      You may also want to try to identify the disease gene/mutation. A molecular diagnosis is the most accurate diagnosis possible. With the genetic information you can check the medical databases to see what, if any research is being done. You also may be able to 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/genetic_testing_booklet_201311rev.pdf
      Whether you identify your disease gene or not, you should consider participating 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/
      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.
      I hope you find this information helpful. Please let me know if there is anything else I can help you with.
      Thank you for your support!

  9. Rudolf Kambinda says:

    I’m 24 years and i have been living with RP and i would like to take pat in the clinical trials. I’m in Namibia, Windhoek.

    • EyeOnTheCure says:

      In order to participate in any of the ongoing clinical trials for RP, you will need to be able to travel back and forth to the trial site. Most trials, although they are free, do not provide financial assistance for travel. You may also want to try to identify the disease gene/mutation. A molecular diagnosis is the most accurate diagnosis possible. With the genetic information you can check the medical databases to see what, if any research is being done. You also may be able to 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/genetic_testing_booklet_201311rev.pdf

      Whether you identify your disease gene or not, you should consider participating 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/

      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.

  10. jeff cohen says:

    63 y.o. who had two retinsl detachments, cataract surgery and a cornea transplantin May 2014 leaves me with limited vision in right eye. Can see light from dark and motion from short distances only

  11. chris schiavone says:

    please help my brother. he’s 29 and suffers from rp. I pray every day for a cure.

    • EyeOnTheCure says:

      You should know that there are three inherited forms of RP: recessive, dominant and X-linked. If you are not sure which type your brother has, you should ask your ophthalmologist. For information on inheritance types, please see the following web link to download a PDF document on inheritance:
      http://www.blindness.org/sites/default/files/inheritance_of_retinal_degeneration_-_july_2012.compressed.pdf
      Your brother may also want to try to identify his disease gene/mutation. A molecular diagnosis is the most accurate diagnosis possible. With the genetic information he can check the medical databases to see what, if any research is being done. He also may be able to 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/genetic_testing_booklet_201311rev.pdf
      Whether your brother identifies his disease gene or not, he should consider participating 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/

      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.

  12. Matt Miller says:

    This was very informative and helpful. I am particularly in agreement with the statement that “a person’s motivation for participating in the study should be to help advance the research.” There are ways in which everyone can help to advance medical and clinical research and I applaud all that donate their time towards doing so. It is interesting to see the rise in ophthalmic trials and treatments. It is a very interesting field worth exploring, and I hope to see more and more corrective vision treatments in the near future.

  13. Christopher DeLeo says:

    Im 17 and I have RP and I will do what ever it takes to not lose anymore vision. Im from Chicago btw.

    • EyeOnTheCure says:

      You should know that there are three different inheritance forms of RP: recessive, dominant and X-linked. If you are not sure which type you have, you should ask your ophthalmologist. For information on inheritance types, please see the following web link to download a PDF document on inheritance:
      http://www.blindness.org/sites/default/files/inheritance_of_retinal_degeneration_-_july_2012.compressed.pdf
      You may also want to try to identify the disease gene/mutation. A molecular diagnosis is the most accurate diagnosis possible. With the genetic information you can check the medical databases to see what, if any research is being done. You also may be able to 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/genetic_testing_booklet_201311rev.pdf
      Whether you identify your disease gene or not, you should consider participating 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/
      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.

  14. Michelle cincotti says:

    My son has RP caused by a metabolic disease (LCHAD), having hard time reading and diffficulty seeing at night. Interested in clinical trials. Preferably close to Boston,Ma area.

  15. Stacey says:

    I’m 42 and was diagnosed “clinically” with SD a couple years ago but have gotten two negative genetic tests back now showing no mutations. I would love to be a candidate for any trials as my vision is fading fast. I’m currently a patient at the Kellogg Eye Institute.

  16. Max H. says:

    My wife was diagnosed with Cone-Rod Dystrophy by
    Dr. Fishman about 7-8 years ago. We are certainly
    interested in trials.

  17. Lee Simms says:

    Would like to take part in the trials. I’m in Farmington, New Mexico. Farmington is in southwest USA, you don’t need a Visa to get there.

  18. ujjwal says:

    I am interested in trials i have rp i am 30 years old and i live in India

  19. Tanya says:

    I’m 45, diagnosed with cone dystrophy. My son, 14 years old, diagnosed with Stargardt. Looking for any opportunity to get into clinical trial. We live in New York.

  20. ahmad says:

    I have RP I am 45 years old I am welling to be in any stem cell trials

  21. Dan says:

    I have RP, but is not clear as to which form, I am willing to participate along with my Brother who also, has the condition. I am from England

  22. Philippe Dhossou says:

    I’m 49 year old and leave in Aldan in Pennsylvania USA.
    I have RP still having some central Vision, but have serious problem of light sensitivity.
    I’m strongly interested by any clinical trial for RP (Gene therapy or Stem Cell therapy)
    Please let me know as soon as you get an opportunity.

    Thank you

    • EyeOnTheCure says:

      If you have not done so already, you may want to try to identify your disease gene/mutation. A molecular diagnosis is the most accurate diagnosis possible. With the genetic information you can check the medical databases to see what, if any research is being done. You also may be able to 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/genetic_testing_booklet_201311rev.pdf
      Whether you identify your disease gene or not, you should consider participating 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/
      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.

  23. Mateen says:

    Hi i am 25 yrs old and i have RP
    I am from india i intrested in trial

  24. David miller says:

    I am 68 years old and have unilateral rp which started in 1985.I would love to help in a clinical trial.

    • EyeOnTheCure says:

      You should 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. You should also consider participating in FFB’s “My Retina Tracker”, a free registry that can help him 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/

  25. ELIZABETH NELSON says:

    I have Glaucoma and macular degeneration caused by extreme nearsightedness I am losing vision in my right eye which sees poorly to begin with and also very dry eyes in my good eye. I am looking for
    information about any stem cell trials . Is there anything I can do to save my vision. I also have
    monovision and now I am seeing double in some
    circumstances.

    • EyeOnTheCure says:

      You should 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.

  26. Steve Geruso says:

    Have CSR in right eye and now have either CSR or MD in left eye. Tries “Avastin” and “Eylea” no luck. Definitely interested in clinical trial. Sight in left eye is starting to diminish….

    • EyeOnTheCure says:

      You should 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.

  27. mahtab says:

    hi
    i have rp.
    i am 27.
    i have tunnel vision.
    my peripheral vision has been destroyed.
    my central vision is good 20/20.
    my tunnel vision is so constricted.
    Looking for any opportunity to get into clinical trial.
    i live in anaheim, CA.
    thank you

    • EyeOnTheCure says:

      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 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

      Whether the disease gene is identified or not, one should still 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/

      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 provides information on what the study is about, the requirements for participating and contact information.

  28. Rick McClellan says:

    I was born with Stargartz and am 61 years old. I found out through the Lions Club that there is a new procedure that may help me regain some vision. I live in Concord,NC and am interested in participating in research on Stargartz.

  29. Raymond Juliano says:

    I sustained a CRVO of my left eye in the summer of 2008. This led to ischemic glaucoma and a complete shut down of my left eye leaving me permanently blind in that eye. My retinal doctor told me that nothing short of a retinal stem cell implant would restore the vision in my left eye. I was wondering what the status of the research and/or clinical trials on CRVO is at this time? Thank you.

  30. I am 49 tomorrow and experienced BRAO on March 23, 2015. I am scheduled for a stem cell procedure on October 6 in Florida. I am very interested in talking to someone who has gone through this. I am in Indiana

  31. Alex Sindalovsky says:

    Hey, My GF have RP – if to be more specific – she have Usher Syndrome – so it affect the sight & the hearing together :(
    We are Looking for any opportunity to get into clinical trial.
    we are from Israel, not in the US – but with a lot of hope to get into one of those trials.

    • EyeOnTheCure says:

      Usher syndrome is the most common condition that affects both hearing and vision. There are three general types of Usher syndrome. People with Usher syndrome type 1 (USH1) are born completely deaf and experience problems with balance. The first signs of RP — night blindness and loss of peripheral vision — usually appear in early adolescence. In Usher syndrome type 2 (USH2), newborns have moderate to severe hearing impairment. Symptoms of RP typically start shortly after adolescence. Visual problems progress less rapidly than in Usher type 1 and hearing loss usually remains stable. A rarer third type of Usher syndrome (USH3) was documented in 1995. Children with USH3 are usually born with good or only mild impairment of hearing. Their hearing and vision loss is progressive, starting around puberty. Balance may be affected. For more detailed information on Usher syndrome, please see the following web link: http://www.blindness.org/eye-conditions/usher-syndrome

      The Foundation Fighting Blindness is currently supporting a gene therapy trial for Usher type 1B. For more information on this clinical trial, please see the following link: http://www.blindness.org/foundation-news/usher-syndrome-gene-therapy-study-begins-oregon

      Presently, there are currently no clinical trials for Usher type 2 or 3. However, FFB is funding a study at the University of Oklahoma to develop a non-viral gene therapy approach for treating Usher type 2A. For more information, please see the following link: http://www.blindness.org/blog/index.php/a-bigger-boat-for-nanoparicles/

      To participate in any gene therapy trial, you must first positively identify the gene mutation. For more information on genetic testing, please see the following link: http://www.blindness.org/sites/default/files/pages/pdfs/Genetic-Testing-Booklet-V5.2-20151023.pdf

      You may also want to consider enrolling in “My Retina Tracker”, a free registry that can help people find out about clinical trials that are recruiting. For more information on “My Retina Tracker” please see the following web link: https://www.myretinatracker.org/

      Additional clinical trials for Usher syndrome can be found on the website Clinical Trials.gov U.S. citizenship is not required to participate in most trials but you may have to pay for your own transportation and housing. See the following link for more information: https://clinicaltrials.gov/ct2/results?term=usher+syndrome&Search=Search

  32. Sheila Murphy says:

    I am 45, went in to get contacts and was told I have Starguards. I see a lot of lights in my vision. I’m very light sensitive and have semi poor vision in one eye, a little worse in the other. I’m very interested in a clinical trial. Thanks.

    • EyeOnTheCure says:

      Dear Sheila, You will be pleased to know that the Foundation Fighting Blindness is partnering with Sanofi Pharmaceuticals on a gene therapy clinical trial for Stargardt disease. For more information on this trial, see the following link:
      http://clinicaltrials.gov/ct2/show/NCT01367444?term=stargardt&recr=Open&no_unk=Y&rank=2 However, before you can apply to participate, you must obtain a molecular (genetic) diagnosis. 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 in “My Retina Tracker”, a free registry that helps link people with retinal disease to appropriate clinical trials that are recruiting. For more information on “My Retina Tracker” please see the following web link: https://www.myretinatracker.org/

      Of general interest, there is a “Stargardt – Macular Degeneration” Facebook page where people can communicate with other people affected by Stargardt disease. Here is the link: https://www.facebook.com/groups/Stargardts/

      Finally, below is a list of pharmaceutical companies that are developing therapies for Stargardt Disease:

      Acucela,( http://www.acucela.com/) is a Seattle-based biotechnology company that is developing several drugs for retinal diseases such as AMD, dry eye, diabetic retinopathy, retinopathy of prematurity and Stargardt disease. Acucela’s visual cycle modulators (VCM) reduce the activity of the rod visual system — in essence, “slowing it down” and reducing the metabolic load on the retina. Reducing the speed of the visual cycle has been shown to protect the retina from light damage and reduce the accumulation of retinal-related toxic by-products, including A2E, which is implicated in both Stargardt disease and dry AMD. The Company’s lead investigational compound (Emixustat™) is currently in Phase 3 trials for dry AMD. Once approved by the FDA, Emixustat could be prescribed for Stargardt disease.

      Ocata Therapeutics (https://www.ocata.com/), a Santa Monica-based biotechnology company, has developed an RPE cell line that is derived from embryonic stem cells (ESC). Studies have shown that the subretinal transplantation of ESC-RPE cells in a rat RP model resulted in 100% visual function rescue. Functional rescue was also achieved in the Stargardt mouse model with near-normal functional measurements recorded at more than 70 days. The RPE cell transplantation studies are now in Phase 2 human clinicals. Here is the link to the Clinical Trials.Gov recruitment web page: http://www.clinicaltrials.gov/ct2/show/NCT01469832?term=advanced+cell+technology&rank=2
      *Note: It is not known how long the transplanted RPE cells will last in a human patient with Stargardt disease. Unless gene or pharmaceutical-based augmentation treatment is coupled to the RPE transplant, toxic A2E will continue to be produced and eventually kill the RPE cells.

      Alkeus, (http://alkeus.com/) Alkeus has developed a form of vitamin A that upon light interaction, does not form toxic vitamin A metabolites and A2E. Alkeus’ lead compound, ALK-001, is an oral compound with a well-understood mechanism of action. ALK-001 was specifically designed to treat Stargardt disease by preventing the formation of these toxic vitamin A dimers in the eye. Alkeus is currently recruiting patients for a Phase 2 human clinical trial. Here is the link to the clinical trials.gov recruitment page: https://www.clinicaltrials.gov/ct2/show/NCT02402660
      Vision Medicine (Previously Visum) The Foundation Fighting Blindness is partnering with Vision Medicine to develop a small molecule therapy Stargardt disease. Vision Medicine’s novel approach proposes to develop drugs that will temporarily control levels of A2E in the eye and preserve the natural vision cycle, leading to a therapeutic treatment. Vision Medicine has discovered a unique chemical approach to sequester rather than eliminate A2E. Through this process, 25 diverse FDA approved drugs demonstrating both mechanistic and in vivo efficacy have been identified. Vision Medicine has identified a lead compound, VM 200, which is an enantiomer of an FDA approved drug that demonstrates complete retinal protection in preclinical studies. Vision Medicine plans to conduct Phase I and Phase II clinical trials in the near future. To read more about the partnership between Vision Medicine and FFB, see the following web link:
      http://www.blindness.org/foundation-news/foundation-fighting-blindness-partners-vision-medicines-develop-stargardt-disease

      I hope you find this information helpful. Please feel free to contact me if you have any other questions or concerns.Thank you for your support that is enabling the development of new treatments for degenerative retinal disease.

  33. ANIL says:

    I AM EFFECTED FROM EALES VASCULITE RETINAL DISEASE IN AGE 17 YR MALE INDIA AND 4 TIME SURGERY DONE IN LEFT TILL DATE AND VISION NOW ALMOST NIL IN LEFT EYE AND VISION IN RIGHT EYE 6/6 HOWEVER 5-10 TIME 360 DEGREE LASER ALREADY DONE IN RIGHT EYE PLEASE EXPLAIN HOW STEM CELL THERAPY WILL HELP IN MY POSITION AND STEM CELL THERAPY TREATMENT STARTED OR NOT IN INDIA AND THIS TREATMENT TO WHOM BENEFICIAL AND IN WHAT AGE AND ON WHICH STAGE AND IN WHICH RETINAL DISEASE? PLS GUIDE ME AND HELP ME GOD MAY BLESS YOU FOR THIS SERVICE TO THE WHOLE WORLD RELATING TO FIGHT AGAINST BLINDNESS THANKS VERY MUCH.

  34. Taylor Hosea says:

    I am a 26 year old Caucasian female who was diagnosed last month with PIC and CNV in my right eye. I have received one avastin injection but sight is diminishing rapidly. This has been devastating to me and my family and would love to be a part of a clinical trial research.

  35. Stephen Kempster says:

    I am a 61 year old male and suffer from Sorsbys Fundus
    Dystrophy. It is a genetic disorder and I would love to participate in any relevant clinical trials. I am based in the UK.

  36. Dylan says:

    My dad has rp and has always said he would love to sometime be able to drive again and be able to see the beautiful sights and his vision is just getting worse I would love to he able to help get him able to see again he is 42

  37. alex says:

    I’m 25 yrs of age ive got a scar on near my macular where I cant see me central vision in the middle where theres a blur.. I can see most left an right vision. if its suitable I’m up for a trial.. if you could please get back to me it would much apeeriated. I live in Australia and I dnt mind to go anywhere that I can get in a trial or treated so my eye doesn’t go any worse.

  38. frank covich says:

    my son skylar was tested by carver labs, he has lebers amarousis. we do not know the genetic marker yet.

    skyar is now 29 married and a professor soon to be PHD,

    I continue to look for anyone who has had some successes in treating lebers, thank you frank covich 2016 june.

  39. Purvi Turakhia says:

    We have a patient with LCA(Mumbai, India) wanting get enrolled for trial of gene therapy. can someone help with how to go about it?

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