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Ophthotech Launching Human Study of Emerging Therapy for Stargardt Disease

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Ophthotech, a biopharmaceutical company developing therapies for eye diseases, has enrolled the first patient in its Phase 2b clinical trial of Zimura® for people with Stargardt disease caused by mutations in the gene ABCA4. The 120-participant study will be taking place at more than 30 sites. Data and knowledge gleaned from ProgStar, a natural history study for people with Stargardt disease funded by the Foundation Fighting Blindness, was used in the design of the clinical trial.

Zimura is also in human studies for the treatment of the dry and wet forms of age-related macular degeneration (AMD) and idiopathic polypoidal choroidal vasculopathy (IPCV), an age-related retinal disease.

Zimura is administered by an intravitreal injection — an injection into the soft gel in the middle of the eye. Intravitreal injections are commonly performed in a doctor’s office.

Ophthotech’s emerging therapy is designed to inhibit activity of the complement system. An overactive complement system has been implicated in the development of retinal conditions such as AMD and Stargardt disease. The complement system is a part of the immune system that normally protects the body from harmful, invading bacteria. However, it can also attack healthy cells leading to vision-robbing retinal diseases and other conditions. Zimura blocks a complement protein known as C5. In lab studies, inhibiting C5 prevented retinal degeneration.

“Recent scientific literature emphasizes the role of complement in Stargardt disease, supporting our strategy for the development of Zimura in this devastating orphan retinal condition,” said Kourous A. Rezaei, MD, senior vice president and chief medical officer at Ophthotech, in a press release. “Our work with the highly distinguished organization, Foundation Fighting Blindness, provided us access to publicly available data from ProgStar, the largest natural history study in autosomal recessive Stargardt disease to date, playing an integral role in the design of our clinical trial.”

“Stargardt disease is a devastating orphan retinal disease for which there is currently no treatment,” said Benjamin Yerxa, PhD, FFB’s chief executive officer. “We are excited that Ophthotech has joined the mission to fight against orphan degenerative retinal diseases as both organizations share the common goal of transforming the lives of these patients and their families by developing potential new treatments.”

Stargardt disease is the leading cause of inherited macular degeneration, affecting 30,000 people in the U.S. and tens of thousands more around the world. The condition, usually diagnosed in childhood or adolescence, leads to loss of central vision.

37 Responses to 'Ophthotech Launching Human Study of Emerging Therapy for Stargardt Disease'

  1. Diane Franklin Dunbar says:

    Thank you for your consideration in informing me on what my niece is going through. Through it all she is a trooper full of courage and strength.

  2. Jill says:

    thanks I was diagnosed with Stargardts at age 8. I am now 54. I remain open and hopeful. I would like to see “normally” in my lifetime. While it has been a funfilled journey so far, patience is not my best quality. My faith in a “Stargardts miracle “ holds on.

  3. Amelia Todd says:

    This is exciting news for those of us afflicted with Stargardt’s,. While reading this, 2 questions come to mind: 1) how can one participate in this study? 2) are there research studies testing the affects of nutrition on this disease? With current research showing a link between inflammation and neurological diseases such as Parkinson’s and dementia I am very Interested in finding out if this type of research is being conducted.

    • EyeOnTheCure says:

      Hi Amelia, if you’d like to participate in this study, you can enroll on here:

      So far, we are not involved in any studies related to nutrition and Stargardt disease, as it’s an inherited disease. But an FFB-funded study recently suggests that vitamin A palmitate supplementation may slow the decline of cone function in children with retinitis pigmentosa (RP). Although this is a different disease, this will help open the door for other similar development.

  4. Wanda hedrick says:

    Would love for someone to find a cute for stargodt disease. Have suffered many years with it.

  5. Phyllis Hasty says:

    My 31 year old daughter has Stargardt’s disease. She is experiencing greater central vision loss in these last few months. She has a 4 year old and a 2 year old daughter and a one year old son. It is discouraging. Praying for a cure. How does one sign up for a trial? Thank you!

  6. MARY says:

    My son has been diagnosed with Stargardt’s disease he has lost all center vision praying for cure for all who have been effected with this terrible disease. I am desperately looking for a clinical trail for my son. How do we find one ?

    • EyeOnTheCure says:

      Hi Mary, you can find a trial for your son by visiting and searching for recruiting trials for Stargardt. Each clinical trial listing will provide you with information on what the study is about, the requirements for participating and contact information. Good luck!

  7. Tatiana Tayt-son says:

    My name is Tatiana and I live in Brazil in Rio de Janeiro in Copacabana. My son is 10 years old and has stargardt. I wonder if he can take part in any research. We are confident that we will have healing soon. Amen

  8. Cristina says:

    My son just got diagnosed he is 10 years old and I need more information about this disease, whats the average time for the disease starts to develop? can it happen from one day to another I dont know what to do the doctors provided me very lack information im very stressed and depressed

    • EyeOnTheCure says:

      Hi Cristina, the progression of symptoms in Stargardt disease is variable. Visual acuity (the ability to distinguish details and shape) may decrease slowly at first, accelerate, and then level off. You can find more information about Stargardt disease on our website here: Our website also provides a list of recommended specialist that may be more helpful in your search to learn more. Hope this helps and let us know if you have any other questions.

    • Desiree says:

      I started to lose my vision dramastically around the age 14. Sun rays will make it worsen faster. Be sure to have him wear sunglasses and hats at all times. And have him take lots of vitamin A. Wish I new those when I was younger. GOODLUCK

  9. Petr Johanes says:

    My soon is 14 years old and he has been diagnosed with Satrgardts disease. If possible we would like to participate in the trial, we can fly our son where necessary.. we are ready to do whatever it takes to help our child..

  10. Rolf Spelz says:

    My son Rolf was diagnosed with Stargardt 3disease 30 years ago.
    He is now married with 3 kids, (Rolf, Eleny and Emma)
    We would be grateful giving my son an opportunity and participate in this program
    We all live in Guadalajara Mexico,

  11. santosh says:

    Is there any update on this ?

  12. Tina Feast says:

    Would like more information about this. My son suffers from this disease

  13. Jenny Stetson says:

    I would like as much information as possible. My daughter was diagnosed with Stargardt disease when she was 12. She is now 24 and has 2 kids herself. I know this is an inheritable disease. How does one get tested for this gene abnormality.

  14. Sal says:

    Hello – My sister is diagnosed with Stargardt and she is now 31 years old. I would love to have more information on the clinical trials. Thank you

  15. Wayne says:

    My daughter was diagnosed last year at 10 yrs old. We are adapting with equipment that helps her see, but a cure like this would be nothing short of a miracle. I have been praying for this to happen in her lifetime, preferably while she still is young. How do we get on the clinical trial list when it is released?

  16. Prakash pandey says:

    I was diagnosed at the age 25 n now I’m plus 34.can’t see what on computer screen even from 7 inch off the screen. That’s why no one is giving me job as I can focus on less area at a glance on documents n take mmuch time to read the whole.can’t drive any vehicle as I’m color blind n can’t see traffic lights.I’m also night blind so no late nights alone.diagnosed with BEST disease also.I’m frustrated now n feel like handicap. Pls any cure can end this nightmare.

  17. Ragu says:

    If a child 9yrs has BCVA over 20/200 is excluded from study?

  18. Tabby Tab says:

    My husband was diagnosed 10 years ago we would love to find information about a cure for this

  19. Amy Ooi says:

    Hi, I am suffering from this disease. I have 2 sons. Really hope to have a treatment as soon as possible. Wish to see the world and my families normally. How can I participate in the trial? I am from Malaysia.

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