TEASE – Stargardt Clinical Studies by Alkeus Pharmaceuticals2020-02-22T14:17:00+00:00

Introducing the TEASE Studies

Stargardt disease, also known as “Stargardt macular degeneration”, is an inherited eye condition that leads to loss of central vision during childhood or early adulthood. The majority of people with Stargardt experience their first symptoms before their late teens, although some people may experience symptoms much earlier, and others much later (“late-onset Stargardt”). Although Stargardt is rare, affecting approximately 1 in every 8,000 to 10,000 people, it is the most common form of inherited macular degeneration and the 3rd most common recessive monogenic disease. There is currently no treatment for Stargardt disease.

“TEASE” is a group of clinical studies performed to evaluate the safety and effects of ALK-001, an investigational oral drug, in its ability to slow or prevent vision loss in patients with Stargardt disease.

Clinical studies, also called clinical trials, are conducted to determine if investigational treatments are safe, effective and medically helpful to patients, before these treatments can become approved for use in the general population. Clinical studies are organized around a team of healthcare professionals and scientists, including physicians, research coordinators, pharmaceutical companies, etc.

About ALK-001

ALK-001 is a modified vitamin A that prevents the formation of toxic vitamin A clumps in the eye. ALK-001 is the experimental drug being tested in the TEASE study.

Vitamin A is a natural molecule found in food, and is essential to enable vision. However, during its processing by the eye, vitamin A can form toxic clumps called “vitamin A dimers.” Scientists believe that these toxic clumps build up more quickly in people with Stargardt disease, which contributes to their vision loss. This is because Stargardt disease is caused by mutations on a gene called ABCA4, which is responsible for controlling the transport of vitamin A in the eye. When ABCA4 is defective, transport of vitamin A is impaired, accelerating the formation of vitamin A dimers.

ALK-001 acts as a vitamin A replacement and functions just like vitamin A, except for the fact that it does not readily forms clumps. In mice, replacing the vitamin A diet with ALK-001 prevents the ocular degeneration of mice with ABCA4 gene defects. In humans, when taken once a day as a pill, ALK-001 rapidly replaces vitamin A in the body, thereby slowing the formation of vitamin A clumps in the eye. Researchers hope that by preventing the formation of vitamin A clumps, one could slow, stop or prevent vision loss in Stargardt disease.

Frequently Asked Questions for People Interested in Participating

  • A total of 12 visits will take place during the clinical study, about every 3 months with more frequent visits earlier on.
  • The study doctor may decide to skip some of the visits.
  • If a participant misses a visit it may be rescheduled to a nearby date within the allowed visit window.
  • Most procedures performed in the study are already done as part of standard eye care.
  • Additional procedures may involve blood draws, visual function tests and filling out questionnaires.

Patients who are at least 8 years old and have been diagnosed with Stargardt disease may be eligible for TEASE. Prospective participants should talk to their doctor and contact the study team to see if they may qualify. Eligibility is ultimately determined by the clinical study team.

The clinical study team will discuss in further detail the possible risks and benefits with prospective participants.


Clinical study participants have access to investigational treatments and their potential benefits before the general public. The study drug may slow or prevent vision loss in Stargardt disease. However, there is no guarantee of any clinical benefit.

Clinical study participants help advance the understanding of diseases and expand treatment options available to other patients, which benefits society as a whole.


Participants’ health will be closely monitored throughout the study. Based on safety data from previous clinical studies, significant side effects are not expected.

Participants in TEASE should:

  • Take the study drug once daily,
  • Attend all scheduled study visits,
  • Inform the clinical study team of any changes in health,
  • Not become pregnant and abide by study contraception requirements, and
  • Not enroll in other clinical trials.

The study drug ALK-001 is designed to stop or slow the progression of Stargardt disease. ALK-001 is not expected to restore lost eyesight. However, it may preserve remaining vision.

For the most part, participants are allowed to continue taking their normal medications. Participants may also continue taking supplements that do not contain vitamin A or beta-carotene, although certain supplements may have to be discontinued. The study doctor and the study team will provide more detail.

There is currently no treatment for Stargardt disease.

  • Stargardt patients should talk to their doctor about genetic testing and should collect a copy of their medical records,
  • Prospective participants should further discuss with their doctor to determine if they may benefit from enrolling in a clinical study.
  • If you have Stargardt, the following tips may be helpful for you:
    • See a low vision rehabilitation specialist who can help you make best use of your residual vision.
    • Use a magnifier and extra lighting for reading, or zoom in on your cell phone and enlarge fonts on a computer
    • Avoid exposure to bright light and wear sunglasses when possible.

Interested in TEASE

The TEASE clinical study is taking place in the United States at multiple clinical study centers. For more information, please contact the sponsor by:

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The TEASE clinical study is taking place in the United States at multiple clinical study centers. For more information, please contact the sponsor by:

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