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Phase 3 Study of Gildeuretinol Acetate (ALK‑001) to Assess Safety and Efficacy in Slowing Progression of Stargardt Disease in Adults

Fast replyInvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

The study focuses on Stargardt Disease, a rare inherited condition that slowly damages the central part of the retina and leads to loss of sharp vision. The investigational medicine being tested is an oral capsule that contains Gildeuretinol Acetate, a new drug intended to protect the retina, and it will be compared with a matching placebo capsule that contains no active ingredient.

The main goal of the trial is to find out whether the medication can slow the increase in the size of the damaged areas of the retina. The study is designed so that participants are randomly assigned to receive either the active drug or the placebo, and neither the participants nor the study staff will know which treatment is given. The trial will last about two years, with regular clinic visits for assessments.

During the study, participants will take one capsule each day and attend scheduled visits where several eye tests are performed. A special camera called FAF will take pictures that highlight unhealthy retinal tissue, while an imaging technique known as OCT will create cross‑sectional views of the eye’s layers. Vision will be measured using a standard eye chart (ETDRS) to determine BCVA, and low‑light reading ability will also be checked. Reading speed will be evaluated with two tests, IReST and MNRead. Sensitivity of the retina will be mapped with microperimetry, and participants will complete questionnaires such as the NEI-VFQ-25 to report how the condition affects daily activities.

The research process

The trial runs in 8 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Randomization and assignment

    After enrollment, you will be randomly assigned to receive either the active drug or a matching placebo. the assignment is kept unknown to you and the study staff to maintain a double‑masked design.

  2. Step 2

    Baseline evaluations

    At the first visit, a series of baseline tests will be performed. these include retinal imaging using fundus autofluorescence (faf), optical coherence tomography (oct), and microperimetry to measure retinal sensitivity.

    Visual acuity will be measured with the etdrs chart, and low‑light visual acuity (llva) will also be assessed. you will complete several questionnaires about vision‑related quality of life, such as the nei‑vfq‑25 and eq‑5d.

    Blood samples will be taken to check general health and laboratory safety parameters.

  3. Step 3

    Start of study medication

    You will begin taking the assigned study medication orally. the active medication is gildeuretinol acetate, a capsule containing 14 mg of the drug. the placebo capsule looks identical but contains no active ingredient.

    The capsule is taken by mouth as directed by the study protocol. the exact schedule (for example, once daily) and the total duration (up to 24 months) will be explained by the study team.

  4. Step 4

    Regular safety visits and medication dispensing

    Throughout the study, you will attend regular clinic visits, typically every month, for safety monitoring. during these visits, vital signs and any adverse effects will be recorded.

    New supplies of the study medication will be provided at each visit, and you will be reminded of the correct way to take the capsule.

  5. Step 5

    Month 6 assessment

    At month 6, a comprehensive evaluation will be repeated. retinal atrophic lesion growth will be measured again with faf, and the change from baseline will be recorded as the primary endpoint.

    Visual function tests, including llva, bcva (best‑corrected visual acuity), and microperimetry, will be performed. questionnaires about vision‑related daily activities will be completed again.

  6. Step 6

    Month 12 assessment

    The same set of imaging and functional tests performed at month 6 will be repeated at month 12 to track any further changes.

    Laboratory safety tests will also be reviewed at this visit.

  7. Step 7

    Month 18 assessment

    A third set of retinal imaging, visual acuity, and microperimetry assessments will be conducted to continue monitoring disease progression.

    Patient‑reported outcome questionnaires will be administered again.

  8. Step 8

    Month 24 final assessment

    At the end of the 24‑month period, a final comprehensive evaluation will be performed, including faf imaging, oct, microperimetry, bcva, llva, and all quality‑of‑life questionnaires.

    The final safety laboratory tests will be taken, and the study medication will be discontinued according to the protocol.

Who can join the trial?

9 criteria

  • Age between 8 and 45 years.
  • Has been diagnosed with Stargardt Disease (STGD), an inherited eye condition, by an eye doctor.
  • Genetic test shows a change in the ABCA4 gene, which is known to cause this condition.
  • Visible damage to the retina (the light‑sensing part at the back of the eye) in at least one eye, seen on standard eye scans (pictures taken with special eye cameras).
  • Eye images show that the condition has gotten worse over time (disease progression).
  • Ability to see large letters on a vision chart during an eye exam (adequate vision level).
  • Willing and able to understand the study, follow instructions, and attend all required visits and tests.
  • Women who can become pregnant and men with partners who can become pregnant must agree to use birth control during the study and for a short time after it ends.
  • Signed the informed consent (or assent with parent/guardian consent) form agreeing to take part in the study.

Who cannot join the trial?

5 criteria

  • If you have taken any Vitamin A supplements, beta‑carotene, liver‑based products, or prescription medicines that belong to the retinoid group (for example, the acne drug isotretinoin, also called Accutane) within the last 30 days, you cannot join the study. Retinoid means a type of medication that works like Vitamin A.
  • You cannot take part if you are currently pregnant, are breastfeeding, plan to become pregnant during the study, or if you are a man who plans to father a child during the study.
  • If you have already participated in another research trial for Stargardt disease (the eye condition being studied), you are excluded unless you only received a placebo (an inactive treatment) and did not have any eye surgery as part of that earlier trial.
  • You are not allowed to join if you have recently been in another clinical study or if you intend to take part in a different study while this one is in progress.
  • If you have any other medical or eye problems that could affect your eyes, interfere with the study procedures, make participation unsafe, or make it difficult to follow the study rules, you cannot participate.
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Investigated drugs

Gildeuretinol acetate is an oral capsule being studied as a new treatment for Stargardt disease, a genetic eye condition that causes loss of vision. In the trial, participants take the capsule to see if the medicine can safely slow down the growth of the damaged areas in the retina. The drug is called an orphan drug, which means it is being developed for a rare disease with few treatment options. Researchers are looking at how well it works compared with a placebo, as well as how well patients tolerate it.

What is already known about the treatment

  • Placebo Capsules

    The capsules are taken by mouth as a regular oral pill, but they contain only inactive ingredients and no drug. They are used in clinical trials to keep the study blind and to compare against the active drug, and they have no therapeutic effect. Because they contain no active substance, they are not a medicine in clinical practice and are only described in trial protocols. Their classification is simply an inert oral capsule used as a control.

  • Gildeuretinol Acetate

    The drug is given as an oral capsule, each containing 14 mg of the active substance, and is taken by swallowing the capsule with water. It is currently being studied in a Phase 3 trial and is classified as an orphan drug for Stargardt disease, a rare inherited eye disorder. The medication works by slowing the visual cycle, which reduces the buildup of harmful vitamin‑A by‑products that damage retinal cells. It belongs to the class of visual‑cycle modulators used to protect the retina.

Investigated diseases

Stargardt disease - Stargardt disease is an inherited eye disorder that affects the retina, the light‑sensing tissue at the back of the eye. It usually begins in childhood or early adulthood and causes a gradual loss of central vision. The disease is characterized by the buildup of yellowish pigment and the formation of atrophic (dead) areas in the macula. Over time, these atrophic lesions enlarge, leading to further reduction in the ability to see fine details and read. Peripheral vision typically remains intact. The condition progresses at different rates in different people.
Trial detailsLast updated 4 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2026-525296-60-00Protocol codeALK001-P1004Estimated enrolment224 patients

sourced from the EU Clinical Trials Register and site verification

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