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A study to evaluate the safety and effectiveness of AAVB-039 in patients with Stargardt disease

Verified siteInvestigationalNo placebo
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What is this trial about?

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

This study focuses on Stargardt Disease, a rare condition that affects the retina, which is the light-sensitive tissue at the back of the eye. This disease often leads to a gradual loss of central vision. The research involves testing a treatment called AAVB-039, which is an experimental solution for injection delivered via subretinal administration, a method where the medicine is placed directly under the retina.

The purpose of the study is to evaluate the safety and effectiveness of AAVB-039. The investigation is divided into two parts. The first part focuses on finding the safest and most appropriate amount of the medicine to use. The second part examines how well the treatment works at that specific dose over time.

Participants in the study will be monitored to observe any changes in their vision and the health of their eyes. This includes checking the ellipsoid zone, which is a specific layer of cells in the retina that is important for clear vision, and the macula, the part of the eye responsible for detailed central sight. The study will track these changes over several years to understand the long-term impact of the treatment.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    <b>subretinal administration</b> of <b>aavb-039</b>

    An injection of aavb-039, which is a solution for injection, is performed under the retina (the light-sensitive layer at the back of the eye).

    This medication contains a combination of two viral vectors designed to deliver genetic material to the eye.

  2. Step 2

    <b>safety and dose assessment</b>

    During the first part of the trial, the focus is on determining the safety and tolerability of the medication, as well as identifying the most appropriate dose level.

    Monitoring includes checking for adverse events (side effects), changes in clinical laboratory parameters (blood or other tests), vital signs, and physical examination findings from the day of the injection until the end of the trial.

  3. Step 3

    <b>efficacy evaluation</b>

    The second part of the trial evaluates how well the medication works at the preferred dose level.

    The main measurement is the rate of change in the ellipsoid zone (ez) loss, which refers to a specific area in the retina, over a period of 12 months.

  4. Step 4

    <b>long-term monitoring</b>

    The effects of the medication are monitored over 12, 24, and 60 months.

    Assessments include measuring the area of atrophy (damaged tissue) using specialized eye imaging, macular sensitivity (how the center of the retina responds to light), best corrected visual acuity (the clearest vision possible with glasses), and low-luminance visual acuity (vision in dim light).

Who can join the trial?

10 criteria

  • You must have two specific changes (called biallelic mutations) in your ABCA4 gene that are known to cause disease, confirmed by a certified medical laboratory.
  • For the first part of the study (Part A), you must be between 18 and 55 years old.
  • For the second part of the study (Part B), you must be between 8 and 55 years old.
  • The clear parts of your eye (ocular media) must be clear enough, and your pupils must be able to open wide enough (pupillary dilation) to allow for high-quality pictures of the macula, which is the central part of the retina responsible for detailed vision.
  • The study doctor must confirm that your eye shows signs of macular atrophy, which is the wasting away or loss of tissue in the center of the retina.
  • In Part A, for the first group of participants (sentinel participants), the area of tissue loss (measured by DAF or macular atrophy) must be between 0.05 and 13.5 square millimeters, and your vision (BCVA, or best-corrected visual acuity) must be between 20/320 and 20/80.
  • In Part A, for all other participants, the area of tissue loss must be between 0.05 and 13.5 square millimeters, and your vision must be between 20/200 and 20/32.
  • All participants in Part A must show a continuous loss of the EZ band (an area in the eye that helps with sharp vision) measuring at least 500 microns in the center of the retina, as seen on an OCT (a specialized light scan of the eye).
  • In Part B, the area of tissue loss must be between 0.05 and 13.5 square millimeters, and your vision must be between 20/200 and 20/32.
  • Participants in Part B must show signs that the disease is progressing through eye scans called OCT (optical coherence tomography) or FAF (fundus autofluorescence, a way to map the health of retinal cells).

Who cannot join the trial?

9 criteria

  • You cannot participate if you have macular atrophy, which is the thinning or loss of tissue in the central part of the eye, caused by something other than Stargardt Disease.
  • You are excluded if you have DAF (a specific type of tissue change) that touches an area of peripapillary atrophy, which is the thinning of tissue around the optic nerve, in your target eye.
  • You cannot join if you have already been confirmed to have genetic mutations (changes in your DNA) in specific genes such as ELOVL4, PROM1, PRPH2, CRX, BEST-1, CDH3, RPE65, PDE6A, PDE6B, RLBP1, RAB28, RDH11, RP25, DRAM-1, or MT-TL1.
  • You are excluded if you have any other eye disease that might interfere with testing how well you see, such as choroidal neovascularization (abnormal blood vessel growth), epiretinal membrane (a thin layer of tissue over the retina), diabetic retinopathy (damage to the retina caused by diabetes), diabetic macular edema (swelling in the center of the retina due to diabetes), uveitis (inflammation inside the eye), or other diseases of the macula (the part of the eye responsible for sharp central vision).
  • You cannot participate if you have moderate or severe glaucomatous optic neuropathy, which is damage to the optic nerve caused by glaucoma, especially if your intraocular pressure (the fluid pressure inside your eye) is not under control using more than two eye drops.
  • You are excluded if you have a history of glaucoma surgery involving filters or valves in the eye.
  • You cannot join if you have had any surgery on the inside of the eye or the surface of the eye within 3 months before the study begins.
  • You are excluded if you have had certain types of vitreoretinal surgery, which are operations performed on the back of the eye, such as a vitrectomy (removing the gel inside the eye) or procedures to fix a fallen lens.
  • You cannot participate if your retinal degeneration (damage to the light-sensitive layer at the back of the eye) is so advanced that doctors cannot accurately measure the EZ, which is a specific, healthy layer of cells in the retina, using a specialized eye scan called an OCT.
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Investigated drugs

AAVB-039 is a gene therapy designed to treat Stargardt disease. It uses a modified, harmless virus to deliver functional pieces of the human ABCA4 gene directly into the area under the retina of the eye. The goal is to help the eye cells work correctly by providing the genetic instructions they are missing.

What is already known about the treatment

AAVB-039 - This experimental gene therapy is administered as a liquid solution through a direct injection into the space under the retina of the eye. It is currently being studied in clinical trials and is not yet available for general medical use. The treatment is designed to address Stargardt disease by using modified, harmless viruses to deliver pieces of a functional human gene into the eye's cells. Once inside, these pieces join together to help the body produce the necessary protein that is missing or broken in patients with this condition.

Investigated diseases

Stargardt disease - This is a genetic eye disorder that affects the central part of the retina. It is caused by mutations in the ABCA4 gene, which prevents the proper processing of vitamin A in the eye. Over time, fatty deposits build up in the retina and damage the light-sensing cells. This process leads to the gradual loss of the area known as the macula. As the disease progresses, the damaged areas expand, causing a reduction in central vision.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase I/IITrial ID2025-522207-15-01Protocol code039-101Estimated enrolment75 patientsSponsorAavantgarde Bio S.r.l.

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