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Gene therapy injection aims to save sight in rare eye disease

NCT ID NCT07002398

Disease control Sponsor: VeonGen Therapeutics GmbH Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Sep 03, 2026 · Updated 2 times

Summary

This early-phase trial tests a new gene therapy called VG801 for Stargardt disease, a genetic condition that causes vision loss. Fifteen people aged 6 and older will receive a single injection under the retina. The main goal is to check safety, but researchers will also measure any changes in vision.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
VG801 (a gene therapy drug injected under the retina)
What this could lead to
If it works, this could point toward a treatment that slows or stops vision loss in people with Stargardt disease.
What could go wrong
This is a very early, first-in-human trial with only 15 participants. It may not work, and there are risks from the injection and gene therapy itself.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 18 people

The number the study aims to enrol. It can still change while the study runs.

Started

Dec 2024

Expected to finish

Dec 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

6 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: To be eligible for study entry, subjects must satisfy all the following criteria: 1. Written informed consent. 2. Subjects aged ≥ 6 years. 3. Clinical diagnosis of a macular lesion phenotypically consistent with a recessive hereditary macular dystrophy (Stargardt disease). 4. Confirmed molecular diagnosis of ABCA4 mutations (homozygotes or compound heterozygotes). 5. Poor vision in the study eye. Exclusion Criteria: Subjects will be excluded from the study if one or more of the following statements are applicable to either eye: 1. Pre-existing eye conditions such as uveitis, glaucoma, or diabetic retinopathy or implantation of a medical device in the vitreous cavity or subretinal space. 2. Systemic diseases that would preclude the planned surgery or interfere with the interpretation of study results. 3. History of intraocular surgery within the previous 6 months. 4. Previous participation in a gene therapy trial. 5. Participation in a clinical trial (investigational drug or medical device) within the previous 6 months. 6. Any other eye disease that may affect the outcome of the study (e.g., ocular opacities, advanced cataracts, amblyopia, etc.).

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Conditions

The condition(s) this trial relates to.

ABCA4-related retinopathy eye disorder retinal degeneration retinal disorder severe early-childhood-onset retinal dystrophy Stargardt disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Retinal dystrophy due to biallelic ABCA4 mutations Stargardt disease 1

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Shanghai General Hospital

    RECRUITING

    Shanghai, Shanghai Municipality, 200080, China

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