Gene therapy injection aims to halt blinding eye disease

NCT ID NCT07705568

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jul 15, 2026 · Last updated Jul 17, 2026 · Updated 2 times

Summary

This trial tests a gene therapy called PST-611 in people aged 65 and older with geographic atrophy, an advanced form of age-related macular degeneration that causes blind spots. PST-611 is a DNA plasmid injected into the eye muscle that instructs cells to produce human transferrin, a protein that may protect the retina. Participants receive one injection every 20 weeks for three doses and are followed for a year to check safety and any changes in vision or retinal structure.

What this could mean

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

Active substance
a gene therapy called PST-611, a DNA plasmid that instructs cells to produce human transferrin, injected into the eye muscle
What this could lead to
If successful, PST-611 could slow or stop the progression of geographic atrophy, a leading cause of blindness in older adults, for which there are limited treatments.
What could go wrong
This is a small, early-phase trial focused on safety, so it is not designed to prove effectiveness. The treatment may not slow vision loss and could cause side effects like eye inflammation or increased pressure.

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 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 24 people

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

Expected to start

Sep 2026

An estimate. Start dates often move.

Expected to finish

Jun 2028

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

65 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: * Subjects must give written informed consent, be able to make the required trial visits and follow instructions. * Female and male subjects must be 65 years of age or older. * In the study eye (SE): cRORA must be present on OCT and attributed to AMD, as evaluated by the Investigator. * Documented recent history (i.e., over 3 to 12 months prior to first PST-611 administration) of GA lesion area progression, with a yearly growth projection of ≥ 1.6 mm2, in the SE * GA lesion area prior to the first PST-611 administration must be between 1.25 mm2 and 16 mm2, as assessed by OCT, in SE. * BCVA must be \< or = 75 ETDRS letters (Snellen \< or = 20/32) in the SE. * Fixation, either central or eccentric, of both eyes, must be compatible with the performance of the ocular imaging and functional assessments included in the trial, as evaluated by the Investigator. * If both eyes are eligible, the SE will be selected by the Investigator based on the totality of the clinical evaluation. Exclusion Criteria: * Both eyes (OU): any active intraocular or periocular infection or inflammation (eg, infectious blepharitis, infectious conjunctivitis, keratitis, scleritis, endophthalmitis), or history of intraocular or periocular infection or inflammation in the 12 weeks (84 days) prior to Dose 1. * SE: any intraocular surgery (including cataract surgery) or intravitreal (IVT) or periocular corticosteroid injection in the 12 weeks (84 days) prior to Dose 1. * SE: the documented need for more than 2 anti-VEGF IVT treatments per year as well as any anti-VEGF IVT treatment within 3 months prior to Dose 1, and lesion must be clinically inactive. * SE: media opacity that interferes with fundus imaging or is likely to require surgery during the trial period. * SE: subject with history of glaucoma filtering surgery (e.g., trabeculectomy or aqueous shunt implant) or who underwent eye surgery in the 12 weeks (84 days) prior to Dose 1. * SE: subject who has uncontrolled intraocular pressure of ≥ 25 mmHg in the SE at the Screening and Trial Baseline Visits. * SE: subject with intraocular hypotension (\<6 mmHg) in the SE that in the opinion of the Investigator would interfere with the PST-611 administrations or the evaluation of its safety or efficacy. * SE: subject with history of scleritis, scleral thinning, cicatrizing conjunctival diseases, severe ocular allergies, severe ocular surface disease, ocular scarring or intraocular hardware (e.g., retained implant device) that could interfere with the PST-611 administrations or the evaluation of its safety. * SE: any other concurrent ocular surface or intra-ocular condition, including retinal disease other than AMD, which, in the opinion of the Investigator, may pose a safety risk for the PST-611 administrations or interfere with the evaluation of its safety. * Subject has any condition, which in the opinion of the Investigator, could compromise the subject's safety or adherence to the trial protocol or follow-up. * Known/suspected hypersensitivity to any standard of care topical or local analgesics/anesthetics or other standard of care treatments used in the preparation of PST-611 administration procedure. * Treatment with investigational medicinal products in the 12 weeks (84 days) prior to Dose 1. * Subject previously exposed to any gene therapy product other than the non-viral gene therapy PST-611.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

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

How to take part

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

  1. The places running it

    3 sites. 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

  • CHU de Grenoble-Hôpital Michallon

    Grenoble, France, 38043, France

  • Hôpital Cochin

    Paris, France, 75014, France

  • Hôpital Lariboisière

    Paris, France, 75010, France

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