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Experimental gene therapy aims to halt vision loss in rare blindness

NCT ID NCT03328130

Disease control Sponsor: eyeDNA Therapeutics Source: ClinicalTrials.gov ↗

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
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 Results may not be published yet.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 This study
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This status has not been confirmed recently, so it may be out of date.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 Jun 27, 2026

Summary

This study tested a gene therapy for retinitis pigmentosa, an inherited eye disease that causes gradual vision loss. The treatment involved injecting a corrected gene under the retina in one eye. The trial included 19 people aged 13 and older with a specific genetic defect. The main goal was to check safety and see if vision improved.

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

19 people

The number who actually took part.

Started

Nov 2017

Finished

Jun 2025

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

13 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.

Key Inclusion Criteria: * Clinical and molecular diagnosis of retinitis pigmentosa caused by defect in PDE6B gene without other syndromic manifestations * Aged above 13 years * Ability to give informed consent Key Exclusion Criteria: * Previous ocular surgery or thermal laser within 6 months before the surgery * Lens opacities or obscured ocular media upon recruitment such reliable evaluation or grading of the posterior segment cannot be performed * Known serious allergies to the fluorescein dye used in angiography, to the mydriatic, steroidal and non-steroidal eye drops * Participation in another clinical trial with an investigational agent * Enrolled or being enrolled in another gene therapy clinical trial * Active, extraocular infection requiring the prolonged or chronic use of antimicrobial agents * Chronic medical conditions, cancer * Abnormal laboratory values * On immunosuppressive therapy

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Conditions

The condition(s) this trial relates to.

Eye Diseases, Hereditary eye disorder inherited retinal dystrophy retinal degeneration retinal disorder retinitis pigmentosa retinitis pigmentosa 40 vision disorder

As listed by the trial registrant

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

Retinitis pigmentosa

Contacts and locations

Locations

  • Clinique Ophtalmologique, CHU de Nantes

    Nantes, 44093, France

More trials for these conditions

Other studies related to the condition(s) this trial covers.