Can a single gene injection preserve sight in inherited blindness?

NCT ID NCT07807111

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Sep 08, 2026 · Last updated Sep 09, 2026 · Updated 1 time

Summary

This trial tests a gene therapy called SPVN20, given as a single injection into the eye, for people with advanced rod-cone dystrophy, a genetic condition that causes progressive vision loss. The study includes adults with a confirmed genetic diagnosis and some remaining cone cells in the central retina. Researchers will evaluate the safety of the injection and measure changes in visual acuity and light sensitivity over six months to see if the therapy can help preserve or improve vision.

What this could mean

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

Active substance
SPVN20 gene therapy
What this could lead to
If SPVN20 works, it could slow or stop vision loss in people with advanced rod-cone dystrophy, potentially preserving sight and delaying blindness.
What could go wrong
This is an early-stage trial with a small number of participants, so the therapy may not be effective or safe in the long term. Gene therapy carries risks like inflammation or immune reactions.

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 27 people

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

Started

Oct 2025

Expected to finish

Mar 2032

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

18 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: 1. Age ≥ 18 years old at enrollment. 2. Participant with a genetically confirmed clinical diagnosis of advanced RCD in both eyes due to non-syndromic RCD. 3. Participants enrolled in the Dose-Escalation cohorts must have a BCVA meeting the study eye criteria. 4. Documented preservation of foveal cone cell bodies in the study eye (as shown on SD-OCT imaging). 5. Participant with a history of formed vision. 6. Participant willing and able to provide informed consent. Exclusion Criteria: 1. Participant participating in another clinical trial and receiving an investigational medicinal product (IMP) within either 5 half-lives of that IMP, or 90 days prior to the injection of SPVN20. 2. Participant with cortical visual impairment. 3. Participant with systemic disease or other pathology not related to their diagnosis of RCD, and whose symptoms or associated treatments may affect vision. 4. Participant with known allergies to corticosteroids, or who will be unable to tolerate the corticosteroid regimen. 5. Participant who has received immunosuppressive therapies or any other therapy known to impact the immune system during the last month prior to SPVN20 administration. 6. Active ocular inflammation or recurrent history of idiopathic or autoimmune-associated uveitis. 7. Participant known to be allergic to any of the delivery vehicle constituents or to any other drugs planned to be used during the clinical study. 8. Active alcohol or substance abuse. 9. Participant positive for human immunodeficiency virus (HIV) or any other systemic immunocompromising disease. 10. Participant with active Hepatitis B or Hepatitis C. 11. Female participant currently pregnant or breastfeeding or intending to become pregnant. 12. Participant with clinically active ocular infection of herpetic diseases. 13. Participant with active coronavirus disease (COVID-19) infection. 14. Participant who received any vaccination/immunization within 28 days prior to screening and/or during screening. 15. Participant who previously received any gene therapy product, stem cell therapy, cell-based therapy for ocular or non-ocular disease. 16. Participant with a retinal implant at enrollment. 17. Participant with pre-existing eye conditions that would interfere with the interpretation of study endpoints, or increase the risk of surgical complications. 18. Participant with significant media opacity impacting evaluation of the retina or vitreous. 19. Participant who had intraocular surgery within 90 days of study treatment administration. 20. Participant with history of corticosteroid-induced raised IOP of \>25 mmHg following corticosteroid exposure, despite topical IOP-lowering pharmacologic therapy. 21. Participant with history of unresolved rhegmatogenous retinal detachment in the fovea. 22. Participant with history of unresolved ocular hypotony. 23. Participant unwilling or unable (based on the Investigator's judgment) to comply with the study protocol. 24. Participant with any condition that would not allow them to complete follow-up examinations during the study and, in the opinion of the Investigator, would make them unsuitable for the study. 25. Participant currently taking restricted classes of medications listed in protocol 26. Participant with ongoing cardiac or neurological disorders not managed by a restricted medication can be excluded based on the Investigator's judgement. 27. Women of childbearing potential (WOCBP) and men and/or their partner(s) of childbearing potential who do not agree to use a highly effective contraceptive method.

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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 study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    3 sites in 3 countries. The list below names each one and where it is.

  3. 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 ↗

  4. 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

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • CHNO XV-XX Paris - CIC 1423

    RECRUITING

    Paris, 75012, France

  • Ghent University Hospital

    RECRUITING

    Ghent, Belgium

  • Royal Victoria Eye And Ear Hospital

    RECRUITING

    Dublin, Ireland

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