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Gene therapy shows promise for rare blindness disease

NCT ID NCT04794101

Disease control Sponsor: Janssen Research & Development, LLC 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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Aug 28, 2026 · Updated 3 times

Summary

This study is a follow-up to a Phase 3 trial testing a gene therapy called AAV5-hRKp.RPGR for people with X-linked retinitis pigmentosa, a genetic eye disease that causes vision loss. The treatment aims to slow or stop vision decline by delivering a working copy of the RPGR gene to the retina. Researchers are monitoring 97 participants for side effects and changes in eyesight over time.

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 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

97 people

The number who actually took part.

Started

Dec 2020

Expected to finish

Dec 2029

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

3 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: * Male or female, 3 years of age or older, has XLRP confirmed by a retinal specialist and has a predicted disease-causing sequence variant in RPGR confirmed by an accredited laboratory. Exclusion Criteria: * None

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Conditions

The condition(s) this trial relates to.

retinitis pigmentosa retinitis pigmentosa 3

As listed by the trial registrant

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

X-linked retinitis pigmentosa

Contacts and locations

Locations

  • Azienda Ospedaliera Univ.- Università Degli studi della Campania - Luigi Vanvitelli

    Naples, 80131, Italy

  • Azienda Ospedaliero Universitaria Careggi

    Florence, 50134, Italy

  • Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts

    Paris, 75012, France

  • Childrens Hospital Los Angeles

    Los Angeles, California, 90027, United States

  • Duke Eye Center

    Durham, North Carolina, 27705, United States

  • Emory University

    Atlanta, Georgia, 30322, United States

  • Gartnavel General Hospital

    Glasgow, G12 0YN, United Kingdom

  • Ghent University Hospital

    Ghent, 9000, Belgium

  • Hadassah Medical Center

    Jerusalem, 91120, Israel

  • Hosp Univ Fund Jimenez Diaz

    Madrid, 28040, Spain

  • Hospital For Sick Children

    Toronto, Ontario, M5G 1X8, Canada

  • IRCCS Fondazione G.B. Bietti per lo Studio e la Ricerca in Oftalmologia ONLUS

    Rome, 00184, Italy

  • Massachusetts Eye and Ear Infirmary

    Boston, Massachusetts, 02114, United States

  • Moorfields Eye Hospital

    London, EC1V 2PD, United Kingdom

  • NHS Lothian

    Edinburgh, EH3 9HA, United Kingdom

  • Ospedale San Paolo

    Milan, 20142, Italy

  • Radboudumc

    Nijmegen, 6525EX, Netherlands

  • Retina Consultants of Houston

    Bellaire, Texas, 77401, United States

  • Rigshospitalet Glostrup

    Glostrup Municipality, 2600, Denmark

  • Shiley Eye Institute Jacobs Retina Center

    La Jolla, California, 92093 0946, United States

  • St James University Hospital

    Leeds, LS9 7TF, United Kingdom

  • Stanford Health Care

    Palo Alto, California, 94303, United States

  • Univ of Michigan Medical Center

    Ann Arbor, Michigan, 48105, United States

  • Universite de Lausanne, Hopital ophtalmique Jules-Gonin

    Lausanne, 1004, Switzerland

  • University Hospital Basel, Eye Clinic/Institute of Molecular and Clinical

    Basel, 4031, Switzerland

  • University of Pittsburgh Medical Center (UPMC)

    Pittsburgh, Pennsylvania, 15213, United States

  • VUMC Amsterdam

    Amsterdam, 1105AZ, Netherlands

  • VitreoRetinal Associates, PA

    Gainesville, Florida, 32607, United States

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