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Gene therapy offers hope for rare blindness

NCT ID NCT04671433

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
Running, but no longer taking on new participants.
Completed This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 27, 2026

Summary

This Phase 3 trial tests a gene therapy called botaretigene sparoparvovec for X-linked retinitis pigmentosa, a genetic condition that causes progressive vision loss. The study involves 105 participants who receive the therapy via injection under the retina. The main goal is to see if it improves their ability to navigate a maze after one year.

What this could mean

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

Active substance
botaretigene sparoparvovec (AAV5-RPGR gene therapy)
What this could lead to
If successful, this gene therapy could slow or partially reverse vision loss in people with X-linked retinitis pigmentosa, potentially preserving independence.
What could go wrong
This is an early-stage Phase 3 trial with only 105 participants. The treatment involves eye surgery and may not work for everyone. Long-term safety and effectiveness are still unknown.

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

105 people

The number who actually took part.

Started

Dec 2020

Finished

Sep 2024

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: * Has had ocular surgery within 3 months prior to screening or is anticipated to require ocular surgery within 6 months after the study intervention administration * Any investigational ocular treatment or any other ocular treatment that could confound the interpretation of the efficacy results or affect participant compliance with the visit schedule * Has undergone prior retinal surgery involving the macula, macular laser photocoagulation, external-beam radiation therapy, transpupillary thermotherapy, glaucoma filtration surgery or corneal surgery

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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, 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

  • 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

    Roma, 00198, Italy

  • Massachusetts General Hospital - Center for Celiac Research and Treatment

    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

  • UZ Gent

    Ghent, 9000, Belgium

  • 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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