Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

March 12, 2026 updated by: Janssen Research & Development, LLC

Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

A clinical trial of AAV5-RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

Deferred Treatment

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
105

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Belgium
      • Ghent, Belgium, 9000
        • UZ Gent
  • Canada
    • Ontario
      • Toronto, Ontario, Canada, M5G 1X8
        • Hospital for Sick Children
  • Denmark
      • Glostrup Municipality, Denmark, 2600
        • Rigshospitalet Glostrup
  • France
      • Paris, France, 75012
        • Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts
  • Israel
      • Jerusalem, Israel, 91120
        • Hadassah Medical Center
  • Italy
      • Florence, Italy, 50134
        • Azienda Ospedaliero Universitaria Careggi
      • Milan, Italy, 20142
        • Ospedale San Paolo
      • Napoli, Italy, 80131
        • Azienda Ospedaliera Univ.- Università Degli studi della Campania - Luigi Vanvitelli
      • Roma, Italy, 00198
        • IRCCS Fondazione G.B. Bietti per lo Studio e la Ricerca in Oftalmologia ONLUS
  • Netherlands
      • Amsterdam, Netherlands, 1105AZ
        • VUMC Amsterdam
      • Nijmegen, Netherlands, 6525EX
        • Radboudumc
  • Spain
      • Madrid, Spain, 28040
        • Hosp Univ Fund Jimenez Diaz
  • Switzerland
      • Basel, Switzerland, 4031
        • University Hospital Basel, Eye Clinic/Institute of Molecular and Clinical
      • Lausanne, Switzerland, 1004
        • Universite de Lausanne, Hopital ophtalmique Jules-Gonin
  • United Kingdom
      • Edinburgh, United Kingdom, EH3 9HA
        • NHS Lothian
      • Glasgow, United Kingdom, G12 0YN
        • Gartnavel General Hospital
      • Leeds, United Kingdom, LS9 7TF
        • St James University Hospital
      • London, United Kingdom, EC1V 2PD
        • Moorfields Eye Hospital
  • United States
    • California
      • La Jolla, California, United States, 92093 0946
        • Shiley Eye Institute Jacobs Retina Center
      • Los Angeles, California, United States, 90027
        • Childrens Hospital
      • Palo Alto, California, United States, 94303
        • Stanford Health Care
    • Florida
      • Gainesville, Florida, United States, 32607
        • VitreoRetinal Associates, PA
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Emory University
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Massachusetts General Hospital - Center for Celiac Research and Treatment
    • Michigan
      • Ann Arbor, Michigan, United States, 48105
        • Univ of Michigan Medical Center
    • North Carolina
      • Durham, North Carolina, United States, 27705
        • Duke Eye Center
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15213
        • University of Pittsburgh Medical Center (UPMC)
    • Texas
      • Bellaire, Texas, United States, 77401
        • Retina Consultants of Houston

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

3 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Single

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Other: Deferred Treatment
Biological: Genetic: AAV5-hRKp.RPGR
Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group
Other Names:
  • botaretigene sparoparvovec
Biological: Genetic: AAV5-hRKp.RPGR
No intervention - deferred treatment group (Bilateral, sub-retinal administration of AAV5-hRKp.RPGR to be administered in the follow-up study)
Experimental: Experimental - Immediate Treatment
Intermediate dose.
Biological: Genetic: AAV5-hRKp.RPGR
Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group
Other Names:
  • botaretigene sparoparvovec
Biological: Genetic: AAV5-hRKp.RPGR
No intervention - deferred treatment group (Bilateral, sub-retinal administration of AAV5-hRKp.RPGR to be administered in the follow-up study)
Experimental: Experimental Immediate Treatment
Low dose.
Biological: Genetic: AAV5-hRKp.RPGR
Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group
Other Names:
  • botaretigene sparoparvovec
Biological: Genetic: AAV5-hRKp.RPGR
No intervention - deferred treatment group (Bilateral, sub-retinal administration of AAV5-hRKp.RPGR to be administered in the follow-up study)

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Change From Baseline to Week 52 in Vision-guided Mobility Assessment (VMA) as Measured by the Ability of the Participant to Navigate Through a VMA Maze
Time Frame: From Baseline to 52 Weeks
Change from baseline to Week 52 in VMA as measured by the ability of the participant to navigate through a VMA maze.
From Baseline to 52 Weeks

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Change From Baseline in Mean Retinal Sensitivity Within the Central 10 Degrees Excluding Scotoma (Mean Retinal Sensitivity Within the Central 10 Degree Excluding Scotoma in Static Perimetry [MRS10]) in Static Perimetry at Week 52
Time Frame: From Baseline to Week 52
Change from baseline in mean retinal sensitivity within the central 10 degrees excluding scotoma (MRS10) in static perimetry at Week 52 will be assessed.
From Baseline to Week 52
Change From Baseline in Mean Retinal Sensitivity of Worse-seeing Eye Within the Central 10 Degrees Excluding Scotoma in Static Perimetry (MRS10) at Week 52
Time Frame: From Baseline to Week 52
Change from baseline in mean retinal sensitivity of worse-seeing eye within the central 10 degrees excluding scotoma in static perimetry (MRS10) at Week 52 will be assessed.
From Baseline to Week 52
Change in Retinal Function as Assessed by Pointwise Response in Full Visual Field at Week 52
Time Frame: From Baseline to Week 52
Pointwise response in full visual field at Week 52 will be assessed.
From Baseline to Week 52
Change in Retinal Function as Assessed by Pointwise Response in Worse-seeing Eye in Full Visual Field at Week 52
Time Frame: From Baseline to Week 52
Pointwise response in worse-seeing eye in full visual field at Week 52 will be assessed.
From Baseline to Week 52
Change in Retinal Function as Assessed by Pointwise Response in the Central 30 Degrees Visual Field at Week 52
Time Frame: From Baseline to Week 52
Pointwise response in the central 30 degrees visual field at Week 52 will be assessed.
From Baseline to Week 52
Change in Retinal Function as Assessed by Pointwise Response in Worse-seeing Eye in the Central 30 Degrees Visual Field at Week 52
Time Frame: From Baseline to Week 52
Pointwise response in worse-seeing eye in the central 30 degrees visual field at Week 52 will be assessed.
From Baseline to Week 52
Change From Baseline in Retinal Function as Assessed by Mean Retinal Sensitivity Within the Full Visual Field (MRS90) in Static Perimetry at Week 52
Time Frame: From Baseline to Week 52
Change from baseline in retinal function as assessed by mean retinal sensitivity within the full visual field (MRS90) in static perimetry at Week 52 will be assessed.
From Baseline to Week 52
Change in Functional Vision by Using Vision-guided Mobility Assessment (VMA) Response in the "Worse-seeing Eye" at Week 52
Time Frame: From Baseline to Week 52
Change in functional vision by using VMA assessment in the "Worse-seeing Eye" at Week 52.
From Baseline to Week 52
Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ) Extreme Lighting Domain score at Week 52
Time Frame: From Baseline to Week 52
Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ) Extreme Lighting Domain score at Week 52.
From Baseline to Week 52
Change From Baseline in Visual Function as Assessed by Monocular Low Luminance Visual Acuity Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter score at Week 52
Time Frame: From Baseline to Week 52
Change from baseline in visual function as assessed by monocular low luminance visual acuity using the ETDRS chart letter score at Week 52.
From Baseline to Week 52
Change From Baseline in Visual Function as Assessed by monocular Best Corrected Visual Acuity (BCVA) Using the ETDRS Chart Letter Score at Week 52
Time Frame: From Baseline to Week 52
Change From Baseline in visual function as assessed by monocular BCVA using the ETDRS chart letter score at Week 52.
From Baseline to Week 52
Change From Baseline in Visual Function as Assessed by Low Luminance Visual Acuity Using the ETDRS Chart Letter Score in Worse-seeing Eye at Week 52
Time Frame: From Baseline to Week 52
Change from baseline in visual function as assessed by low luminance visual acuity using the ETDRS chart letter score in worse-seeing eye at Week 52.
From Baseline to Week 52
Number of Participants with Ocular and Non-ocular Adverse Events
Time Frame: Day 1 - Week 52
Number of participants with ocular and non-ocular adverse events will be assessed.
Day 1 - Week 52
Number of Participants With Abnormalities in Laboratory Assessments
Time Frame: Day 1 - 52 Weeks
Number of participants with abnormalities in laboratory assessments will be assessed.
Day 1 - 52 Weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Janssen Research & Development, LLC

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Janssen Research & Development, LLC

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Janssen Research & Development, LLC Clinical Trial, Janssen Research & Development, LLC

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
December 4, 2020

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
September 30, 2024

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
September 30, 2024

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
November 5, 2020

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 15, 2020

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
December 17, 2020

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 16, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 12, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
March 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • CR109258
  • MGT-RPGR-021 (Other Identifier: Janssen Research & Development, LLC)
  • 2020-002873-88 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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