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

April 23, 2024 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

Active, not recruiting

Conditions

Intervention / Treatment

Detailed Description

Deferred Treatment

Study Type

Interventional

Enrollment (Actual)

97

Phase

  • 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 Locations

  • Belgium
      • Gent, Belgium, 9000
        • UZ Gent
  • Canada
    • Ontario
      • Toronto, Ontario, Canada, M5G 1X8
        • Hospital For Sick Children
  • Denmark
      • Glostrup, Denmark, 2600
        • Rigshospitalet Glostrup
  • France
      • Paris, France, 75012
        • Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts
  • Israel
      • Jerusalem, Israel, 91120
        • Hadassah Medical Center
  • Italy
      • Firenze, Italy, 50134
        • Azienda Ospedaliero Universitaria Careggi
      • Milano, 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

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
Intervention / Treatment
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

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

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

Janssen Research & Development, LLC

Collaborators

Janssen Research & Development, LLC

Investigators

  • 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)

December 4, 2020

Primary Completion (Estimated)

September 20, 2024

Study Completion (Estimated)

September 20, 2024

Study Registration Dates

First Submitted

November 5, 2020

First Submitted That Met QC Criteria

December 15, 2020

First Posted (Actual)

December 17, 2020

Study Record Updates

Last Update Posted (Actual)

April 24, 2024

Last Update Submitted That Met QC Criteria

April 23, 2024

Last Verified

April 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID 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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