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

March 25, 2024 updated by: Janssen Research & Development, LLC

Phase 3 Follow-up 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-hRKp.RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)

Study Overview

Status

Active, not recruiting

Conditions

Intervention / 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
        • Ghent University Hospital
  • 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
      • Rome, Italy, 00184
        • 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 Los Angeles
      • 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 Eye and Ear Infirmary
    • 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:

  • None

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
Experimental: Deferred Treatment From MGT-RPGR-021 of Intermediate Dose
Deferred treatment
Biological: Genetic: AAV5-hRKp.RPGR Intermediate Dose
Bilateral, subretinal administration of AAV5-hRKp.RPGR - deferred treatment group
Other Names:
  • botaretigene sparoparvovec
Experimental: Already Treated in MGT-RPGR-021
Already treated
Biological: Genetic: AAV5-hRKp.RPGR Intermediate Dose
Bilateral, subretinal administration of AAV5-hRKp.RPGR - deferred treatment group
Other Names:
  • botaretigene sparoparvovec
Biological: Genetic: AAV5-hRKp.RPGR Low Dose
Bilateral, subretinal administration of AAV5-hRKp.RPGR - deferred treatment group
Other Names:
  • botaretigene sparoparvovec
Experimental: Deferred Treatment From MGT-RPGR-021 Low Dose
Deferred treatment
Biological: Genetic: AAV5-hRKp.RPGR Low Dose
Bilateral, subretinal administration of AAV5-hRKp.RPGR - deferred treatment group
Other Names:
  • botaretigene sparoparvovec

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from baseline in vision-guided mobility assessment (VMA), as measured by the ability of the participant to navigate through a VMA maze, after bilateral subretinal delivery of AAV5-hRKp.RPGR
Time Frame: From Baseline to Month 60
Change from baseline in vision-guided mobility assessment (VMA), as measured by the ability of the participant to navigate through a VMA maze, after bilateral subretinal delivery of AAV5-hRKp.RPGR
From Baseline to Month 60
Number of participants with Ocular and Non-ocular Adverse Events of AAV5-hRKp.RPGR in Participants with RPGR-XLRP
Time Frame: Day 1 - Month 60
Number of participants with ocular and non-ocular adverse events of AAV5-hRKp.RPGR in participants with RPGR-XLRP will be assessed.
Day 1 - Month 60
Number of Participants With Abnormalities in Laboratory Assessments
Time Frame: Day 1 - Month 60
Number of participants with abnormalities in laboratory assessments will be assessed.
Day 1 - Month 60

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change From Baseline in Mean Retinal Sensitivity Within the Central 10 Degrees Excluding Scotoma (MRS10) in Static Perimetry
Time Frame: From Baseline to Month 60
Change from baseline in mean retinal sensitivity within the central 10 degrees excluding scotoma (MRS10) in static perimetry will be assessed.
From Baseline to Month 60
Change From Baseline in Mean Retinal Sensitivity of Worse-seeing Eye Within the Central 10 Degrees Excluding Scotoma (MRS10) in Static Perimetry
Time Frame: From Baseline to Month 60
Change from baseline in mean retinal sensitivity of worse-seeing eye within the central 10 degrees excluding scotoma (MRS10) in static perimetry will be assessed.
From Baseline to Month 60
Change in Retinal Function as Assessed by Pointwise Response in Full Visual Field
Time Frame: From Baseline to Month 60
Pointwise response in full visual field will be assessed.
From Baseline to Month 60
Change in Retinal Function as Assessed by Pointwise Response in Worse-seeing Eye in Full Visual Field
Time Frame: From Baseline to Month 60
Pointwise response in worse-seeing eye in full visual field will be assessed.
From Baseline to Month 60
Change in Retinal Function as Assessed by Pointwise Response in the Central 30-degree Visual Field
Time Frame: From Baseline to Month 60
Pointwise response in the central 30-degree visual field will be assessed.
From Baseline to Month 60
Change in Retinal Function as Assessed by Pointwise Response in Worse-seeing Eye in the Central 30-degree Visual Field
Time Frame: From Baseline to Month 60
Pointwise response in worse-seeing eye in the central 30-degree visual field will be assessed.
From Baseline to Month 60
Change From Baseline in Mean Retinal Sensitivity Within the Full Visual Field Excluding Scotoma (MRS90) in Static Perimetry
Time Frame: From Baseline to Month 60
Change from baseline in mean retinal sensitivity within the full visual field excluding scotoma (MRS90) in static perimetry will be assessed.
From Baseline to Month 60
Vision-guided Mobility Assessment (VMA) Response in the "Worse-seeing Eye" as Assessed by VMA
Time Frame: From Baseline to Month 60
Vision-guided mobility assessment response in the "worse-seeing eye" as assessed by VMA will be assessed.
From Baseline to Month 60
Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ) Extreme Lighting Domain Score
Time Frame: From Baseline to Month 60
Change from baseline in the mLLQ extreme lighting domain score will be assessed.
From Baseline to Month 60
Change From Baseline in Low Luminance Visual Acuity by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score in Monocular Assessment
Time Frame: From Baseline- Month 60
Change from baseline in low luminance visual acuity by ETDRS chart letter score in monocular assessment will be assessed.
From Baseline- Month 60
Change From Baseline in Best Corrected Visual Acuity (BCVA) by ETDRS Chart Letter Score in Monocular Assessment
Time Frame: From Baseline - Month 60
Change from baseline in BCVA by ETDRS chart letter score in monocular assessment will be assessed.
From Baseline - Month 60
Change From Baseline in Low Luminance Visual Acuity by ETDRS Chart Letter Score in Worse-seeing Eye
Time Frame: From Baseline to Month 60
Change from baseline in low luminance visual acuity by ETDRS chart letter score in worse-seeing eye will be assessed.
From Baseline to Month 60

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, 2029

Study Completion (Estimated)

December 19, 2029

Study Registration Dates

First Submitted

December 15, 2020

First Submitted That Met QC Criteria

March 8, 2021

First Posted (Actual)

March 11, 2021

Study Record Updates

Last Update Posted (Actual)

March 27, 2024

Last Update Submitted That Met QC Criteria

March 25, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MGT-RPGR-022 (Other Identifier: Janssen Research & Development, LLC)
  • 2020-002255-37 (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

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.

Clinical Trials on X-Linked Retinitis Pigmentosa

Clinical Trials on Genetic: AAV5-hRKp.RPGR Intermediate Dose

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in France

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe