Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
- Conditions
- X-Linked Retinitis Pigmentosa
- Registration Number
- NCT04794101
- Lead Sponsor
- Janssen Research & Development, LLC
- Brief Summary
A clinical trial of AAV5-hRKp.RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 97
- 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.
- None
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Primary Outcome Measures
Name Time Method Number of participants with Ocular and Non-ocular Adverse Events of AAV5-hRKp.RPGR in Participants with RPGR-XLRP 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.
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 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
Number of Participants With Abnormalities in Laboratory Assessments Day 1 - Month 60 Number of participants with abnormalities in laboratory assessments will be assessed.
- Secondary Outcome Measures
Name Time Method Change From Baseline in Mean Retinal Sensitivity Within the Central 10 Degrees Excluding Scotoma (MRS10) in Static Perimetry 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.
Change in Retinal Function as Assessed by Pointwise Response in the Central 30-degree Visual Field From Baseline to Month 60 Pointwise response in the central 30-degree visual field will be assessed.
Change in Retinal Function as Assessed by Pointwise Response in Full Visual Field From Baseline to Month 60 Pointwise response in full visual field will be assessed.
Change in Retinal Function as Assessed by Pointwise Response in Worse-seeing Eye in Full Visual Field From Baseline to Month 60 Pointwise response in worse-seeing eye in full visual field will be assessed.
Change From Baseline in Best Corrected Visual Acuity (BCVA) by ETDRS Chart Letter Score in Monocular Assessment From Baseline - Month 60 Change from baseline in BCVA by ETDRS chart letter score in monocular assessment will be assessed.
Change From Baseline in Mean Retinal Sensitivity of Worse-seeing Eye Within the Central 10 Degrees Excluding Scotoma (MRS10) in Static Perimetry 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.
Change From Baseline in Mean Retinal Sensitivity Within the Full Visual Field Excluding Scotoma (MRS90) in Static Perimetry 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.
Vision-guided Mobility Assessment (VMA) Response in the "Worse-seeing Eye" as Assessed by VMA From Baseline to Month 60 Vision-guided mobility assessment response in the "worse-seeing eye" as assessed by VMA will be assessed.
Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ) Extreme Lighting Domain Score From Baseline to Month 60 Change from baseline in the mLLQ extreme lighting domain score will be assessed.
Change in Retinal Function as Assessed by Pointwise Response in Worse-seeing Eye in the Central 30-degree Visual Field From Baseline to Month 60 Pointwise response in worse-seeing eye in the central 30-degree visual field will be assessed.
Change From Baseline in Low Luminance Visual Acuity by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score in Monocular Assessment From Baseline- Month 60 Change from baseline in low luminance visual acuity by ETDRS chart letter score in monocular assessment will be assessed.
Change From Baseline in Low Luminance Visual Acuity by ETDRS Chart Letter Score in Worse-seeing Eye 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.
Trial Locations
- Locations (28)
Universite de Lausanne, Hopital ophtalmique Jules-Gonin
🇨🇭Lausanne, Switzerland
NHS Lothian
🇬🇧Edinburgh, United Kingdom
Shiley Eye Institute Jacobs Retina Center
🇺🇸La Jolla, California, United States
Emory University
🇺🇸Atlanta, Georgia, United States
Retina Consultants of Houston
🇺🇸Bellaire, Texas, United States
Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts
🇫🇷Paris, France
Hospital For Sick Children
🇨🇦Toronto, Ontario, Canada
Ospedale San Paolo
🇮🇹Milano, Italy
Azienda Ospedaliera Univ.- Università Degli studi della Campania - Luigi Vanvitelli
🇮🇹Napoli, Italy
IRCCS Fondazione G.B. Bietti per lo Studio e la Ricerca in Oftalmologia ONLUS
🇮🇹Rome, Italy
Radboudumc
🇳🇱Nijmegen, Netherlands
VUMC Amsterdam
🇳🇱Amsterdam, Netherlands
Hosp Univ Fund Jimenez Diaz
🇪🇸Madrid, Spain
St James University Hospital
🇬🇧Leeds, United Kingdom
Moorfields Eye Hospital
🇬🇧London, United Kingdom
Childrens Hospital Los Angeles
🇺🇸Los Angeles, California, United States
Stanford Health Care
🇺🇸Palo Alto, California, United States
Rigshospitalet Glostrup
🇩🇰Glostrup, Denmark
Hadassah Medical Center
🇮🇱Jerusalem, Israel
Azienda Ospedaliero Universitaria Careggi
🇮🇹Firenze, Italy
Gartnavel General Hospital
🇬🇧Glasgow, United Kingdom
Ghent University Hospital
🇧🇪Gent, Belgium
University Hospital Basel, Eye Clinic/Institute of Molecular and Clinical
🇨🇭Basel, Switzerland
VitreoRetinal Associates, PA
🇺🇸Gainesville, Florida, United States
Massachusetts Eye and Ear Infirmary
🇺🇸Boston, Massachusetts, United States
Univ of Michigan Medical Center
🇺🇸Ann Arbor, Michigan, United States
Duke Eye Center
🇺🇸Durham, North Carolina, United States
University of Pittsburgh Medical Center (UPMC)
🇺🇸Pittsburgh, Pennsylvania, United States