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
- NCT04671433
- Lead Sponsor
- Janssen Research & Development, LLC
- Brief Summary
A clinical trial of AAV5-RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- 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
- 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 & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Primary Outcome Measures
Name Time Method 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 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.
- Secondary Outcome Measures
Name Time Method Change in Retinal Function as Assessed by Pointwise Response in Full Visual Field at Week 52 From Baseline to Week 52 Pointwise response in full visual field at Week 52 will be assessed.
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 the Modified Low Luminance Questionnaire (mLLQ) Extreme Lighting Domain score at 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 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.
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 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.
Change in Retinal Function as Assessed by Pointwise Response in Worse-seeing Eye in the Central 30 Degrees Visual Field at Week 52 From Baseline to Week 52 Pointwise response in worse-seeing eye in the central 30 degrees visual field at Week 52 will be assessed.
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 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.
Change in Retinal Function as Assessed by Pointwise Response in Worse-seeing Eye in Full Visual Field at Week 52 From Baseline to Week 52 Pointwise response in worse-seeing eye in full visual field at Week 52 will be assessed.
Change From Baseline in Retinal Function as Assessed by Mean Retinal Sensitivity Within the Full Visual Field (MRS90) in Static Perimetry at Week 52 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.
Change in Functional Vision by Using Vision-guided Mobility Assessment (VMA) Response in the "Worse-seeing Eye" at Week 52 From Baseline to Week 52 Change in functional vision by using VMA assessment in the "Worse-seeing Eye" at Week 52.
Number of Participants With Abnormalities in Laboratory Assessments Day 1 - 52 Weeks Number of participants with abnormalities in laboratory assessments will be assessed.
Change in Retinal Function as Assessed by Pointwise Response in the Central 30 Degrees Visual Field at Week 52 From Baseline to Week 52 Pointwise response in the central 30 degrees visual field at Week 52 will be assessed.
Change From Baseline in Visual Function as Assessed by monocular Best Corrected Visual Acuity (BCVA) Using the ETDRS Chart Letter Score at Week 52 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.
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 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.
Number of Participants with Ocular and Non-ocular Adverse Events Day 1 - Week 52 Number of participants with ocular and non-ocular adverse events will be assessed.
Trial Locations
- Locations (27)
Emory University
🇺🇸Atlanta, Georgia, United States
University of Pittsburgh Medical Center (UPMC)
🇺🇸Pittsburgh, Pennsylvania, United States
Rigshospitalet Glostrup
🇩🇰Glostrup, Denmark
Shiley Eye Institute Jacobs Retina Center
🇺🇸La Jolla, California, United States
Stanford Health Care
🇺🇸Palo Alto, California, United States
Childrens Hospital
🇺🇸Los Angeles, California, United States
University Hospital Basel, Eye Clinic/Institute of Molecular and Clinical
🇨🇭Basel, Switzerland
Gartnavel General Hospital
🇬🇧Glasgow, United Kingdom
Hadassah Medical Center
🇮🇱Jerusalem, Israel
Azienda Ospedaliera Univ.- Università Degli studi della Campania - Luigi Vanvitelli
🇮🇹Napoli, Italy
Massachusetts General Hospital - Center for Celiac Research and Treatment
🇺🇸Boston, Massachusetts, United States
Azienda Ospedaliero Universitaria Careggi
🇮🇹Firenze, Italy
Hosp Univ Fund Jimenez Diaz
🇪🇸Madrid, Spain
Radboudumc
🇳🇱Nijmegen, Netherlands
UZ Gent
🇧🇪Gent, Belgium
Moorfields Eye Hospital
🇬🇧London, United Kingdom
Ospedale San Paolo
🇮🇹Milano, Italy
NHS Lothian
🇬🇧Edinburgh, United Kingdom
Universite de Lausanne, Hopital ophtalmique Jules-Gonin
🇨🇭Lausanne, Switzerland
Duke Eye Center
🇺🇸Durham, North Carolina, United States
St James University Hospital
🇬🇧Leeds, United Kingdom
Hospital For Sick Children
🇨🇦Toronto, Ontario, Canada
IRCCS Fondazione G.B. Bietti per lo Studio e la Ricerca in Oftalmologia ONLUS
🇮🇹Roma, Italy
VUMC Amsterdam
🇳🇱Amsterdam, Netherlands
Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts
🇫🇷Paris, France
VitreoRetinal Associates, PA
🇺🇸Gainesville, Florida, United States
Univ of Michigan Medical Center
🇺🇸Ann Arbor, Michigan, United States