A clinical trial of AAV5-RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Enrollment
105
Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group
No intervention - deferred treatment group (Bilateral, sub-retinal administration of AAV5-hRKp.RPGR to be administered in the follow-up study)
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
Change from baseline to Week 52 in 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 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
Change from baseline in mean retinal sensitivity within the central 10 degrees excluding scotoma (MRS10) in static perimetry at Week 52 will be assessed.
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
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.
Time frame: From Baseline to Week 52
Change in Retinal Function as Assessed by Pointwise Response in Full Visual Field at Week 52
Pointwise response in full visual field at Week 52 will be assessed.
Time frame: 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
Pointwise response in worse-seeing eye in full visual field at Week 52 will be assessed.
Time frame: From Baseline to Week 52
Change in Retinal Function as Assessed by Pointwise Response in the Central 30 Degrees Visual Field at Week 52
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Shiley Eye Institute Jacobs Retina Center
La Jolla, California, United States
Childrens Hospital
Los Angeles, California, United States
Stanford Health Care
Palo Alto, California, United States
VitreoRetinal Associates, PA
Gainesville, Florida, United States
Emory University
Atlanta, Georgia, United States
Massachusetts General Hospital - Center for Celiac Research and Treatment
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
Retina Consultants of Houston
Bellaire, Texas, United States
...and 18 more locations
Pointwise response in the central 30 degrees visual field at Week 52 will be assessed.
Time frame: 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
Pointwise response in worse-seeing eye in the central 30 degrees visual field at Week 52 will be assessed.
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
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.
Time frame: 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
Change in functional vision by using VMA assessment in the "Worse-seeing Eye" 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
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 Visual Function as Assessed by Monocular Low Luminance Visual Acuity Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter score at 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.
Time frame: 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
Change From Baseline in visual function as assessed by monocular 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 Low Luminance Visual Acuity Using the ETDRS Chart Letter Score in Worse-seeing Eye at 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
Number of Participants with Ocular and Non-ocular Adverse Events
Number of participants with ocular and non-ocular adverse events will be assessed.
Time frame: Day 1 - Week 52
Number of Participants With Abnormalities in Laboratory Assessments
Number of participants with abnormalities in laboratory assessments will be assessed.
Time frame: Day 1 - 52 Weeks