The purpose of this study is to assess the safety and tolerability of subretinal delivery of Adeno-associated Virus Vector (AAV5 hRKp.RPGR) gene therapy in adults and children with X-linked retinitis pigmentosa.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
24
AAV5-hRKp.RPGR will be administered sub-retinally.
Participants will not receive any intervention and will undergo follow-up assessment.
Massachusetts Eye and Ear Infirmary
Boston, Massachusetts, United States
University of Michigan Kellogg Eye Center
Ann Arbor, Michigan, United States
Moorfields Eye Hospital
London, United Kingdom
Number of Participants With Adverse Events (AE)
An AE is any untoward medical occurrence in a clinical study participant administered a pharmaceutical (investigational or non investigational) product. An AE does not necessarily have a causal relationship with the intervention. An AE can be any unfavorable and unintended sign (including an abnormal finding), symptom, or disease temporally associated with the use of a medicinal (investigational or non investigational) product, whether or not related to that medicinal (investigational or non investigational) product.
Time frame: From baseline up to 5.5 years
Change from Baseline in Best Corrected Visual Acuity (BCVA) by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Scores
Change from baseline in BCVA by ETDRS chart letter scores in monocular assessment of the second treated eye will be assessed. BCVA was measured using the ETDRS Scale (ranging from 0 to 100 letters) in the study eye. An increase in the number of letters read correctly means that vision has improved and vice-versa.
Time frame: Baseline, Months 12, 24, 36, 48 and 60
Change from Baseline in Low Luminance Visual Acuity (LLVA) by ETDRS Chart Letter Score
Change in LLVA by ETDRS chart letter scores from baseline in monocular assessment of second treated eye will be assessed. Visual function assessments included LLVA assessment in each eye by ETDRS letters. The letter score ranges from 0 (worse score) to 100 (best score), and a gain in LLVA from baseline indicates an improvement in visual acuity.
Time frame: Baseline, Months 12, 24, 36, 48 and 60
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