The purpose of the study is to assess the safety and tolerability of bilateral subretinal delivery of adeno-associated virus vector with a serotype 5 capsid human rhodopsin kinase promoter. retinitis pigmentosa guanosine triphosphatase regulator (AAV5-hRKp.RPGR).
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
4
AAV5-hRKp.RPGR will be administered by subretinal injection using a standardized surgical procedure.
AAV5-hRKp.RPGR will be administered by subretinal injection using a standardized surgical procedure.
National Hospital Organization Tokyo Medical Center
Meguro-ku, Japan
Number of Participants with Adverse Event (AEs)
An AE is any untoward medical occurrence in a clinical study participant administered a investigational or non-investigational medicinal product. An AE does not necessarily have a causal relationship with the treatment.
Time frame: Up to 60 Months
Number of Participants with Abnormalities in Clinical Laboratory Assessments
Number of participants with abnormalities in clinical laboratory assessment (hematology and serum chemistry) will be reported.
Time frame: Up to 60 Months
Change From Baseline in Low Luminance Visual Acuity by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score in Monocular Assessment at Week 52
Change from baseline in low luminance visual acuity by ETDRS chart score in monocular assessment at week 52 will be reported.
Time frame: Baseline - Week 52
Change From Baseline in Best Corrected Visual Acuity (BCVA) by Early Treatment Diabetic Retinopathy Study Chart Letter Score in Monocular Assessment at Week 52
Change from baseline in BCVA by ETDRS chart letter score in monocular assessment at week 52 will be reported.
Time frame: Baseline - Week 52
Change From Baseline in Low Luminance Visual Acuity by Early Treatment Diabetic Retinopathy Study Chart Letter Score in Worse-seeing Eye at Week 52
Change from baseline in low luminance visual acuity by ETDRS chart letter score in worse-seeing eye at week 52 will be reported.
Time frame: Baseline - Week 52
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