This study will evaluate the safety and tolerability of ATSN-201 in male subjects ≥ 6 years of age with RS1-associated X-linked retinoschisis (XLRS).
Eligible patients who enroll in this study will receive a one-time subretinal injection of ATSN-201 in one eye. Safety and tolerability will be evaluated for 5 years.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
21
AAV.SPR-hGRK1-hRS1syn
Children's Hospital of Los Angeles
Los Angeles, California, United States
RECRUITINGBascom Palmer Eye Institute
Miami, Florida, United States
RECRUITINGOregon Health Sciences University
Portland, Oregon, United States
RECRUITINGSafety and tolerability as assessed by dose-limiting toxicities and treatment-emergent adverse events
Incidence of dose-limiting toxicities (DLTs) and treatment-emergent adverse events (TEAEs).
Time frame: From baseline to week 52
Visual acuity as assessed by best-corrected visual acuity
Change in best-corrected visual acuity (BCVA).
Time frame: From baseline to week 52
Visual acuity as assessed by low-luminance visual acuity
Change in low-luminance visual acuity (LLVA).
Time frame: From baseline to week 52
Visual function as assessed by contrast sensitivity
Change in contrast sensitivity.
Time frame: From baseline to week 52
Visual function as assessed by full-field electroretinogram parameters
Change in full-field electroretinogram (ffERG) parameters.
Time frame: From baseline to week 52
Visual function as assessed by microperimetry
Change in microperimetry.
Time frame: From baseline to week 52
Visual function as assessed by static perimetry
Change in static perimetry.
Time frame: From baseline to week 52
Macular structure as assessed by spectral domain optical coherence tomography
Change in spectral domain optical coherence tomography (SD-OCT).
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
RECRUITINGTime frame: From baseline to week 52
Macular structure as assessed by fundus autofluorescence
Change in fundus autofluorescence (FAF).
Time frame: From baseline to week 52
Subject-reported visual function as assessed by the NEI VFQ-25 in adult subjects
Change in the National Eye Institute's Visual Function Questionnaire 25 (NEI VFQ-25) score for adult subjects with scores from 0 to 100 where a higher score indicates a better outcome.
Time frame: From baseline to week 52
Subject-reported visual function as assessed by the CVAQC in pediatric subjects
Change in the Cardiff Visual Ability Questionnaire for Children (CVAQC) score for pediatric subjects with scores from -3.00 to +2.80 where a higher score indicates a worse outcome.
Time frame: From baseline to week 52