This trial is to evaluate the safety and efficacy of JWK002 treatment of X-linked retinoschisis(XLRS). This study will enroll subjects aged 5-18 years old to receive a sub-retinal injection of JWK002.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Enrollment
12
Subretinal administration of gene therapy vector JWK002 to one eye.
West China Hospital
Chengdu, Sichuan, China
RECRUITINGSafety(Participants With Ocular and Non-ocular AEs (Adverse Events) and SAEs (Serious Adverse Events)
The primary outcome measures are safety, determined by the number of ocular and non-ocular Study Drug-related adverse events (SDAE), treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs).
Time frame: baseline to day 7, day 14, month 1, 3, 6, 12
Best corrected visual acuity (BCVA)
Visual acuity of the study eye was assessed using the Early Treatment Diabetic Retinopathy Study (ETDRS)
Time frame: baseline to day 7, day 14, month 1, 2, 3, 6, 12
Full-field electroretinogram(ff-ERG)
The ERG measurement will be performed based on the standards of international society for clinical electrophysiology of vision (ISCEV).
Time frame: baseline to month 1, 3, 6, 12
Macular structure as assessed by swept source optical coherence tomography
Change in swept source optical coherence tomography(SS-OCT)
Time frame: baseline to month 1, 3, 6, 12
Visual field
Change in visual field assessed by Humphrey perimetry
Time frame: baseline to month 1, 3, 6, 12
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