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.
Fully understand the purpose and requirements of the trial, voluntarily participate in the clinical trial and sign the informed consent, and be able to cooperate with the testing required in various studies according to the requirements of the program;
Male, ≥5 years old and < 18 years old (subject to the date of signing the informed consent);
Clinically diagnosed XLRS, identified by genetic testing as RS1 gene mutation, and do not carry other mutations causing ophthalmic genetic diseases;
Best corrected visual acuity (BCVA) of the study eye ≤63 letters (equivalent to Snellen 20/63); Best corrected visual acuity (BCVA) ≥34 letters (equivalent to Snellen 20/200).
Carbonic anhydrase inhibitor treatment within 3 months;
The presence of other conditions in the study eye that may cause vision loss (e.g., atrophy of the optic nerve, advanced glaucoma, uveitis, the presence of vesicular splitting chambers or vesicular retinal detachment that may affect the subretinal injection operator);
Those who study the retinal observation and examination caused by lens, cornea or other refractive interstitial turbidity;
There are eye conditions affecting subretinal injection or eye conditions affecting the determination of the study endpoint;
Sequential
JWK002
DrugSubretinal administration of gene therapy vector JWK002 to one eye.
Safety(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).
Best corrected visual acuity (BCVA)
Visual acuity of the study eye was assessed using the Early Treatment Diabetic Retinopathy Study (ETDRS)
Full-field electroretinogram(ff-ERG)
The ERG measurement will be performed based on the standards of international society for clinical electrophysiology of vision (ISCEV).
Macular structure as assessed by swept source optical coherence tomography
Change in swept source optical coherence tomography(SS-OCT)
Visual field
Change in visual field assessed by Humphrey perimetry
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