X-linked retinoschisis
Conditions
Interventions
Low-dose group:Subretinal injection low dose of JWK002 in one eye
Medium dose group:Subretinal injection medium dose of JWK002 in one eye
Sponsors
West China Hospital, Sichuan University
Eligibility
Sex/Gender
Male
Age
5 Years to 17 Years
Inclusion criteria
Inclusion criteria: 1. 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; 2. Male, aged >= 5 years old and = 34 letters (equivalent to Snellen 20/200).
Exclusion criteria
Exclusion criteria: 1. Carbonic anhydrase inhibitor treatment within 3 months; 2. 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); 3. Those who study the retinal observation and examination caused by lens, cornea or other refractive interstitial turbidity; 4. There are eye conditions affecting subretinal injection or eye conditions affecting the determination of the study endpoint; 5. Patients who had undergone internal eye surgery within 6 months; 6. Patients with AAV8 neutralizing antibody titer >= 1:1000; 7. Have previously received any gene therapy or cell therapy; 8. Fertile subjects are unwilling to use contraception; 9. The presence of any of the following: an active infection that the investigator believes may affect the patient's participation in the study or affect the study results that requires systematic treatment; Hepatitis B surface antigen positive, hepatitis B virus deoxyribonucleic acid (HBV DNA) copy number > ULN; Hepatitis C virus (HCV) antibody positive and HCV-RNA copy number > ULN; Treponema pallidum antibody positive; Positive for human immunodeficiency virus (HIV) antibodies; 10. Malignant tumors (basal cell or squamous cell skin cancer) were diagnosed within 5 years prior to screening; 11. Has or has had a systemic immune system disease; 12. Laboratory values considered to be clinically significant were abnormal: alanine and/or aspartate transaminase > 2.5×ULN, total bilirubin > 1.5×ULN, serum creatinine > 1.5×ULN, prothrombin time >= 1.5×ULN, activated partial thromboplastin time >= 1.5×ULN; 13. Have a severe allergy or a known allergy to the drugs used in the treatment or examination in the study protocol, including the investigational drug allergy; 14. Other situations that the investigator considers inappropriate for participation in this study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| adverse events (AE);Any clinically significant abnormal changes in vital signs or physical examination;Clinically significant abnormal laboratory test results emerging after treatment; | — |
Secondary
| Measure | Time frame |
|---|---|
| Best Corrected Visual Acuity, BCVA;intraocular pressure;fundus condition;Fundus auto fluorescence;Retinal structural morphology;Central retinal thickness, CRT;Dark adaptation 3.0 a-wave amplitude, b-wave amplitude, and b/a ratio.; | — |
Countries
China
Contacts
Public ContactFang Lu
West China Hospital, Sichuan University
Outcome results
None listed