Safety and Efficacy of a Single Subretinal Injection of JWK002 Gene Therapy in Subjects With X-linked Retinoschisis(XLRS)

Clinical Study on the Safety and Efficacy of JWK002 Injection Administered as a Single Subretinal Injection in Subjects With X-linked Retinoschisis

NCT06345898•West China Hospital

View on ClinicalTrials.gov

Summary

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.

Eligibility

Age

5 - 18 years

Sex

MALE

Healthy Volunteers

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, ≥5 years old and \< 18 years old (subject to the date of signing the informed consent);
•3. Clinically diagnosed XLRS, identified by genetic testing as RS1 gene mutation, and do not carry other mutations causing ophthalmic genetic diseases;
•4. 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).

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;
+3 more criteria

Locations (1)

West China Hospital

Chengdu, Sichuan, China

Key Dates

Start Date

November 17, 2023

Primary Completion Date

November 30, 2033

Completion Date

November 30, 2033

Last Updated

February 7, 2025

Enrollment

ESTIMATED participants

Conditions

X Linked Retinoschisis

Interventions

JWK002

DRUG

Contacts

Licong Liang

CONTACT

932968425@qq.com

Kaiqin She

CONTACT

shekaiqin@wchscu.cn