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Clinical Trials in China / NCT06345898
Recruiting Phase EARLY_PHASE1

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

NCT06345898 · tracked via the Priya Life Science China tracker
Sponsor
West China Hospital
Phase
Phase EARLY_PHASE1
Started
2023-11-17
Last updated
2025-02-07

Condition(s) studied

X Linked Retinoschisis

Investigational drug(s) / intervention(s)

JWK002

JWK002: Subretinal administration of gene therapy vector JWK002 to one eye.

Study 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

Sex
MALE
Min age
5 Years
Max age
18 Years
Healthy volunteers
No
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; 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.

Primary outcome measure(s)

Trial sites (1)

FacilityCityRegionStatus
West China Hospital Chengdu Sichuan Recruiting

More West China Hospital trials in China

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06345898 on ClinicalTrials.gov ↗ ← All trials in China