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Recruiting NCT06345898

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

Early Phase I Interventional X Linked Retinoschisis

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: JWK002.
Who it may be relevant to
Registry conditions: X Linked Retinoschisis. Basic parameters: 5 years — 18 years · Male.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
China
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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.

Interventions

  • Drug JWK002
    Subretinal administration of gene therapy vector JWK002 to one eye.

Primary outcome measures

  • Safety(Participants With Ocular and Non-ocular AEs (Adverse Events) and SAEs (Serious Adverse Events) [Time frame: baseline to day 7, day 14, month 1, 3, 6, 12]
Secondary outcome measures (4)
  • Best corrected visual acuity (BCVA) [Time frame: baseline to day 7, day 14, month 1, 2, 3, 6, 12]
  • Full-field electroretinogram(ff-ERG) [Time frame: baseline to month 1, 3, 6, 12]
  • Macular structure as assessed by swept source optical coherence tomography [Time frame: baseline to month 1, 3, 6, 12]
  • Visual field [Time frame: baseline to month 1, 3, 6, 12]

Eligibility criteria

Inclusion criteria

  • 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).

Exclusion criteria

  • 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;
  • Patients who had undergone internal eye surgery within 6 months;
  • Patients with AAV8 neutralizing antibody titer ≥1:1000;
  • Have previously received any gene therapy or cell therapy;
  • fertile subjects are unwilling to use contraception;
  • 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;
  • Malignant tumors (basal cell or squamous cell skin cancer) were diagnosed within 5 years prior to screening;
  • has or has had a systemic immune system disease;
  • 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Non-randomized
Model
Sequential
Masking
Single blind
Primary purpose
Treatment

Study locations

China · 1 center
  • West China Hospital — Chengdu

Identifiers

NCT: NCT06345898 · 2023-1479

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗