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

NCT06345898 | Recruiting Early Phase 1

• 1 other identifier: 2023-1479
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

X Linked Retinoschisis

Keywords

X Linked Retinoschisis

Outcome Measures

Primary Outcomes (1)

• 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).

  baseline to day 7, day 14, month 1, 3, 6, 12

Secondary Outcomes (4)

• Best corrected visual acuity (BCVA)

  baseline to day 7, day 14, month 1, 2, 3, 6, 12

• Full-field electroretinogram(ff-ERG)

  baseline to month 1, 3, 6, 12

• Macular structure as assessed by swept source optical coherence tomography

  baseline to month 1, 3, 6, 12

• Visual field

  baseline to month 1, 3, 6, 12

Study Arms (2)

JWK002 at Low Dose Group

EXPERIMENTAL

Subretinal injection low dose of JWK002 in one eye

Drug: JWK002

JWK002 at Medium Dose Group

EXPERIMENTAL

Subretinal injection medium dose of JWK002 in one eye

Drug: JWK002

Interventions

JWK002 DRUG

Subretinal administration of gene therapy vector JWK002 to one eye.

JWK002 at Low Dose Group; JWK002 at Medium Dose Group

Eligibility Criteria

• Age: 5 Years - 18 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• West China Hospital: lead

Study Sites (1)

West China Hospital

Chengdu, Sichuan, 610023, China

RECRUITING

MeSH Terms

Conditions

Retinoschisis

Condition Hierarchy (Ancestors)

Retinal Degeneration; Retinal Diseases; Eye Diseases

Study Officials

• Fang Lu

  lufang@wchscu.cn

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Licong Liang

CONTACT

932968425@qq.com

Kaiqin She

CONTACT

shekaiqin@wchscu.cn

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NON RANDOMIZED
• Masking: SINGLE
• Who Masked: PARTICIPANT
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: March 28, 2024
• First Posted: April 3, 2024
• Study Start: November 17, 2023
• Primary Completion (Estimated): November 30, 2033
• Study Completion (Estimated): November 30, 2033
• Last Updated: February 7, 2025

Record last verified: 2025-02

Locations

CN (1)