Safety and Efficacy of a Single Subretinal Injection of JWK006 Gene Therapy in Subjects With Stargardt Disease(STGD1)

NCT06300476 | Active Not Recruiting Phase 1 DMC

• 1 other identifier: 2023-1511
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of the study is to assess the safety and efficacy of JWK006 in Stargardt Disease(STGD1). JWK006 is packed by adeno-associated virus vector that expressing ABCA4 gene.

Conditions

Stargardt Disease 1

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).

  5 years (Screening to 5 years post JWK006 administration)

Secondary Outcomes (4)

• Visual acuity

  5 years (Screening to 5 years post JWK006 administration)

• Ophthalmoscope Measurement

  5 years (Screening to 5 years post JWK006 administration)

• Qualitative and quantitative assessments of autofluorescence pattern (FAF)

  5 years (Screening to 5 years post JWK006 administration)

• multifocal electroretinogram (mfERG)

  5 years (Screening to 5 years post JWK006 administration)

Study Arms (3)

Low dose group

EXPERIMENTAL

Subretinal injection low dose of JWK006 in one eye

Genetic: JWK006

Medium dose group

EXPERIMENTAL

Subretinal injection medium dose of JWK006 in one eye

Genetic: JWK006

High dose group

EXPERIMENTAL

Subretinal injection high dose of JWK006 in one eye

Genetic: JWK006

Interventions

JWK006 GENETIC

Subretinal administration of gene therapy vector JWK006 to one eye.

High dose group; Low dose group; Medium dose group

Eligibility Criteria

• Age: 10 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Fully understand the purpose and requirements of this trial, voluntarily participate in the clinical trial and sign the informed consent form (underage subjects must be signed by their guardians), and be able to cooperate with the tests required in various studies according to the protocol requirements;
• Aged 10 to 18 years old (including the critical value, subject to the time of signing the informed consent form), regardless of gender;
• Clinical diagnosis of Stargardt disease(STGD1), macular changes consistent with the clinical manifestations of Stargardt disease(STGD1);
• The biallelic ABCA4 pathogenic mutation has been confirmed by genetic testing and does not carry other pathogenic mutations for ophthalmic genetic diseases;
• The best corrected visual acuity of the study eye is ≤ETDRS 68 letters (20/50); the best corrected visual acuity of the fellow eye is ≥20/400.

You may not qualify if:

• \) The study eye has other conditions that may cause vision loss (such as optic atrophy, advanced glaucoma, and uveitis); (2) The presence of lens, cornea or other refractive stromal opacity in the study eye affects retinal observation and examination; (3) There are eye conditions that affect subretinal injection, or eye conditions that affect the judgment of the study endpoint; (4) Those who have undergone intraocular surgery in the study eye within 6 months; (5) Patients with AAV8 neutralizing antibody titer ≥1:1000; (6) Have received any gene therapy or cell therapy in the past; (7) Subjects of childbearing age are unwilling to use contraceptive measures; (8) One of the following situations exists: the researcher believes that there is an active infection that may affect the patient's participation in the study or that affects the study results and requires systemic treatment; hepatitis B surface antigen is positive and hepatitis B virus deoxyribonucleic acid (HBV DNA) copies number \> ULN; hepatitis C virus (HCV) antibody positive, and HCV-RNA copy number \> ULN; Treponema pallidum antibody positive; human immunodeficiency virus (HIV) antibody positive; (9) Malignant tumors diagnosed within 5 years before screening (except for adequately treated cervical carcinoma in situ, basal cell or squamous epithelial cell skin cancer, and breast ductal carcinoma in situ after radical mastectomy); (10) Suffering or having suffered from systemic immune system diseases; (11) Abnormal laboratory values considered to be clinically significant: alanine aminotransferase and/or aspartate aminotransferase \>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; (12) There is severe allergy or known allergy to the drugs used for treatment or examination in the research protocol, including allergy to study drugs; (13) Pregnant and lactating women; subjects with childbearing potential who are unable to take effective contraceptive measures within 2 weeks before screening to 6 months after administration; (14) Other circumstances that the researcher believes are not suitable for participating in this study.

Sponsors & Collaborators

• West China Hospital: lead

Study Sites (1)

West China Hospital

Chengdu, Sichuan, 610044, China

Location

MeSH Terms

Conditions

Stargardt Disease

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Macular Degeneration; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Fang Lu, MD

  West China Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: 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 3, 2024
• First Posted: March 8, 2024
• Study Start: November 20, 2023
• Primary Completion (Estimated): December 30, 2026
• Study Completion (Estimated): December 30, 2029
• Last Updated: March 8, 2024

Record last verified: 2024-03

Locations

CN (1)