The Safety and Efficacy of ZVS106e in the Treatment of IRDs Caused by Biallelic Mutations in ABCA4

NCT07241169 | Recruiting Early Phase 1

• 1 other identifier: ZYA-2025-002
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 2

Brief Summary

To evaluate the preliminary safety of monocular and single subretinal injection of ZVS106e injection in the treatment of patients with hereditary retinal degeneration (ABCA4-IRDS) caused by ABCA4 biallelic mutations.

Conditions

Stargardt

Keywords

ZVS106e

Outcome Measures

Primary Outcomes (1)

• Evaluate the safety and tolerability of subretinal injection of ZVS106e solution

  Types, severity, and incidence of adverse events (AE) and serious adverse events (SAE) in the eyes and throughout the body within 52 weeks post-treatment, including dose-limiting toxicities (DLT) during the dose escalation phase.

  52 weeks post-treatment

Secondary Outcomes (4)

• Change from baseline in best-corrected visual acuity (BCVA)

  52 weeks post-treatment

• Change from Baseline in Visual function metrics

  52 weeks post-treatment

• Change from Baseline in OCT

  52 weeks post-treatment

• Change from Baseline in Fundus autofluorescence (FAF)

  52 weeks post-treatment

Study Arms (1)

Single arm

EXPERIMENTAL

All patients enrolled in the study will receive a single subretinal injection of ZVS106e in one eye

Drug: ZVS106e

Interventions

ZVS106e DRUG

ZVS106e is A colorless, clear and transparent liquid, containing two active ingredients, ZVS106E-A and ZVS106E-B. The viral vectors used for both active ingredients are rAAV8

Also known as: rAAV8

Single arm

Eligibility Criteria

• Age: 8 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Age ≥8 years old;
• Patients diagnosed with hereditary retinal degeneration caused by ABCA4 biallelic mutations through genetic testing, and without other ophthalmic genetic diseases;
• The target eye must meet the following requirements: the best corrected visual acuity is 0.5 to 2.0 LogMAR (including 0.5 and 2.0 LogMAR, equivalent to the decimal visual acuity index up to 0.3);
• The subject and his or her spouse agree to take effective contraceptive measures during the trial period and for at least one year after administration.
• Voluntarily participate in clinical trials and sign informed consent forms, and be able to complete all trial processes as required by the protocol.

You may not qualify if:

• The researchers determined that the target eye currently has or has previously had other macular lesions such as retinal schisis or epiretinal membrane. Or have other eye diseases that may hinder the surgery or interfere with the interpretation of the study endpoint;
• Having received drug treatment that may affect the observation of the trial within the three months prior to screening;
• The target eye has undergone the following intraocular surgeries: retinal repositioning and vitrectomy;
• There are known eye/visual diseases, disorders or lesions that cause or are related to vision loss, or whose related treatments or therapies are known to cause or are related to vision loss;
• Having suffered from a viral infectious disease that may affect the efficacy and safety evaluation of the investigational drug or having received an antiviral vaccine within one month prior to enrollment;
• Systemic medications that are currently in use or may be required to cause eye toxicity, such as psoralen, risselinic acid or tamoxifen, etc.
• Known to be allergic to the drugs planned to be used in the study;
• Suffering from poorly drug-controlled hypertension: systolic blood pressure ≥160 mmHg or diastolic blood pressure ≥100 mmHg;
• The following laboratory test abnormalities have clinical significance:
• Liver function: Chronic liver disease, elevation of ALT or AST \>2 times the upper limit of the normal value; Those with abnormal coagulation function (prothrombin time ≥ 3 seconds above the upper limit of the normal value, activated partial thromboplastin time ≥ 10 seconds above the upper limit of the normal value); Serum virological examination: Positive for active hepatitis B, hepatitis C virus antibody (HCV-Ab), human immunodeficiency virus antibody (HIV-Ab), or syphilis antibody;
• There is any past or current medical history that may affect the safety of the trial or the in vivo process of the drug, especially a history of diseases such as cardiovascular, liver, kidney, endocrine, digestive tract, lung, nervous, hematological, tumor, immune or metabolic disorders that the investigators consider to have clinical significance;
• Those who have participated in any clinical trials of drugs or medical devices within the three months prior to screening;
• Pregnant or lactating women; According to the researcher's judgment, those who are deemed unsuitable to participate in this clinical trial for other reasons

Sponsors & Collaborators

• Zhongshan Ophthalmic Center, Sun Yat-sen University: lead
• Chigenovo Co., Ltd: collaborator

Study Sites (2)

Peking University Third Hospital

Beijing, Beijing Municipality, China

RECRUITING

Zhongshan Ophthalmic Center, Sun Yat-sen University

Guangzhou, Guangdong, China

NOT YET RECRUITING

Study Officials

• qingjiong zhang, M.D

  PI

  STUDY DIRECTOR

• lin Lv, M.D

  PI

  PRINCIPAL INVESTIGATOR

Central Study Contacts

dayou Ding

CONTACT

010-82266699; dingdayou@chinagene.cc

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: PI

Study Record Dates

• First Submitted: September 29, 2025
• First Posted: November 21, 2025
• Study Start: December 29, 2025
• Primary Completion (Estimated): November 30, 2028
• Study Completion (Estimated): December 30, 2028
• Last Updated: April 16, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

CN (2)