Biallelic mutations in ABCA4 can cause inherited retinal diseases (IRDs), including Stargardt disease type 1 (STGD-1).
Conditions
Interventions
Low-dose group:ZVS106e injection: 2.25 × 10^10 vg/eye
Medium-dose group:ZVS106e injection: 4.5×10^10 vg/eye
High-dose group:ZVS106e injection: 9.0×10^10 vg/eye
Sponsors
The Zhongshan Ophthalmic Center,Sun Yat-sen University
Eligibility
Sex/Gender
All
Age
8 Years to No maximum
Inclusion criteria
Inclusion criteria: 1. Age >= 8 years; 2. Diagnosed with inherited retinal degeneration caused by biallelic ABCA4 mutations, with no other inherited ocular diseases; 3. The target eye must meet the following criterion: best-corrected visual acuity of 0.5~2.0 LogMAR (inclusive of 0.5 and 2.0 LogMAR, equivalent to decimal visual acuity ranging from finger counting to 0.3); 4. The subject and their spouse agree to use effective contraception during the trial and for at least 1 year after dosing; 5. Voluntarily participate in the clinical trial and sign the informed consent form, and are able to complete all trial procedures as required by the protocol.
Exclusion criteria
Exclusion criteria: 1. The investigator judges that the target eye currently has or has previously had other macular disorders such as retinoschisis or epiretinal membrane; or has other ocular diseases that may interfere with surgery or interpretation of study endpoints; 2. Received pharmacological treatment within 3 months prior to screening that may affect trial observations; 3. The target eye has previously undergone the following intraocular surgeries: retinal reattachment surgery, vitrectomy; 4. Has a known ocular/visual disease, disorder, or lesion that causes or is associated with vision loss, or whose related treatments or therapies are known to cause or be associated with vision loss; 5. Had a viral infection within 1 month prior to enrollment that may affect the assessment of trial drug efficacy and safety, or received an antiviral vaccine; 6. Currently using or may require systemic medications that cause ocular toxicity, such as psoralen, rituximab, or tamoxifen; 7. Known hypersensitivity to any drug planned for use in this study; 8. Poorly controlled hypertension: systolic blood pressure >=160 mmHg or diastolic blood pressure >=100 mmHg; 9. Clinically significant abnormalities in the following laboratory tests: liver function—chronic liver disease with ALT or AST >=2 times the upper limit of normal (ULN); coagulation dysfunction—prothrombin time >=3 seconds above ULN, activated partial thromboplastin time >=10 seconds above ULN; serological viral testing—active hepatitis B, positive hepatitis C virus antibody (HCV-Ab), human immunodeficiency virus antibody (HIV-Ab), or syphilis antibody; 10. Has a past or current medical history that may affect trial safety or drug pharmacokinetics, particularly cardiovascular, hepatic, renal, endocrine, gastrointestinal, pulmonary, neurological, hematological, oncological, immunological, or metabolic disorders deemed clinically significant by the investigator; 11. Participated in any drug or medical device clinical trial within 3 months prior to screening; 12. Pregnant or lactating females; 13. Other reasons, as determined by the investigator, that render the subject unsuitable for participation in this clinical trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Types, severity, and incidence of ocular and systemic adverse events (AEs) and serious adverse events (SAEs) within 52 weeks after treatment (including dose-limiting toxicities (DLTs) during the dose; | — |
Secondary
| Measure | Time frame |
|---|---|
| Change in the score of the National Eye Institute Visual Functioning Questionnaire-25 (NEI-VFQ-25) for the treated eye of participants at 52 weeks after treatment;Change in the Best Corrected Visual Acuity (BCVA) of the treated eye at 52 weeks after treatment compared to the baseline;Assessment of the improvement in macular atrophy in the treated eye via Fundus Autofluorescence (FAF) at 52 weeks after treatment;Change in the Low-Luminance Visual Acuity (LLVA) of the treated eye at 52 weeks after treatment relative to the baseline;The mean change in microperimetry functional parameters of the treated eye at 52 weeks after treatment compared to the baseline;The mean change in dynamic perimetry functional parameters of the treated eye at 52 weeks after treatment compared to the baseline;Change in retinal structure assessed by Optical Coherence Tomography (OCT) in the treated eye at 52 weeks after treatment compared to the baseline;Change in the multifocal electroretinography (mfERG) waveform parameters of the treated eye at 52 weeks after treatment compared to the baseline;The mean change in color vision function parameters of the treated eye at 52 weeks after treatment compared to the baseline;Change in the Multiluminance Mobility Test (MLMT) of the treated eye in participants at 52 weeks after treatment compared to the baseline; | — |
Countries
China
Contacts
Public ContactZhang Qingjiong
The Zhongshan Ophthalmic Center,Sun Yat-sen University
Outcome results
None listed