Bietti Crystalline Corneoretinal Dystrophy
Conditions
Brief summary
To provide treatment options for the control group of subjects who completed the 52-week follow-up of the Phase III clinical trial (Protocol number: ZYA-2024-001) of ZVS101e in subjects with Bietti crystalline corneoretinal dystrophy (BCD).
Detailed description
The trial period lasts for a total of 8 weeks, including a 4-week screening period, a administration period, and visits on the 1st day, 7th day, and 4th week after administration. The patients will be followed up at the research center on the 1st day, 7th day and 4th week after administration to evaluate the safety.
Interventions
DRUGZVS101e
ZVS101e is a recombinant adeno-associated virus serotype 8 (rAAV8) vector expressing human CYP4V2 protein
Sponsors
Chigenovo Co., Ltd
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Subjects who were randomly assigned as the control group in a multicenter, randomized controlled, Phase III clinical trial (Protocol number: ZYA-2024-001) on the efficacy and safety of a single subretinal injection of ZVS101e in subjects with BCD and completed a 52-week safety follow-up were included. 2. Voluntarily participate in clinical trials and sign informed consent forms, and be willing to complete all trial procedures as required by the protocol. 3. Study the BCVA of the eye ≤60 ETDRS letters.
Exclusion criteria
1. The study eye currently has or has had macular degeneration such as macular holes or neovascularization in the macular area; Other ocular diseases such as glaucoma and diabetic retinopathy that may hinder surgery or interfere with the interpretation of the study endpoint. 2. The study eye had undergone retinal repositioning surgery, vitrectomy, or had undergone any intraocular surgery within 3 months prior to enrollment, such as phacoemulsification for cataract. 3. Within one month prior to enrollment, the patient had a viral infectious disease that may affect the efficacy and safety evaluation of the investigational drug or had received an antiviral vaccine. 4. Any eye has previously received gene therapy or stem cell therapy for BCD and other ocular diseases, including but not limited to other viral vector gene therapies, mRNA therapies, etc. 5. Known to be allergic to the drugs planned to be used in the study. 6. The following laboratory test abnormalities have clinical significance: Liver function: ALT or AST\>2 times the upper limit of the normal value; Abnormal coagulation function (prothrombin time ≥ the upper limit of normal value by 3 seconds, activated partial thromboplastin time ≥ the upper limit of normal value by 10 seconds). 7. Pregnant or lactating women.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence and severity of adverse events and serious adverse events | 4 weeks | This study specifies that the recording of adverse events begins on Day 0 (D0). Adverse events (AEs) include, but are not limited to, the following: 1. Worsening of pre-existing medical conditions/diseases (i.e., conditions present prior to enrollment in the clinical trial), including exacerbation of symptoms, signs, frequency of episodes, or abnormal laboratory test results; 2. Any newly occurring adverse medical conditions (including symptoms, signs, or newly diagnosed diseases); 3. Abnormal laboratory test values or results with clinical significance, or other safety assessments (e.g., vital signs measurements). |
Countries
China
Outcome results
None listed