Bietti Crystalline Corneoretinal Dystrophy, Bietti Crystalline Dystrophy
Conditions
Keywords
gene replacement therapy, ZVS101e, AAV8-hCYP4V2, phase 3, Bietti crystalline corneoretinal dystrophy
Brief summary
This is a multi-center, randomized and controlled phase 3 clinical trial.
Detailed description
Subjects who have signed the written informed consent form and who were eligible for enrollment after screening will be randomly assigned to the treatment group or untreated control group. Each Participant in the treatment group will receive a unilateral subretinal injection of ZVS101e in the study eye. Participants in the control group will receive no treatment during the first 52 weeks of enrollment.
Interventions
DRUGZVS101e
subretinal injection of ZVS101e
Sponsors
Chigenovo Co., Ltd
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
SINGLE (Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. All of the following inclusion criteria should be met for enrollment into the trial: 2. Fully understand the purpose and requirements of this trial, voluntarily participate in the clinical trial and sign the informed consent form, and be able to complete all trial procedures as required by the protocol; 3. Clinical diagnosis of Bietti's crystalline dystrophy (BCD), age≥18 years ; 4. Genetic testing confirmed biallelic CYP4V2 mutations without other ophthalmic genetic diseases; 5. Best-corrected visual acuity of 5-60 ETDRS letters.
Exclusion criteria
1. The study eye has or has had macular lesions such as macular hole or macular neovascularization; glaucoma, diabetic retinopathy, or any other ocular disease that may preclude surgery or interfere with interpretation of the study endpoints 2. The study eye had received the following intraocular surgical treatments: retinal reattachment, vitrectomy; 3. The study eye had received any intraocular surgery, such as phacoemulsification 3 months prior to enrollment; 4. Previously treatment of either eye with gene therapy or stem cell therapy for BCD and other ocular diseases, including but not limited to viral vector gene therapy, RNA therapy; 5. Pregnant or lactating women;
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of study eyes with a ≥15-letter improvement from baseline in BCVA | From baseline to Week 52 | Best corrected visual acuity (BCVA) will be assessed using the Early Treatment Diabetic Retinopathy Study (ETDRS) chart. A 15-letter improvement over the baseline is clinical meaningful. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from Baseline in BCVA | From baseline to Week 52 | Best corrected visual acuity (BCVA) will be assessed using the Early Treatment Diabetic Retinopathy Study (ETDRS) chart. The larger the letter number, the better the vision. |
| Proportion of study eyes with a ≥10-letter improvement from baseline in BCVA | From baseline to Week 52 | Best corrected visual acuity (BCVA) will be assessed using the Early Treatment Diabetic Retinopathy Study (ETDRS) chart. |
| Change from baseline in multi-luminance mobility test (MLMT) score | From baseline to Week 52 | MLMT will be assessed using both eyes at different levels of illumination. A higher score indicates better functional vision. |
| Change from baseline in 25-Item visual function questionnaire (VFQ-25) score | From baseline to Week 52 | National eye institute 25-item visual function questionnaire (NEI VFQ-25) consists of a base set of 25 vision-targeted questions representing 11 vision-related constructs. All items are scored so that a high score represents better functioning. Each item is then converted to a 0 to 100 scale so that the lowest and highest possible scores are set at 0 and 100 points, respectively. |
| Change from Baseline in microperimetry | From baseline to Week 52 | Microperimetry will be measured using MP-3，changes in retinal sensitivity (dB) will be analyzed. |
| Change from baseline in OCT | From baseline to Week 52 | Retinal thickness will be assessed using OCT. |
| Incidence and severity of ocular and systemic adverse events | From baseline to Week 52 | An adverse event (AE) is any untoward medical occurrence in a clinical investigation participant administered a product; the event will not need to have a causal relationship with the treatment. |
Countries
China
Contacts
STUDY_DIRECTORJinlu Zhang, MD
Chigenovo Co., Ltd
Outcome results
None listed