This is a multi-center, randomized and controlled phase 3 clinical trial.
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
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;
Primary outcome measure(s)
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.
Trial sites (8)
Facility
City
Region
Status
Peking University Third Hospital
Beijing
Beijing Municipality
Zhongshan Ophthalmic Center, Sun Yat-sen University
Guangzhou
Guangdong
The First Affiliated Hospital of Harbin Medical University
Haerbin
Heilongjiang
Henan Provincial People's Hospital
Zhengzhou
Henan
Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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