AAV-mediated gene-replacement therapy restores viability of BCD patient iPSC derived RPE cells and vision of Cyp4v3 knockout mice.
Ruixuan Jia, Xiang Meng, Shaohong Chen...
https://pubmed.ncbi.nlm.nih.gov/35925866Actively Recruiting
Phase 3
Age: 18Years +
All Genders
ID06743646
A Phase 3 Randomized Trial Evaluating the Safety and Effect of a Single Subretinal Injection of ZVS101e in Patients With Bietti's Crystalline Dystrophy
Led by Chigenovo Co., Ltd · Updated on 2025-01-03
62
Participants Needed
8
Research Sites
287 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This research aims to evaluate the safety and effectiveness of ZVS101e for treating patients with Bietti's Crystalline Dystrophy (BCD), a rare eye condition affecting vision. The study is a multicenter, randomized, controlled phase 3 clinical trial involving adults aged 18 years and older who have confirmed genetic mutations linked to BCD and specific levels of visual acuity. Participants are randomly assigned to either a treatment group or a control group. Those in the treatment group receive a single subretinal injection of ZVS101e in one eye, while participants in the control group do not receive any treatment during the first 52 weeks. This approach allows comparison between treated and untreated patients over one year. Throughout the 52-week study, researchers will monitor participants' vision, specifically measuring the proportion of study eyes that improve by 15 or more letters in best-corrected visual acuity (BCVA) from baseline. Participants will undergo various assessments, including genetic testing confirmation, visual acuity tests, and regular follow-ups to evaluate safety and outcomes. The trial also carefully monitors for any eye conditions or complications that might affect surgery or study results.
CONDITIONS
Official Title
Efficacy and Safety of ZVS101e in Patients With Bietti 's Crystalline Dystrophy
Who Can Participate
Age: 18Years +
All Genders
Eligibility Criteria
You may qualify if you...
- Understand the trial purpose and requirements, agree to participate voluntarily, and can complete all procedures
- Clinical diagnosis of Bietti's crystalline dystrophy (BCD), age 18 years or older
- Genetic testing confirming biallelic CYP4V2 mutations without other eye genetic diseases
- Best-corrected visual acuity between 5 and 60 ETDRS letters
You will not qualify if you...
- Study eye has or had macular lesions like macular hole or neovascularization, glaucoma, diabetic retinopathy, or other eye diseases that affect surgery or study results
- Study eye had retinal reattachment or vitrectomy surgery
- Study eye had any intraocular surgery such as phacoemulsification within 3 months before enrollment
- Previous gene therapy or stem cell treatment in either eye for BCD or other eye diseases
- Pregnant or breastfeeding women
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Trial Site Locations
Total: 8 locations
1
Peking University Third Hospital
Beijing, Beijing Municipality, China
Not Yet Recruiting
2
Zhongshan Ophthalmic Center, Sun Yat-sen University
Guangzhou, Guangdong, China
Not Yet Recruiting
3
The First Affiliated Hospital of Harbin Medical University
Haerbin, Heilongjiang, China
Not Yet Recruiting
4
Henan Provincial People's Hospital
Zhengzhou, Henan, China
Not Yet Recruiting
5
Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, China
Not Yet Recruiting
6
West China Hospital of Sichuan University
Chengdu, Sichuan, China
Not Yet Recruiting
7
Tianjin Medical University Eye Hospital
Tianjin, Tianjin Municipality, China
Actively Recruiting
8
Eye Hospital, Wenzhou Medical University
Wenzhou, Zhejiang, China
Not Yet Recruiting
Loading map...
Research Team
J
Jinlu Zhang, MD
T
Tingting Wu, BS
How is the study designed?
Study Type
INTERVENTIONAL
Masking
SINGLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here
Published Research Related To This Trial
Gene replacement therapy in Bietti crystalline corneoretinal dystrophy: an open-label, single-arm, exploratory trial.
Jinyuan Wang, Jinlu Zhang, Shicheng Yu...
https://pubmed.ncbi.nlm.nih.gov/38653979