Treatment for Bietti's Crystalline Dystrophy using ZVS101e
A Multicenter, Randomized，Controlled，Phase 3 Clinical Trial to Evaluate the Efficacy and Safety of ZVS101e Administered as a Single Subretinal Injection in Subjects With Bietti's Crystalline Dystrophy (BCD)
This study is testing a new gene therapy called ZVS101e to see if it can improve vision in people with Bietti's crystalline dystrophy.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 62 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Chigenovo Co., Ltd Research network |
| Locations | 8 sites (Beijing, Beijing Municipality and 7 other locations) |
| Trial ID | NCT06743646 on ClinicalTrials.gov |
What this trial studies
This phase 3 clinical trial evaluates the efficacy and safety of ZVS101e, a gene replacement therapy, in patients diagnosed with Bietti's crystalline dystrophy. Participants will be randomly assigned to receive either a unilateral subretinal injection of ZVS101e or no treatment for the first 52 weeks. The study aims to assess visual acuity improvements and overall safety of the intervention compared to a control group. Eligible participants must have confirmed genetic mutations and meet specific visual acuity criteria.
Who should consider this trial
Good fit: Ideal candidates are adults aged 18 and older with a clinical diagnosis of Bietti's crystalline dystrophy and confirmed biallelic CYP4V2 mutations.
Not a fit: Patients with other significant ocular diseases or prior intraocular surgeries may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve vision and quality of life for patients with Bietti's crystalline dystrophy.
How similar studies have performed: Other studies using gene replacement therapies for retinal diseases have shown promising results, indicating potential for success in this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
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;
Where this trial is running
Beijing, Beijing Municipality and 7 other locations
- Peking University Third Hospital — Beijing, Beijing Municipality, China (Not_yet_recruiting)
- Zhongshan Ophthalmic Center, Sun Yat-sen University — Guangzhou, Guangdong, China (Not_yet_recruiting)
- The First Affiliated Hospital of Harbin Medical University — Haerbin, Heilongjiang, China (Not_yet_recruiting)
- Henan Provincial People's Hospital — Zhengzhou, Henan, China (Not_yet_recruiting)
- Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine — Shanghai, Shanghai Municipality, China (Not_yet_recruiting)
- West China Hospital of Sichuan University — Chengdu, Sichuan, China (Not_yet_recruiting)
- Tianjin Medical University Eye Hospital — Tianjin, Tianjin Municipality, China (Recruiting)
- Eye Hospital, Wenzhou Medical University — Wenzhou, Zhejiang, China (Not_yet_recruiting)
Study contacts
- Study coordinator: Jinlu Zhang, MD
- Email: zhangjinlu@chinagene.cc
- Phone: +86-15810570898
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.