Efficacy and Safety of ZVS101e in Patients With Bietti 's Crystalline Dystrophy

Phase 3

Recruiting

• Conditions: Bietti Crystalline Corneoretinal Dystrophy; Bietti Crystalline Dystrophy
• Interventions: Drug: ZVS101e

• Registration Number: NCT06743646
• Lead Sponsor: Chigenovo Co., Ltd

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.

Study Timeline

• Study First Submitted: 2024-12-17
• Study First Submitted QC: 2024-12-17
• Study First Posted: 2024-12-20
• Study Start: 2024-12-27
• Status Verified: 2025-01
• Last Update Submitted: 2025-01-01
• Last Update Posted: 2025-01-03
• Primary Completion: 2026-06-30
• Study Completion: 2030-06-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 62

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.

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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;

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Treatment group	ZVS101e	Each Participant in this group will receive a unilateral subretinal injection of ZVS101e in the study eye

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
Change from baseline in OCT	From baseline to Week 52	Retinal thickness will be assessed using OCT.
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.
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.

Trial Locations

Locations (8)

Peking University Third Hospital; 🇨🇳 Beijing, Beijing, China

Zhongshan Ophthalmic Center, Sun Yat-sen University; 🇨🇳 Guangzhou, Guangdong, China

The First Affiliated Hospital of Harbin Medical University; 🇨🇳 Haerbin, Heilongjiang, China

Henan Provincial People's Hospital; 🇨🇳 Zhengzhou, Henan, China

Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine; 🇨🇳 Shanghai, Shanghai, China

West China Hospital of Sichuan University; 🇨🇳 Chengdu, Sichuan, China

Tianjin Medical University Eye Hospital; 🇨🇳 Tianjin, Tianjin, China

Eye Hospital, Wenzhou Medical University; 🇨🇳 Wenzhou, Zhejiang, China