ZVS101e in Patients With Bietti's Crystalline Dystrophy

Официальное название

Clinical Translation and Application Protocol for ZVS101e in the Gene Therapy of Bietti Crystalline Corneoretinal Dystrophy

Обзор

The goal of this clinical trial is to evaluate the safety and early effectiveness of ZVS101e in patients with Bietti crystalline dystrophy who meet the eligibility criteria for treatment under the translational application program in the Hainan Boao Lecheng International Medical Tourism Pilot Zone. Participants will: undergo screening and baseline assessments to confirm eligibility; receive a single subretinal injection of ZVS101e in the study eye; complete follow-up visits over 4 weeks after treatment for safety monitoring and assessment of early effectiveness.

Вмешательства

• Препарат ZVS101e
  ZVS101e injection contains a recombinant adeno-associated virus serotype 8 (rAAV8) vector that expresses human CYP4V2 protein.

Первичные конечные точки

• Change from baseline in Best corrected visual acuity (BCVA) [Срок оценки: From enrollment to the end of treatment at 52 weeks]
• The incidence and severity of adverse events and serious adverse events [Срок оценки: From enrollment to the end of treatment at 52 weeks]

Критерии участия

Критерии включения

• Voluntarily participate in the research and sign the informed consent form, and be willing to complete the entire process according to the protocol requirements;
• Clinically diagnosed with Bietti's Crystalline Dystrophy (BCD), age ≥ 18 years;
• Confirmed by genetic testing to carry homozygous or compound heterozygous mutations in CYP4V2, and not complicated with other ophthalmic genetic diseases;
• Target eye BCVA ≤ 60 ETDRS letters.

Критерии исключения

Subjects will be excluded if they meet any 1 of the following exclusion criteria:

• The target eye currently has or has a history of macular lesions; suffers from ocular diseases that may hinder surgery or interfere with the interpretation of study endpoints;
• The target eye has previously undergone retinal reattachment surgery, vitrectomy, or any intraocular surgery within 3 months before enrollment;
• Within 1 month before enrollment, suffered from viral infectious diseases that may affect the evaluation of the efficacy and safety of the investigational drug, or received antiviral vaccines;
• The target eye has previously received gene therapy or stem cell therapy for BCD or other ocular diseases;
• Known allergy to the drugs planned for use in the study;
• The laboratory abnormalities are considered clinically significant;
• Pregnant or lactating females.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Китай · 2 центра

• Boao Super Hospital — Qionghai
• Hainan Boao Ophthalmology Center, Zhongshan Ophthalmic Center, Sun Yat-sen University — Qionghai

Публикации

• Wang J, Zhang J, Yu S, Li H, Chen S, Luo J, Wang H, Guan Y, Zhang H, Yin S, Wang H, Li H, Liu J, Zhu J, Yang Q, Sha Y, Zhang C, Yang Y, Yang X, Zhang X, Zhao X, Wang L, Yang L, Wei W. Gene replacement therapy in Bietti crystalline corneoretinal dystrophy: an open-label, single-arm, exploratory trial. Signal Transduct Target Ther. 2024 Apr 24;9(1):95. doi: 10.1038/s41392-024-01806-3. PMID 38653979
• Jia R, Meng X, Chen S, Zhang F, Du J, Liu X, Yang L. AAV-mediated gene-replacement therapy restores viability of BCD patient iPSC derived RPE cells and vision of Cyp4v3 knockout mice. Hum Mol Genet. 2023 Jan 1;32(1):122-138. doi: 10.1093/hmg/ddac181. PMID 35925866

Идентификаторы

NCT: NCT07653971 · ZYA-2025-003

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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