A Safety Study of Contralateral Eye Dosing of VGR-R01 in Participants With Bietti's Crystalline Dystrophy (BCD)

Официальное название

A Clinical Study Evaluating Subretinal Injection of VGR-R01 in the Contralateral Eye of Participants With Bietti's Crystalline Dystrophy Who Had Received VGR-R01 Administration

Обзор

This is a multi-centre, single- arm, non-randomized, open-label phase 1/2 clinical trial which enables dosing of the fellow eyes of patients who received VGR-R01 administration in previous studies.

Подробное описание

VGR-R01 is a novel Adeno-associated virus (AAV) vector carrying the human Cytochrome P450 Family 4 Subfamily V Member 2 (CYP4V2) coding sequence. This study will evaluate the safety and efficacy of VGR-R01 administered in the contralateral eye (the second treated eye) of subjects enrolled in the VGR-R01-001 and VGR-R01-101 studies, along with assessments of immunogenicity and vector shedding. Additionally, the long-term safety and efficacy of VGR-R01 treatment will be continuously assessed for up to 5 years after the last dose.

Вмешательства

• Биопрепарат VGR-R01
  CYP4v2-coding gene delivered by AAV vector

Первичные конечные точки

• Incidence of adverse events and serious adverse events [Срок оценки: Up to Year 5]
• Number of participants with clinically significant change from baseline in vital signs, clinically laboratory abnormalities and ophthalmic examination findings [Срок оценки: Up to Year 5]

Вторичные конечные точки (7)

• Change from baseline in BCVA [Срок оценки: Year 5]
• Change from baseline in multi-luminance mobility test (MLMT) score [Срок оценки: Year 5]
• Change from baseline in optical coherence tomography (OCT) [Срок оценки: Year 5]
• Number of subjects with the presence of immunogenicity [Срок оценки: Year 5]
• Number of subjects with the presence of vector shedding [Срок оценки: Year 5]
• Fixation stability [Срок оценки: Year 5]
• Light sensitivity [Срок оценки: Year 5]

Критерии участия

Критерии включения

• Participants received VGR-R01 administration in the VGR-R01-001 or VGR-R01-101 studies.
• Able to provide informed consent and comply with requirements of the study;
• Hand Motion ≤ BCVA ≤ 75 ETDRS letters in the second treated eye.

Критерии исключения

• Have insufficient viable retinal photoreceptor cells based on investigator's decision;
• Have current ocular or periocular infections, or endophthalmitis;
• Have any significant ocular disease/disorder other than BCD, including age-related macular degeneration, diabetic retinopathy, optic neuropathy, significant lens opacity, glaucoma, uveitis, retinal detachment, etc;
• Have intraocular surgery history except cataract surgery in the study eye;
• Have or potentially require of systemic medications that may cause eye injure;
• Have contraindications for corticosteroids or immunosuppressant;
• Abnormal coagulation function or other clinically significant abnormal laboratory results;
• Have malignancies or history of malignancies;
• History of immunodeficiency (acquired or congenital); Other protocol defined Inclusion/Exclusion criteria may apply.

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

• Beijing Tongren Hospital — Пекин
• Shanghai General Hospital (Shanghai First People's Hospital) — Шанхай

Идентификаторы

NCT: NCT07586306 · VGR-R01-102

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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