Prospective, Randomized, Sham-controlled, Dose-finding I/II Trial of Safety and Efficacy of Modified Optogenetic Gene Therapy (ZM-02 Injection)

Официальное название

A Prospective, Randomized, Sham-controlled, Dose-finding Phase 1/2 Two-part Trial to Evaluate the Safety, Tolerability, and Efficacy of ZM-02 Injection in Patients With Advanced Retinitis Pigmentosa

Обзор

This is a Phase 1/2, multi-center, randomized, sham-controlled, dose-escalation study evaluating ZM-02 in patients with advanced retinitis pigmentosa (RP).

Подробное описание

Retinitis pigmentosa (RP) is the most common inherited retinal disease, characterized by progressive loss of visual function that can lead to severe visual impairment or blindness with no effective treatment.

This Phase 1/2 study is designed to evaluate the safety, tolerability, and preliminary efficacy of a single unilateral intravitreal injection of ZM-02 at escalating dose levels in patients with advanced RP. The study includes a sham-controlled design and will assess safety outcomes as well as multiple functional and structural measures of visual performance over long-term follow-up.

Вмешательства

• Генная терапия ZM-02 (low dose)
  Single unilateral IVT injection of low-dose ZM-02 in the study eye
• Генная терапия ZM-02 (high dose)
  Single unilateral IVT injection of high-dose ZM-02 in the study eye
• Генная терапия ZM-02 (selected dose)
  Single unilateral IVT injection of selected-dose ZM-02 in the study eye
• Процедура Sham injection
  Sham IVT procedure in the study eye

Первичные конечные точки

• Safety and tolerability of ZM-02 [Срок оценки: Baseline to Week 52.]

Вторичные конечные точки (3)

• Change from Baseline in Best-Corrected Visual Acuity (BCVA) of Both Eyes [Срок оценки: Baseline to Week 52.]
• Change from Baseline in Functional Vision (MLMT) [Срок оценки: Baseline to Week 52.]
• Change from Baseline in Vision-Related Quality of Life (NEI VFQ-25) [Срок оценки: Baseline to Week 52.]

Критерии участия

Критерии включения

• Male or female participants aged 6 to 60 years.
• Clinical diagnosis of advanced retinitis pigmentosa (RP).
• Best corrected visual acuity at or below the protocol-defined threshold in the study eye.
• Presence of sufficient retinal structure in the study eye as determined by screening assessments.
• Ability to understand and sign informed consent (and assent, when applicable).

Критерии исключения

• Presence of other ocular conditions that could interfere with study assessments.
• History of significant ocular surgery in the study eye within a protocol-defined period.
• Active ocular infection or inflammation.
• Clinically significant systemic disease that could increase study risk or interfere with assessments.
• Positive screening for clinically significant infectious diseases, as defined in the protocol.
• Pregnant or breastfeeding individuals.
• Any condition that, in the investigator's judgment, would make the participant unsuitable for study participation.

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Последовательный дизайн

Маскирование

Четверное слепое

Основная цель

Лечение

Центры проведения

Список центров уточняется — проверьте первичный протокол.

Идентификаторы

NCT: NCT07282457 · ZM-02-201

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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