Safety and Efficacy Evaluation of LX111 Gene Therapy in DME Patients

Официальное название

An Exploratory Clinical Study Evaluating LX111 Gene Therapy in Patients With Diabetic Macular Edema (DME)

Обзор

The goal of this study is to evaluate the safety and efficacy of LX111 treatment of DME. This study will enroll participants aged ≥ 18 vears old to receive a single unilateral intravitreal (lVT) injection of LX111 to evaluate its safety and efficacy.

Подробное описание

This trial is a prospective, multicenter, dose-ranging trial to evaluate the safety and efficacy of LX111 in participants with DME. The trial will be conducted in two parts: Dose Confirmation and Dose Expansion.

Вмешательства

• Генная терапия LX111 Injection
  LX111 is an rAAV gene therapy vector carrying a coding sequence for VEGF-trap.

Первичные конечные точки

• Adverse events and serious adverse events in the eyes and throughout the body within 364 days after LX111 treatment [Срок оценки: 52 weeks]
• Dose Limiting Toxicity [Срок оценки: 4 weeks]

Вторичные конечные точки (4)

• Mean changes in BCVA from Baseline [Срок оценки: 12 weeks, 36 weeks, 52 weeks]
• Mean changes in Central Subfield Thickness (CST) from Baseline [Срок оценки: 12 weeks, 36 weeks, 52 weeks]
• The percentage of participants who received anti-VEGF supplemental injection within 52 weeks after LX111 treatment [Срок оценки: 52 weeks]
• Proportion of participants achieving an improvement or worsening in DR in the study eye per the ETDRS-DRSS on ultra-wide field fundus photography. [Срок оценки: 52 weeks]

Критерии участия

Критерии включения

• Willing to sign the informed consent, and willing to attend follow-up visits;
• Age ≥ 18;
• Type I or Type II diabetes mellitus with macular thickening secondary to DME involving the center of the fovea;
• CST ≥ 300 μm in the study eye at Screening;
• BCVA ETDRS letters between 19 and 73;
• Participants must have received anti-VEGF therapy within 12 months prior to screening and demonstrated a meaningful response;
• Male subjects whose partner is a fertile female or female subjects who are fertile, agree to take effective contraceptive measures from the screening period until the last follow-up.

Критерии исключения

• Active proliferative diabetic retinopathy (PDR);
• Presence of iris neovascularization in the study eye at Screening;
• Retinal laser photocoagulation in the study eye within 3 months prior to Screening;
• Prior gene therapy in the study eye;
• The study eye has been treated with an intravitreal dexamethasone implant (Ozurdex®) within 6 months prior to Screening.
• Systemic anti-VEGF treatment within 3 months before Screening;
• Received an investigational drug, agent, device, or therapy (ocular or non-ocular) in the 3 months (or at least 5 half-lives, whichever is longer) prior to Screening;

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Нерандомизированное

Модель

Последовательный дизайн

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Китай · 2 центра

• Shanghai General Hospital — Шанхай
• Zhongshan Hospital — Шанхай

Идентификаторы

NCT: NCT07362927 · LX111-01

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗