Safety and Efficacy Evaluation of LX111 Gene Therapy in DME Patients

Shanghai Jiao Tong University

Status and phase: Not yet enrolling; Early Phase 1

Conditions: Diabetic Macular Edema (DME)

Treatments: Genetic: LX111 Injection

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT07362927

LX111-01

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 32 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Willing to sign the informed consent, and willing to attend follow-up visits;
2. Age ≥ 18;
3. Type I or Type II diabetes mellitus with macular thickening secondary to DME involving the center of the fovea;
4. CST ≥ 300 μm in the study eye at Screening;
5. BCVA ETDRS letters between 19 and 73;
6. Participants must have received anti-VEGF therapy within 12 months prior to screening and demonstrated a meaningful response;
7. 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.

Exclusion criteria

1. Active proliferative diabetic retinopathy (PDR);
2. Presence of iris neovascularization in the study eye at Screening;
3. Retinal laser photocoagulation in the study eye within 3 months prior to Screening;
4. Prior gene therapy in the study eye;
5. The study eye has been treated with an intravitreal dexamethasone implant (Ozurdex®) within 6 months prior to Screening.
6. Systemic anti-VEGF treatment within 3 months before Screening;
7. 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;

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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32 participants in 3 patient groups

LX111 Dose Escalation up to 2 dose levels

Experimental group

Description:

Qualified participants will receive a single unilateral intravitreal injection of LX111 at Day 0.

Treatment:

Genetic: LX111 Injection

LX111 Dose Expansion Dose 1

Experimental group

Description:

Qualified participants will receive a single unilateral intravitreal injection of LX111 at Day 0.

Treatment:

Genetic: LX111 Injection

LX111 Dose Expansion Dose 2

Experimental group

Description:

Qualified participants will receive a single unilateral intravitreal injection of LX111 at Day 0.

Treatment:

Genetic: LX111 Injection