Safety and Efficacy Evaluation of LX111 Gene Therapy in DME Patients

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

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.

Study Overview

• Status: Recruiting
• Conditions: Diabetic Macular Edema (DME)
• Intervention / Treatment: Genetic: LX111 Injection

Detailed 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.

• Study Type: Interventional
• Enrollment (Estimated): 32
• Phase: Early Phase 1

Contacts and Locations

• Study Contact: Name: Li Su; Phone Number: +86 02136126254; Email: li.su@shgh.cn

Study Locations

• China
  • Shanghai, China
    • Recruiting
    • Shanghai General Hospital
    • Principal Investigator: Li Su
    • Contact: Li Su; Phone Number: +86 02136126254; Email: li.su@shgh.cn
  • Shanghai, China
    • Not yet recruiting
    • ZhongShan Hospital
    • Contact: Yuanzhi Yuan; Phone Number: +86 02164041990; Email: Yuan.yuanzhi@zs-hospital.sh.cn
    • Principal Investigator: Yuanzhi Yuan

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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;

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: LX111 Dose Escalation up to 2 dose levels; Qualified participants will receive a single unilateral intravitreal injection of LX111 at Day 0.	Genetic: LX111 Injection; LX111 is an rAAV gene therapy vector carrying a coding sequence for VEGF-trap.
Experimental: LX111 Dose Expansion Dose 1; Qualified participants will receive a single unilateral intravitreal injection of LX111 at Day 0.	Genetic: LX111 Injection; LX111 is an rAAV gene therapy vector carrying a coding sequence for VEGF-trap.
Experimental: LX111 Dose Expansion Dose 2; Qualified participants will receive a single unilateral intravitreal injection of LX111 at Day 0.	Genetic: LX111 Injection; LX111 is an rAAV gene therapy vector carrying a coding sequence for VEGF-trap.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Dose Limiting Toxicity	The incidence of DLT in each dose group.	4 weeks
Adverse events and serious adverse events in the eyes and throughout the body within 364 days after LX111 treatment	Incidence of adverse events and serious adverse events within 52 weeks of LX111 intravitreal injection in each dose group.	52 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Mean changes in BCVA from Baseline	The mean changes of BCVA scores on the ETDRS chart at different timepoints after LX111 treatment compared with baseline.	12 weeks, 36 weeks, 52 weeks
Mean changes in Central Subfield Thickness (CST) from Baseline	The mean changes of CST at different timepoints after LX111 treatment compared with baseline.	12 weeks, 36 weeks, 52 weeks
The percentage of participants who received anti-VEGF supplemental injection within 52 weeks after LX111 treatment	The proportion of participants who received anti-VEGF supplemental injection within 52 weeks after LX111 treatment	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.	To evaluate the effect of LX111 on DR (ETDRS-DRSS) over time.	52 weeks

Collaborators and Investigators

• Sponsor: Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
• Collaborators: Innostellar Biotherapeutics Co.,Ltd

Study record dates

Study Major Dates

• Study Start (Estimated): March 1, 2026
• Primary Completion (Estimated): September 1, 2027
• Study Completion (Estimated): September 1, 2031

Study Registration Dates

• First Submitted: January 15, 2026
• First Submitted That Met QC Criteria: January 15, 2026
• First Posted (Actual): January 23, 2026

Study Record Updates

• Last Update Posted (Actual): March 10, 2026
• Last Update Submitted That Met QC Criteria: March 9, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Other Study ID Numbers: LX111-01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No