LX111 gene therapy for adults with diabetic macular edema.

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

This treatment will try a single eye injection of LX111 gene therapy in adults with diabetic macular edema to see if it is safe and reduces retinal swelling and improves vision.

Quick facts

• Phase: Early Phase 1
• Study type: Interventional
• Enrollment: 32 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine (Academic / other)
• Locations: 2 sites (Shanghai and 1 other locations)
• Trial ID: NCT07362927 on ClinicalTrials.gov

What this trial studies

This is a prospective, multicenter, dose‑ranging early‑phase trial that delivers a single unilateral intravitreal injection of LX111 to adults with center‑involving DME. The study is conducted in two parts: dose confirmation followed by dose expansion to identify a safe dose and then enroll more participants at that dose. Eligible participants must be ≥18, have central subfield thickness ≥300 μm, BCVA 19–73 ETDRS letters, and a prior meaningful response to anti‑VEGF within 12 months. Primary outcomes emphasize safety and tolerability with secondary measures of retinal thickness and visual acuity during follow‑up.

Who should consider this trial

Good fit: Ideal candidates are adults with center‑involving DME, CST ≥300 μm, BCVA between 19 and 73 ETDRS letters, and a documented meaningful response to anti‑VEGF within the prior 12 months.

Not a fit: Patients with active proliferative diabetic retinopathy, iris neovascularization, recent retinal laser or recent intravitreal steroid implant, prior gene therapy in the study eye, or those outside the specified visual acuity and thickness criteria are unlikely to benefit from this protocol.

Why it matters

Potential benefit: If successful, LX111 could offer a longer‑lasting one‑time treatment that reduces macular swelling and decreases the need for frequent anti‑VEGF injections.

How similar studies have performed: Gene therapy for DME is a novel approach with limited prior data, although ocular gene therapies have succeeded for some inherited retinal diseases and long‑acting anti‑VEGF gene strategies are being explored in early trials.

Eligibility criteria

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;

Where this trial is running

Shanghai and 1 other locations

• Shanghai General Hospital — Shanghai, China (Recruiting)
• Zhongshan Hospital — Shanghai, China (Not_yet_recruiting)

Study contacts

• Study coordinator: Li Su

Conditions Diabetic Macular Edema