Diabetic Macular Edema (DME)
Conditions
Brief summary
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.
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.
Interventions
GENETICLX111 Injection
LX111 is an rAAV gene therapy vector carrying a coding sequence for VEGF-trap.
Sponsors
Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
Innostellar Biotherapeutics Co.,Ltd
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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;
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Adverse events and serious adverse events in the eyes and throughout the body within 364 days after LX111 treatment | 52 weeks | Incidence of adverse events and serious adverse events within 52 weeks of LX111 intravitreal injection in each dose group. |
| Dose Limiting Toxicity | 4 weeks | The incidence of DLT in each dose group. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Mean changes in BCVA from Baseline | 12 weeks, 36 weeks, 52 weeks | The mean changes of BCVA scores on the ETDRS chart at different timepoints after LX111 treatment compared with baseline. |
| Mean changes in Central Subfield Thickness (CST) from Baseline | 12 weeks, 36 weeks, 52 weeks | The mean changes of CST at different timepoints after LX111 treatment compared with baseline. |
| The percentage of participants who received anti-VEGF supplemental injection within 52 weeks after LX111 treatment | 52 weeks | The proportion of participants who received anti-VEGF supplemental injection within 52 weeks after LX111 treatment |
| Proportion of participants achieving an improvement or worsening in DR in the study eye per the ETDRS-DRSS on ultra-wide field fundus photography. | 52 weeks | To evaluate the effect of LX111 on DR (ETDRS-DRSS) over time. |
Countries
China
Contacts
CONTACTLi Su
Outcome results
None listed