Diabetic Macular Edema (DME)Early Phase 1NCT07362927
Safety and Efficacy Evaluation of LX111 Gene Therapy in DME Patients
A Early Phase 1 study testing LX111 Injection in people with Diabetic Macular Edema (DME), run by Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine at 2 sites in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Diabetic Macular Edema (DME)
- What is being tested?
- LX111 Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 32 participants
- Where
- 2 sitesChina
- Study dates
- Mar 2026to Sep 2031
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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;
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
China2 sites, 2 recruiting
- Recruiting
Shanghai General Hospital
Shanghai
Li Su, +86 02136126254, li.su@shgh.cn
Li Su
- Not yet recruiting
Zhongshan Hospital
Shanghai
Yuanzhi Yuan, +86 02164041990, Yuan.yuanzhi@zs-hospital.sh.cn
Yuanzhi Yuan
About this study
Official title: 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.
Read the 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.
- Conditions
- Diabetic Macular Edema (DME)
- Being tested
- LX111 Injection
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LX111 InjectionGenetic
LX111 is an rAAV gene therapy vector carrying a coding sequence for VEGF-trap.
| Study group | Role | What they receive |
|---|---|---|
| LX111 Dose Escalation up to 2 dose levels | Experimental | Qualified participants will receive a single unilateral intravitreal injection of LX111 at Day 0. Receives: Genetic: LX111 Injection |
| LX111 Dose Expansion Dose 1 | Experimental | Qualified participants will receive a single unilateral intravitreal injection of LX111 at Day 0. Receives: Genetic: LX111 Injection |
| LX111 Dose Expansion Dose 2 | Experimental | Qualified participants will receive a single unilateral intravitreal injection of LX111 at Day 0. Receives: Genetic: LX111 Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 52 weeks
Dose Limiting Toxicity
The incidence of DLT in each dose group.
Measured: 4 weeks
Secondary outcomes (4)
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.
Measured: 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.
Measured: 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
Measured: 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.
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jan 2026
- Study startMar 2026 (estimated)
- Primary completionSep 2027 (estimated)
- Study completionSep 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Diabetic Macular Edema (DME)Studies of the same condition, open and under way.
- More trials run by Shanghai General Hospital, Shanghai Jiao Tong University School of MedicineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 32 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Innostellar Biotherapeutics Co.,Ltd
- Responsible party
- Principal investigator, Xiaodong Sun, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07362927
- Sponsor's protocol ID
- LX111-01
- Status last verified
- Mar 2026
- First submitted
- 15 Jan 2026
- Last updated
- 10 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Exploratory Clinical Study Evaluating LX111 Gene Therapy in Patients With Diabetic Macular Edema (DME)
- ClinicalTrials.gov ID
- NCT07362927
- Acronym / sponsor ID
- LX111-01
- Conditions
- Diabetic Macular Edema (DME)
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 32 participants (estimated)
- Lead sponsor
- Shanghai General Hospital, Shanghai Jiao Tong University School of MedicineUniversity, hospital or other
- Collaborators
- Innostellar Biotherapeutics Co.,Ltd
- Study start
- Mar 2026 (estimated)
- Primary completion
- Sep 2027 (estimated)
- Study completion
- Sep 2031 (estimated)
- First posted
- 23 Jan 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| LX111 Dose Escalation up to 2 dose levels | Experimental | Genetic: LX111 InjectionQualified participants will receive a single unilateral intravitreal injection of LX111 at Day 0. |
| LX111 Dose Expansion Dose 1 | Experimental | Genetic: LX111 InjectionQualified participants will receive a single unilateral intravitreal injection of LX111 at Day 0. |
| LX111 Dose Expansion Dose 2 | Experimental | Genetic: LX111 InjectionQualified participants will receive a single unilateral intravitreal injection of LX111 at Day 0. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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;
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LX111 InjectionLX111 is an rAAV gene therapy vector carrying a coding sequence for VEGF-trap. | Genetic | LX111 Dose Escalation up to 2 dose levels, LX111 Dose Expansion Dose 1, LX111 Dose Expansion Dose 2 |
Primary outcome
Adverse events and serious adverse events in the eyes and throughout the body within 364 days after LX111 treatment
Time frame: 52 weeks
Incidence of adverse events and serious adverse events within 52 weeks of LX111 intravitreal injection in each dose group.
Dose Limiting Toxicity
Time frame: 4 weeks
The incidence of DLT in each dose group.
4 secondary outcomes
Mean changes in BCVA from Baseline
Time frame: 12 weeks, 36 weeks, 52 weeks
Mean changes in Central Subfield Thickness (CST) from Baseline
Time frame: 12 weeks, 36 weeks, 52 weeks
The percentage of participants who received anti-VEGF supplemental injection within 52 weeks after LX111 treatment
Time frame: 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.
Time frame: 52 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Mar 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing LX111 Injection in people with Diabetic Macular Edema (DME), run by Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine at 2 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 10 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Li Su (+86 02136126254, li.su@shgh.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse events and serious adverse events in the eyes and throughout the body within 364 days after LX111 treatment (time frame: 52 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Mar 2026.