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NCT Number: NCT07053358

Safety and Efficacy Evaluation of LX111 Gene Therapy in nAMD Patients

The goal of this study is to evaluate the safety and efficacy of LX111 treatment of nAMD. This study will enroll subjects aged ≥ 50 vears old to receive a single unilateral intravitreal (lVT) injection of LX111 to evaluate its safety and efficacy.

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Key information

Age range

50 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Early Phase 1

Primary location

Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

Shanghai, 200080, China

Location status: Recruiting

Location contact

Yuanyuan Gong

CONTACT

[email protected]

+86 02136126254

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Willing to sign the informed consent, and willing to attend follow-up visits.
  • Age ≥ 50
  • Diagnosis of active CNV secondary to neovascular AMD
  • BCVA ETDRS letters between 5 and 63
  • Subjects must have received a minimum of 2 injections within 6 months prior to screening and demonstrated a meaningful response to anti-VEGF therapy
  • 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

  • CNV or macular edema in the study eye secondary to diseases other than nAMD
  • Retinal detachment, uveitis, uncontrolled glaucoma in the study eye, or any condition preventing visual acuity improvement
  • Acute coronary syndrome, myocardial infarction or coronary artery revascularization, CVA, TIA in the last 6 months
  • Uncontrolled hypertension defined as average SBP ≥160 mmHg or an average DBP ≥100 mmHg
  • Uncontrolled diabetes defined as HbA1c >8.0% within 28 days prior to screening

Treatment and study plan

LX111 Injection

Genetic

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

Primary outcomes

  1. 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.

  2. Dose Limiting Toxicity

    Time frame: 4 weeks

    The incidence of DLT in each dose group.

Secondary outcomes

  1. Mean changes in BCVA from Baseline

    Time frame: 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.

  2. Mean changes in Central Subfield Thickness (CST) from Baseline

    Time frame: 12 weeks, 36 weeks, 52 weeks

    The mean changes of CST at different timepoints after LX111 treatment compared with baseline.

  3. The percentage of subjects who received anti-VEGF supplemental injection within 52 weeks after LX111 treatment

    Time frame: 52 weeks

    The proportion of subjects who received anti-VEGF supplemental injection within 52 weeks after LX111 treatment

Sponsors and collaborators

Lead sponsor

Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

Other

Collaborators

  • Innostellar Biotherapeutics Co.,Ltd

Registry information

Official study title

An Exploratory Clinical Study Evaluating LX111 Gene Therapy in Patients With Neovascular Age-related Macular Degeneration (nAMD)

Important dates

Study start
2025
Primary completion
2026
Study completion
2029
First posted
Jul 8, 2025
Registry last updated
Mar 10, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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