LX102 in Patients With Neovascular Age-Related Macular Degeneration (nAMD)

A Dose-Escalation Study of LX102 Gene Therapy for Neovascular Age-Related Macular Degeneration (nAMD)

The goal of this study is to evaluate the safety and efficacy of LX102 gene therapy for nAMD.

Study Overview

• Status: Active, not recruiting
• Conditions: Neovascular Age-Related Macular Degeneration
• Intervention / Treatment: Genetic: LX102 subretinal injection

Detailed Description

This study will enroll subjects aged ≥ 50 years old to receive a single unilateral subretinal injection of LX102 to evaluate its safety and efficacy.

• Study Type: Interventional
• Enrollment (Actual): 12
• Phase: Phase 1

Contacts and Locations

Study Locations

• China
  • Shanghai, China
    • Shanghai General Hospital
  • Anhui
    • Hefei, Anhui, China
      • Anhui Provincial Hospital
  • Zhejiang
    • Hangzhou, Zhejiang, China
      • Zhejiang University Eye Hospital

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 ≥ 50
3. Diagnosis of active CNV secondary to neovascular AMD
4. BCVA ETDRS letters between 5 and 63
5. Subjects must have received a minimum of 2 injections within 6 months prior to screening
6. Demonstrated a meaningful response to anti-VEGF therapy

Exclusion Criteria:

1. CNV or macular edema in the study eye secondary to diseases other than nAMD
2. Retinal detachment, uveitis, uncontrolled glaucoma in the study eye, or any condition preventing visual acuity improvement
3. Acute coronary syndrome, myocardial infarction or coronary artery revascularization, CVA, TIA in the last 6 months
4. Uncontrolled hypertension defined as average SBP ≥160 mmHg or an average DBP ≥100 mmHg
5. Uncontrolled diabetes defined as HbA1c >7.5%

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: LX102 Dose 1; Low dose of LX102	Genetic: LX102 subretinal injection; LX102: AAV-based gene therapy comprised of codon-optimized sequence encoding VEGF-trap
Experimental: LX102 Dose 2; Mid dose of LX102	Genetic: LX102 subretinal injection; LX102: AAV-based gene therapy comprised of codon-optimized sequence encoding VEGF-trap
Experimental: LX102 Dose 3; High dose of LX102	Genetic: LX102 subretinal injection; LX102: AAV-based gene therapy comprised of codon-optimized sequence encoding VEGF-trap

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of dose-limiting toxicity (DLT)	Incidence of dose-limiting toxicity (DLT) following LX102 subretinal injection at different doses	4 weeks
Incidence of adverse events (AEs) and serious adverse events (SAEs)	Incidence of ocular and systemic adverse events (AEs) and serious adverse events (SAEs) following LX102 subretinal injection	52 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Mean change in BCVA from Baseline	BCVA measured by ETDRS	12 weeks, 36 weeks 52 weeks
Mean change in Central Subfield Thickness (CST) from Baseline	CST measured by spectral domain optical coherence tomography (SD-OCT)	12 weeks, 36 weeks 52 weeks
Mean time from LX102 administration to anti-VEGF rescue injection for the first time		52 weeks
Percentage of participants requiring anti-VEGF rescue injection		52 weeks
Mean number of anti-VEGF rescue injections		52 weeks

Collaborators and Investigators

• Sponsor: Innostellar Biotherapeutics Co.,Ltd

Study record dates

Study Major Dates

• Study Start (Actual): February 27, 2023
• Primary Completion (Actual): December 26, 2024
• Study Completion (Estimated): January 1, 2029

Study Registration Dates

• First Submitted: December 25, 2023
• First Submitted That Met QC Criteria: January 8, 2024
• First Posted (Actual): January 10, 2024

Study Record Updates

• Last Update Posted (Estimated): July 1, 2025
• Last Update Submitted That Met QC Criteria: June 26, 2025
• Last Verified: June 1, 2025

More Information

Terms related to this study

• Keywords: AMD; Neovascular AMD; Age-related macular degeneration; Neovascular age-related macular degeneration
• Additional Relevant MeSH Terms: Eye Diseases; Retinal Diseases; Retinal Degeneration; Macular Degeneration; Wet Macular Degeneration
• Other Study ID Numbers: INNOSTELLAR-LX102A01-1

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
• product manufactured in and exported from the U.S.: No