Safety and Efficacy of LX102 Gene Therapy in Patients With Neovascular Age-related Macular Degeneration (nAMD) (VENUS)

A Phase 2, Randomized Controlled, Open-Label Study to Establish the Safety and Efficacy of LX102 in Patients With Neovascular Age-related Macular Degeneration (nAMD) (VENUS)

The goal of this study is to evaluate the overall 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; Biological: Aflibercept intravitreal injection

Detailed Description

In this Phase 2, multi-center, randomized controlled study, subjects will be randomized to receive one of the two dose levels of LX102 (n=20 for each dose level), or aflibercept (n=10).

Safety, tolerability, and efficacy will be evaluated for a period of approximately 1 year from baseline.

• Study Type: Interventional
• Enrollment (Estimated): 50
• Phase: Phase 2

Contacts and Locations

Study Locations

• China
  • Beijing, China
    • Peking Union Medical College Hospital
  • Chengdu, China
    • West China Hospital of Sichuan University
  • Guangzhou, China
    • Guangzhou Aier Eye Hospital
  • Guangzhou, China
    • Zhongshan Ophthalmic Center of Sun Yat-Sen University
  • Shanghai, China
    • Shanghai General Hospital
  • Shanghai, China
    • Shanghai Eye and ENT Hospital
  • Taiyuan, China
    • Shanxi Eye Hospital
  • Tianjin, China
    • Tianjin Medical University Eye Hospital
  • Xuzhou, China
    • Xuzhou No.1 People's 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, and ≤ 89.
3. Diagnosis of active CNV secondary to neovascular AMD.
4. BCVA ETDRS letters between 19 and 73.
5. 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. Absence of RPE tear at Screening.
4. Acute coronary syndrome, myocardial infarction or coronary artery revascularization, CVA, TIA in the last 6 months.
5. Uncontrolled hypertension defined as average SBP ≥160 mmHg or an average DBP ≥100 mmHg.
6. Uncontrolled diabetes defined as HbA1c >8.0%.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: LX102 Dose 1; LX102 will be administered at the assigned dose level as a single dose subretinal injection on Day 0	Genetic: LX102 subretinal injection; LX102: AAV-based gene therapy comprised of codon-optimized sequence encoding VEGF-trap
Experimental: LX102 Dose 2; LX102 will be administered at the assigned dose level as a single dose subretinal injection on Day 0	Genetic: LX102 subretinal injection; LX102: AAV-based gene therapy comprised of codon-optimized sequence encoding VEGF-trap
Active Comparator: Control group; Aflibercept at a fixed regimen will be administered.	Biological: Aflibercept intravitreal injection; Commercially available Active Comparator

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Mean change from baseline in Best Corrected Visual Acuity (BCVA)	BCVA measured by ETDRS	36 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Mean change from baseline in Best Corrected Visual Acuity (BCVA)	BCVA measured by ETDRS	52 weeks
Mean change in Central Subfield Thickness (CST) from Baseline	CST measured by spectral domain optical coherence tomography (SD-OCT)	36 weeks, 52 weeks
Durability of LX102 treatment	Mean time from LX102 administration to anti-VEGF rescue injection for the first time, percentage of participants requiring anti-VEGF rescue injection, and mean number of anti-VEGF rescue injections through 52 weeks following LX102 administration.	52 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	36 weeks, 52 weeks

Collaborators and Investigators

• Sponsor: Innostellar Biotherapeutics Co.,Ltd

Study record dates

Study Major Dates

• Study Start (Actual): January 24, 2024
• Primary Completion (Actual): June 24, 2025
• Study Completion (Estimated): October 1, 2029

Study Registration Dates

• First Submitted: December 25, 2023
• First Submitted That Met QC Criteria: December 25, 2023
• First Posted (Actual): January 9, 2024

Study Record Updates

• Last Update Posted (Actual): March 6, 2026
• Last Update Submitted That Met QC Criteria: March 4, 2026
• Last Verified: March 1, 2026

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; Antineoplastic Agents; Physiological Effects of Drugs; Angiogenesis Inhibitors; Angiogenesis Modulating Agents; Growth Substances; Growth Inhibitors; aflibercept
• Other Study ID Numbers: INNOSTELLAR-LX102A01-2

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