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

Phase 2

Active, not recruiting

• Conditions: Neovascular Age-Related Macular Degeneration
• Interventions: Genetic: LX102 subretinal injection; Biological: Aflibercept intravitreal injection

• Registration Number: NCT06196840
• Lead Sponsor: Innostellar Biotherapeutics Co.,Ltd

Brief Summary

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

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 Timeline

• Study Start: 2023-12-21
• Study First Submitted: 2023-12-25
• Study First Submitted QC: 2023-12-25
• Study First Posted: 2024-01-09
• Status Verified: 2025-06
• Last Update Submitted: 2025-06-26
• Primary Completion: 2025-07
• Last Update Posted: 2025-07-01
• Study Completion: 2029-10-01

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 50

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
LX102 Dose 1	LX102 subretinal injection	LX102 will be administered at the assigned dose level as a single dose subretinal injection on Day 0
LX102 Dose 2	LX102 subretinal injection	LX102 will be administered at the assigned dose level as a single dose subretinal injection on Day 0
Control group	Aflibercept intravitreal injection	Aflibercept at a fixed regimen will be administered.

Primary Outcome Measures

Name	Time	Method
Mean change from baseline in Best Corrected Visual Acuity (BCVA)	36 weeks	BCVA measured by ETDRS

Secondary Outcome Measures

Name	Time	Method
Incidence of adverse events (AEs) and serious adverse events (SAEs)	36 weeks, 52 weeks	Incidence of ocular and systemic adverse events (AEs) and serious adverse events (SAEs) following LX102 subretinal injection
Mean change in Central Subfield Thickness (CST) from Baseline	36 weeks, 52 weeks	CST measured by spectral domain optical coherence tomography (SD-OCT)
Durability of LX102 treatment	52 weeks	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.
Mean change from baseline in Best Corrected Visual Acuity (BCVA)	52 weeks	BCVA measured by ETDRS

Trial Locations

Locations (11)

Peking Union Medical College Hospital; 🇨🇳 Beijing, China

West China Hospital of Sichuan University; 🇨🇳 Chengdu, China

Guangzhou Aier Eye Hospital; 🇨🇳 Guangzhou, China

Zhongshan Ophthalmic Center of Sun Yat-Sen University; 🇨🇳 Guangzhou, China

Shanghai Eye and ENT Hospital; 🇨🇳 Shanghai, China

Shanxi Eye Hospital; 🇨🇳 Taiyuan, China

Tianjin Medical University Eye Hospital; 🇨🇳 Tianjin, China

Xuzhou No.1 People's Hospital; 🇨🇳 Xuzhou, China

Anhui Provincial Hospital; 🇨🇳 Hefei, Anhui, China

Zhejiang University Eye Hospital; 🇨🇳 Hangzhou, Zhejiang, China

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