A Clinical Study Evaluating the Safety and Efficacy of SKG0106 in Patients With Neovascular Age-related Macular Degeneration (nAMD)

An Open-label, Dose-escalation Study to Evaluate the Safety, Preliminary Efficacy, Immunogenicity and Pharmacokinetic Characteristics of SKG0106 Intraocular Solution After Single Intravitreal Injection in Chinese Patients With Neovascular (Wet) Age-related Macular Degeneration

This is a clinical study to evaluate the safety, preliminary efficacy, immunogenicity of SKG0106 in subjects with nAMD.

Study Overview

• Status: Recruiting
• Conditions: Neovascular Age-related Macular Degeneration
• Intervention / Treatment: Genetic: SKG0106

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

Contacts and Locations

• Study Contact: Name: Youchen Chen; Phone Number: 13801025972; Email: chenyouxinpumch@163.com

Study Locations

• China
  • Beijing, China
    • Recruiting
    • Peking Union Medical College Hospital
    • Contact: Youxin Chen
  • Shanghai, China
    • Recruiting
    • Eye & ENT Hospital of Fudan University
    • Contact: Gezhi Xu
  • Shanghai, China
    • Recruiting
    • Xin Hua Hospital Affiliated to Shanghai Jiao Tong University School of MedicineXin Hua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Voluntary and able to sign a dated ICF prior to any study-related procedures and able to complete the study as required by the protocol;

2. Aged ≥ 50 years at screening;

   Study Eye:

3. Diagnosis of nAMD as determined by the PI;
4. Active CNV lesions secondary to age-related macular degeneration (AMD);
5. Subjects must have been responsive to anti-VEGF therapy as assessed by the PI prior to study treatment

Exclusion Criteria:

1. Any active intraocular or periocular infection or active intraocular inflammation (e.g., infectious conjunctivitis, keratitis, scleritis, endophthalmitis, infectious blepharitis, uveitis) in the study eye at baseline;
2. Retinal pigment epithelial tear in the study eye at screening;
3. Current vitreous hemorrhage in the study eye or history of vitreous hemorrhage within 4 weeks prior to baseline;
4. Any condition that, in the opinion of the investigator, may limit visual acuity improvement in the study eye;
5. History of retinal detachment or active retinal detachment in the study eye;
6. Any prior gene therapy.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Dose Level 1; SKG0106 One-Time Intraocular Injection Dose Level 1	Genetic: SKG0106; SKG0106 is a recombinant adeno-associated virus (AAV) vector-based in vivo gene therapeutic product
Experimental: Dose Level 2; SKG0106 One-Time Intraocular Injection Dose Level 2	Genetic: SKG0106; SKG0106 is a recombinant adeno-associated virus (AAV) vector-based in vivo gene therapeutic product
Experimental: Dose Level 3; SKG0106 One-Time Intraocular Injection Dose Level 3	Genetic: SKG0106; SKG0106 is a recombinant adeno-associated virus (AAV) vector-based in vivo gene therapeutic product

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Characteristics of dose limiting toxicities (DLTs)	4 weeks
Type, severity, and incidence of ocular and systemic adverse events (AEs)	48 weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
Mean change from baseline in patient-reported outcome (VFQ-25) scale score at each visit Both the total scale and subscale scores of the VFQ-25 is ranging from 0 to 100, and higher scores mean a better outcome.	48 weeks
Mean change from baseline in best corrected visual acuity (BCVA) at each visit BCVA of the study eye is examined by the Early Treatment of Diabetic Retinopathy Study (ETDRS) testing at screening and baseline.	24 weeks
Mean change from baseline in central subfield thickness (CST) at each visit Spectral domain optical coherence tomography (SD-OCT) will be performed to measure CST at screening and at each visit (prior to study drug injection, if available).	24 weeks

Collaborators and Investigators

• Sponsor: Youxin Chen
• Investigators: Principal Investigator: Youchen Chen, Peking Union Medical College Hospital

Study record dates

Study Major Dates

• Study Start (Actual): May 5, 2023
• Primary Completion (Estimated): May 30, 2025
• Study Completion (Estimated): May 30, 2025

Study Registration Dates

• First Submitted: January 9, 2024
• First Submitted That Met QC Criteria: January 9, 2024
• First Posted (Actual): January 19, 2024

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: January 6, 2025
• Last Verified: January 1, 2025

More Information

Terms related to this study

• Keywords: AMD; wet AMD; wAMD; nAMD
• Additional Relevant MeSH Terms: Eye Diseases; Retinal Diseases; Retinal Degeneration; Macular Degeneration; Wet Macular Degeneration
• Other Study ID Numbers: SKG0106-001

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