Phase I/II Study of SKG0106 Intravitreal Injection in Patients With Neovascular Age-related Macular Degeneration (nAMD)

Phase I/II Study to Evaluate the Safety and Preliminary Efficacy of SKG0106 Intravitreal Injection in Patients With Neovascular Age-related Macular Degeneration (nAMD)

This is a phase 1/2 clinical study to evaluate the safety, preliminary efficacy, immunogenicity, and pharmacokinetic (PK) characteristics of SKG0106 in subjects with nAMD. Based on results from the phase 1 dose escalation study, the phase 2 expansion study will be conducted.

Study Overview

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

• Study Type: Interventional
• Enrollment (Estimated): 68
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Yongqin Wang; Phone Number: +86 18616737445; Email: yongqin.wang@skytx.com

Study Locations

• China
  • Beijing, China
    • Recruiting
    • Beijing Hospital
  • Beijing, China
    • Recruiting
    • Peking Union Medical College Hospital
  • Anhui
    • Hefei, Anhui, China
      • Recruiting
      • The Second Hospital of Anhui Medical University
  • Zhejiang
    • Hangzhou, Zhejiang, China
      • Recruiting
      • Zhejiang Provincial People's Hospital
    • Wenzhou, Zhejiang, China
      • Recruiting
      • Eye Hospital, WMU (Zhejiang Eye Hospital)
• United States
  • Florida
    • Saint Petersburg, Florida, United States, 33711
      • Not yet recruiting
      • Retina Vitreous Associates of Florida - Saint Petersburg
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Recruiting
      • Ophthalmic Consultants of Boston
  • Texas
    • Katy, Texas, United States, 77494
      • Recruiting
      • Retina Consultants of Texas
      • Contact: Marie Chin
      • Principal Investigator: Charles C Wykoff, MD
  • Virginia
    • Norfolk, Virginia, United States, 23502
      • Recruiting
      • Wagner Kapoor Research institute
      • Contact: Chris Riebling
      • Principal Investigator: Alan Wagner, MD

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: Phase I: Low dose; 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: Phase I: Medium dose; 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: Phase I: High dose; 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)	52 Weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	52 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).	52 Weeks
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.	52 Weeks

Collaborators and Investigators

• Sponsor: Skyline Therapeutics (US) Inc.

Study record dates

Study Major Dates

• Study Start (Actual): February 23, 2024
• Primary Completion (Estimated): January 1, 2026
• Study Completion (Estimated): January 30, 2026

Study Registration Dates

• First Submitted: August 3, 2023
• First Submitted That Met QC Criteria: August 3, 2023
• First Posted (Actual): August 14, 2023

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: January 8, 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: SKG0106-101

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: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No