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

Study Overview
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Brief Summary
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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.

Official Title
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A Multicenter, Open-label, Dose Escalation, Phase 1 Clinical Study and Randomized, Double-masked, Controlled, Dose Expansion Phase 2 Clinical Study to Evaluate the Safety, Preliminary Efficacy, Immunogenicity, and Pharmacokinetics of SKG0106 Intraocular Solution in Patients With Neovascular Age-related Macular Degeneration (nAMD)

Conditions
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Neovascular Age-related Macular Degeneration

Intervention / Treatment
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* Genetic: SKG0106


Participation Criteria
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Eligibility Criteria
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Inclusion criteria: 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; Aged ≥ 50 years at screening; Study Eye: Diagnosis of nAMD as determined by the PI; Active CNV lesions secondary to age-related macular degeneration (AMD); Subjects must have been responsive to anti-VEGF therapy as assessed by the PI prior to study treatment. Exclusion Criteria: 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; Retinal pigment epithelial tear in the study eye at screening; Current vitreous hemorrhage in the study eye or history of vitreous hemorrhage within 4 weeks prior to baseline; Any condition that, in the opinion of the investigator, may limit visual acuity improvement in the study eye; History of retinal detachment or active retinal detachment in the study eye; Any prior gene therapy.

Ages Eligible for Study
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Minimum Age: 50 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

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


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Phase I: Low dose<br>SKG0106 One-Time Intraocular Injection Dose Level 1 | Genetic: SKG0106<br>* SKG0106 is a recombinant adeno-associated virus (AAV) vector-based in vivo gene therapeutic product<br>|
| Experimental: Phase I: Medium dose<br>SKG0106 One-Time Intraocular Injection Dose Level 2 | Genetic: SKG0106<br>* SKG0106 is a recombinant adeno-associated virus (AAV) vector-based in vivo gene therapeutic product<br>|
| Experimental: Phase I: High dose<br>SKG0106 One-Time Intraocular Injection Dose Level 3 | Genetic: SKG0106<br>* SKG0106 is a recombinant adeno-associated virus (AAV) vector-based in vivo gene therapeutic product<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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AMD, wet AMD, wAMD, nAMD