Phase I/II Study to Evaluate the Safety, Preliminary Efficacy, Immunogenicity, and Pharmacokinetics of SKG0106 Intraocular Solution in Patients With Neovascular Age-related Macular Degeneration (nAMD)

• Sponsor: Skyline Therapeutics (US) Inc. (Industry)
• Overall Status: Not yet recruiting
• CT.gov ID: NCT05986864
• Collaborator: (none)
• Enrollment: 68
• Locations: 2
• Arms: 3
• Anticipated Duration (Months): 71
• Patients Per Site: 34
• Patients Per Site Per Month: 0.5

Study Details

Study Description

Brief Summary

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.

Condition or Disease	Intervention/Treatment	Phase
Neovascular Age-related Macular Degeneration	Genetic: SKG0106	Phase 1/Phase 2

Study Design

• Study Type: Interventional
• Anticipated Enrollment: 68 participants
• Allocation: Non-Randomized
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: 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)
• Anticipated Study Start Date: Oct 30, 2023
• Anticipated Primary Completion Date: Nov 30, 2025
• Anticipated Study Completion Date: Sep 30, 2029

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures

1. Safety assessments [52 Weeks]

   Safety assessments consist of collecting all AE, SAEs, AESIs, and clinically significant laboratory abnormalities for vital signs, physical examinations, ECGs, and clinical laboratory tests.

2. Best corrected visual acuity [52 Weeks]

   Change in Best Corrected Visual Acuity

Secondary Outcome Measures

1. Intraocular pressure [52 Weeks]

   The same method of IOP measurement must be used for the same subject from screening through the Week 52 (EOS) visit.

2. Optical coherence tomography [52 Weeks]

   SD-OCT will be performed to measure CSTQuestionnaire-25 (VFQ-25)

3. ICGA [52 Weeks]

   ICG will be performed in the study eye at screening

4. Visual Function Questionnaire-25 [52 Weeks]

   visual function following treatment

Eligibility Criteria

Criteria

• Ages Eligible for Study: 50 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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:

1. Diagnosis of nAMD as determined by the PI;

2. Active CNV lesions secondary to age-related macular degeneration (AMD);

3. Total CNV area (classical and occult) at screening must account for more than 50% of the total lesion area in the study eye (confirmed by CRC at Phase 2 clinical study);

4. Intraretinal and/or subretinal fluid in the fovea of the retina in the study eye, confirmed by CRC at Phase 2 clinical study;

5. BCVA between 63 and 19 letters (inclusive) in the study eye examined using the Early Treatment of Diabetic Retinopathy Study testing at screening and baseline.

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. Results of colorful fundus photography assessment at screening showed fibrotic or geographic atrophic changes in the fovea of retina in the study eye, confirmed by CRC at Phase 2 clinical study;

3. Retinal pigment epithelial tear in the study eye at screening;

4. Current vitreous hemorrhage in the study eye or history of vitreous hemorrhage within 4 weeks prior to baseline;

5. Any condition that, in the opinion of the investigator, may limit visual acuity improvement in the study eye (e.g., history of intraocular disease other than nAMD in the study eye or current evidence of intraocular disease [including retinopathy] that, in the judgment of the investigator, may require medical or surgical intervention to prevent or treat visual acuity lost possibly caused by the condition or may limit the possibility of increased visual acuity following study treatment);

6. History of retinal detachment or active retinal detachment in the study eye;

7. Any prior gene therapy.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Retina Consultants of Texas	Katy	Texas	United States	77494
2	Wagner Kapoor Research Institute	Norfolk	Virginia	United States	23502

Sponsors and Collaborators

• Skyline Therapeutics (US) Inc.

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Skyline Therapeutics (US) Inc.
• ClinicalTrials.gov Identifier: NCT05986864
• Other Study ID Numbers: SKG0106-101
• First Posted: Aug 14, 2023
• Last Update Posted: Aug 17, 2023
• Last Verified: Aug 1, 2023

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• 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
• Keywords provided by Skyline Therapeutics (US) Inc.: AMD; wet AMD; wAMD; nAMD
• Additional relevant MeSH terms: Macular Degeneration; Wet Macular Degeneration

Study Results

No Results Posted as of Aug 17, 2023