NG101 AAV Gene Therapy in Subjects With Wet Age-Related Macular Degeneration

NCT05984927 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: NG101WA-01
• Study Type: interventional
• Target: 20
• Locations: 2 countries
• Sites: 5

Brief Summary

This study will evaluate the safety, tolerability, and preliminary efficacy of NG101 AAV gene therapy administered by subretinal injections into a single selected eye as a single selected dose for patients with wet age-related macular degeneration (wAMD).

Conditions

Age-Related Macular Degeneration

Keywords

Wet macular degeneration; Neovascular Age-Related Macular Degeneration; Macular Degeneration; Choroidal Neovascularization; Retinal Degeneration; Eye Diseases; Retinal Diseases; Angiogenesis Inhibitors; Angiogenesis Modulating Agents; Growth Substances; Growth Inhibitors; Antineoplastic Agents; Gene Therapy; Anti-vascular endothelial growth factor therapy; Anti-VEGF therapy

Outcome Measures

Primary Outcomes (1)

• Adverse Events

  Incidence and severity of ocular and non-ocular adverse events (AEs) for each cohort

  From Day 0 (pre-treatment) through Week 24

Secondary Outcomes (9)

• Ocular and Non -Ocular Adverse Events after week 24

  From Day 0 (pre-treatment) and through Weeks 52, 104, 156, 208 and 260

• Systemic Immunogenic Response

  From Day -14 to Weeks 4, 8, 12, 24, 52, and 104

• Systemic Immunogenic Response

  From Day -14 to Weeks 4, 12, and 104

• Systemic Immunogenic Response

  From Day -14 to Day 7, and Weeks 2, 4, and week 12.

• Systemic Immunogenic Response

  From Day 0 (pre-treatment) to Weeks 24, 52, and 104

Study Arms (3)

NG101 Gene Therapy Group 1

EXPERIMENTAL

Single subretinal injection of 1x10\^9 vector genomes of NG101 AAV gene therapy

Genetic: NG101 AAV gene therapy

NG101 Gene Therapy Group 2

EXPERIMENTAL

Single subretinal injection of 3x10\^9 vector genomes of NG101 AAV gene therapy

Genetic: NG101 AAV gene therapy

NG101 Gene Therapy Group 3

EXPERIMENTAL

Single subretinal injection of 8x10\^9 vector genomes of NG101 AAV gene therapy

Genetic: NG101 AAV gene therapy

Interventions

NG101 AAV gene therapy GENETIC

Sub retinal injection of NG101 (a non-replicating adeno-associated virus serotype 8 (AAV8) vector

Also known as: NG101

NG101 Gene Therapy Group 1; NG101 Gene Therapy Group 2; NG101 Gene Therapy Group 3

Eligibility Criteria

• Age: 50 Years - 89 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects ≥ 50 and ≤ 89 years of age with a diagnosis of active subfoveal CNV secondary to wAMD in the Study Eye
• BCVA between 20/40 and 20/400 in the Study Eye, with BCVA decrement primarily attributable to wAMD
• Administration of at least 3 anti-VEGF (vascular endothelial growth factor) injections in the study eye in the past 6 months for clinically active wAMD, the most recent of which was within 1 month prior to Screening Day -14 (Visit 1).
• Must be pseudo phakic (status post cataract surgery) in the Study Eye
• Female and Male reproductive status: Female subjects must be either: (1) of non-childbearing potential; or (2) of childbearing potential and using an acceptable method of birth control with a negative pregnancy test. Male subjects agree to refrain from sperm donations and practice contraception to avoid any pregnancy for 3 months after administration of NG101.
• Normal blood pressure (BP) and heart rate (HR), or near normal BP and HR not considered clinically significant (NCS) by the Investigator at the Screening Day -14 (Visit 1) and Day -7 (Visit 2)
• Willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to the performance of any study-related procedures
• Willingness and ability to comply with schedule for follow-up visits and postoperative evaluations

You may not qualify if:

• CNV or macular edema in the Study Eye secondary to any causes other than AMD
• Any condition preventing visual acuity improvement in the Study Eye, e.g., fibrosis, atrophy, or retinal pigment epithelial tear involving the center of the macula
• Any ophthalmic condition that precludes adequate ophthalmic examination or requires treatment
• Retinal detachment or history of retinal detachment in the Study Eye
• Active uncontrolled glaucoma with intraocular pressure (IOP) ≥ 30 mmHg despite treatment with more than 2 glaucoma medications, advanced glaucoma with cup-to-disc ratio of ≥ 0.9, visual field defects secondary to glaucoma that involve the macula, and/or optic atrophy from glaucoma
• History of intravitreal therapy in the Study Eye, such as intravitreal steroid injection or an investigational product, other than anti-VEGF therapy, in the 6 months prior to Screening
• History of vitrectomy, trabeculectomy, glaucoma tube-shunt procedure, minimally invasive glaucoma surgery (MIGS) device, or other filtration surgery in the Study Eye
• Photodynamic therapy or retinal laser for the treatment of wAMD within the last 6 months
• Any prior therapeutic radiation in the region of the Study Eye such as whole brain radiation, proton beam radiation, gamma knife treatment, or plaque brachytherapy
• Any previous intraocular or refractive surgery on the Study Eye within 6 months
• Any previous gene therapy in the Study Eye
• Presence of an ocular implant in the Study Eye at Screening, excluding intraocular lens and custom flex iris prosthesis
• Any diabetic retinopathy or other retinal vascular disease including retinal vein occlusion, retinal artery occlusion, retinal arterial macro aneurysm, ocular ischemic syndrome, retinal vasculitis, vitritis, posterior uveitis
• Medically uncontrolled diabetes, defined as HbA1C \> 8.0
• History of ocular melanoma

Sponsors & Collaborators

• Elisigen, Inc.: lead
• ORA, Inc.: collaborator

Study Sites (5)

Cincinnati Eye Institute

Cincinnati, Ohio, 45242, United States

Location

Retina Consultants of Texas

Bellaire, Texas, 77401, United States

Location

Vancouver Coastal Health Research Institute

Vancouver, British Columbia, V5Z 3N9, Canada

Location

Sunnybrook Ophthalmology and Vision Services

Toronto, Ontario, M4N 3M5, Canada

Location

Vitreous Retina Macula Specialists of Toronto

Toronto, Ontario, M8X 2X3, Canada

Location

MeSH Terms

Conditions

Macular Degeneration; Wet Macular Degeneration; Choroidal Neovascularization; Retinal Degeneration; Eye Diseases; Retinal Diseases

Condition Hierarchy (Ancestors)

Choroid Diseases; Uveal Diseases; Neovascularization, Pathologic; Metaplasia; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Eye Diseases, Hereditary

Study Officials

• Christopher D Riemann, MD

  Neuracle Genetics, Inc. Medical Director

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Dose Escalation Study with 3 dose cohorts

Study Record Dates

• First Submitted: July 24, 2023
• First Posted: August 9, 2023
• Study Start: September 8, 2023
• Primary Completion: January 1, 2026
• Study Completion (Estimated): January 1, 2030
• Last Updated: January 20, 2026

Record last verified: 2026-01

Locations

US (2); CA (3)