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

Last updated: January 16, 2026
Sponsor: Elisigen, Inc.
Overall Status: Active - Not Recruiting

Phase

1/2

Condition

Aging

Geographic Atrophy

Macular Degeneration

Treatment

NG101 AAV gene therapy

Clinical Study ID

NCT05984927
NG101WA-01
  • Ages 50-89
  • All Genders

Study 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).

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Subjects ≥ 50 and ≤ 89 years of age with a diagnosis of active subfoveal CNVsecondary to wAMD in the Study Eye

  2. BCVA between 20/40 and 20/400 in the Study Eye, with BCVA decrement primarilyattributable to wAMD

  3. 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, themost recent of which was within 1 month prior to Screening Day -14 (Visit 1).

  4. Must be pseudo phakic (status post cataract surgery) in the Study Eye

  5. Female and Male reproductive status: Female subjects must be either: (1) ofnon-childbearing potential; or (2) of childbearing potential and using an acceptablemethod of birth control with a negative pregnancy test. Male subjects agree torefrain from sperm donations and practice contraception to avoid any pregnancy for 3months after administration of NG101.

  6. Normal blood pressure (BP) and heart rate (HR), or near normal BP and HR notconsidered clinically significant (NCS) by the Investigator at the Screening Day -14 (Visit 1) and Day -7 (Visit 2)

  7. Willing and able to provide written, signed informed consent after the nature of thestudy has been explained and prior to the performance of any study-relatedprocedures

  8. Willingness and ability to comply with schedule for follow-up visits andpostoperative evaluations

Exclusion

Exclusion Criteria:

  1. CNV or macular edema in the Study Eye secondary to any causes other than AMD

  2. 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

  3. Any ophthalmic condition that precludes adequate ophthalmic examination or requirestreatment

  4. Retinal detachment or history of retinal detachment in the Study Eye

  5. Active uncontrolled glaucoma with intraocular pressure (IOP) ≥ 30 mmHg despitetreatment with more than 2 glaucoma medications, advanced glaucoma with cup-to-discratio of ≥ 0.9, visual field defects secondary to glaucoma that involve the macula,and/or optic atrophy from glaucoma

  6. History of intravitreal therapy in the Study Eye, such as intravitreal steroidinjection or an investigational product, other than anti-VEGF therapy, in the 6months prior to Screening

  7. History of vitrectomy, trabeculectomy, glaucoma tube-shunt procedure, minimallyinvasive glaucoma surgery (MIGS) device, or other filtration surgery in the StudyEye

  8. Photodynamic therapy or retinal laser for the treatment of wAMD within the last 6months

  9. Any prior therapeutic radiation in the region of the Study Eye such as whole brainradiation, proton beam radiation, gamma knife treatment, or plaque brachytherapy

  10. Any previous intraocular or refractive surgery on the Study Eye within 6 months

  11. Any previous gene therapy in the Study Eye

  12. Presence of an ocular implant in the Study Eye at Screening, excluding intraocularlens and custom flex iris prosthesis

  13. Any diabetic retinopathy or other retinal vascular disease including retinal veinocclusion, retinal artery occlusion, retinal arterial macro aneurysm, ocularischemic syndrome, retinal vasculitis, vitritis, posterior uveitis

  14. Medically uncontrolled diabetes, defined as HbA1C > 8.0

  15. History of ocular melanoma

  16. History of any known inherited retinal disease

  17. Currently taking any anticoagulant therapy, which is deemed medically necessary andcannot be permanently stopped at least 2 weeks prior to NG101 injection, excludingprophylactic low-dose aspirin therapy

  18. Any underlying systemic diseases as unstable or severe cardiovascular,cerebrovascular, dementia or neurodegenerative diseases of a level that preventsadequate evaluation of the subject during the study, active malignancy or currentlyundergoing treatment for active malignancy at Screening or a history of malignancythat precludes completion of this 260 week study, and immunocompromised conditionsand/or need for immunosuppressive therapy

  19. Active hepatitis B or C

  20. History of human immunodeficiency virus (HIV), active tuberculosis, and/or syphilis

  21. Any significant illness that would preclude study compliance and follow-up

  22. Subjects who, in the Investigator's opinion, lack the mental capacity to providewritten informed consent for study participation

Study Design

Total Participants: 20
Treatment Group(s): 1
Primary Treatment: NG101 AAV gene therapy
Phase: 1/2
Study Start date:
September 08, 2023
Estimated Completion Date:
January 31, 2030

Study Description

This is a Phase 1/2a, multicenter, open-label, dose escalation study to evaluate the safety, tolerability, and preliminary efficacy of NG101 AAV gene therapy, administered by subretinal injection in patients with active wAMD symptoms. The study will be conducted at approximately 5 sites in Canada and the USA.

Connect with a study center

  • Vancouver Coastal Health Research Institute

    Vancouver, British Columbia V5Z 3N9
    Canada

    Site Not Available

  • Vancouver Coastal Health Research Institute

    Vancouver 6173331, British Columbia 5909050 V5Z 3N9
    Canada

    Site Not Available

  • Sunnybrook Ophthalmology and Vision Services

    Toronto, Ontario M4N 3M5
    Canada

    Site Not Available

  • Vitreous Retina Macula Specialists of Toronto

    Toronto, Ontario M8X 2X3
    Canada

    Active - Recruiting

  • Sunnybrook Ophthalmology and Vision Services

    Toronto 6167865, Ontario 6093943 M4N 3M5
    Canada

    Site Not Available

  • Vitreous Retina Macula Specialists of Toronto

    Toronto 6167865, Ontario 6093943 M8X 2X3
    Canada

    Site Not Available

  • Cincinnati Eye Institute

    Cincinnati, Ohio 45242
    United States

    Site Not Available

  • Cincinnati Eye Institute

    Cincinnati 4508722, Ohio 5165418 45242
    United States

    Site Not Available

  • Retina Consultants of Texas

    Bellaire, Texas 77401
    United States

    Site Not Available

  • Retina Consultants of Texas

    Bellaire 4673353, Texas 4736286 77401
    United States

    Site Not Available

Map preview placeholder

Not the study for you?

Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.