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Age-Related Macular DegenerationPhase 1/2NCT05984927

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

A Phase 1/2 study testing NG101 AAV gene therapy in people with Age-Related Macular Degeneration, run by Elisigen, Inc. at 4 sites in 2 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial 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). Read more about this study

Who is it for?
Ages 50 to 89 years
Conditions the study is for
Age-Related Macular Degeneration
What is being tested?
NG101 AAV gene therapy
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
20 participants
Where
4 sitesin 2 countries
Study dates
8 Sep 2023to Jan 2030
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 50 to 89 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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
Show all 22Show fewer
  • 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
  • History of any known inherited retinal disease
  • Currently taking any anticoagulant therapy, which is deemed medically necessary and cannot be permanently stopped at least 2 weeks prior to NG101 injection, excluding prophylactic low-dose aspirin therapy
  • Any underlying systemic diseases as unstable or severe cardiovascular, cerebrovascular, dementia or neurodegenerative diseases of a level that prevents adequate evaluation of the subject during the study, active malignancy or currently undergoing treatment for active malignancy at Screening or a history of malignancy that precludes completion of this 260 week study, and immunocompromised conditions and/or need for immunosuppressive therapy
  • Active hepatitis B or C
  • History of human immunodeficiency virus (HIV), active tuberculosis, and/or syphilis
  • Any significant illness that would preclude study compliance and follow-up
  • Subjects who, in the Investigator's opinion, lack the mental capacity to provide written informed consent for study participation

Trial locations and contacts

4 sites in 2 countries.

4 sites in 2 countries

Canada3 sites

  • Sunnybrook Ophthalmology and Vision Services

    Toronto, Ontario, M4N 3M5

  • Vitreous Retina Macula Specialists of Toronto

    Toronto, Ontario, M8X 2X3

  • Vancouver Coastal Health Research Institute

    Vancouver, British Columbia, V5Z 3N9

Other trials in Canada

United States1 site

  • Cincinnati Eye Institute

    Cincinnati, Ohio, 45242

Other trials in United States

About this study

Official title: A Phase 1/2a Open-label Study to Evaluate Safety, Tolerability and Preliminary Efficacy of NG101 AAV Gene Therapy in Subjects With Wet Age-Related Macular Degeneration

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).

Read the detailed 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.

Being tested
NG101 AAV gene therapy
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
Medical subject headings
Macular Degeneration, Wet Macular Degeneration, Choroidal Neovascularization, Retinal Degeneration, Eye Diseases, Retinal Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • NG101 AAV gene therapyGenetic

    Also called NG101

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

Study groups in NCT05984927
Study groupRoleWhat they receive
NG101 Gene Therapy Group 1ExperimentalSingle subretinal injection of 1x10^9 vector genomes of NG101 AAV gene therapy
Receives: Genetic: NG101 AAV gene therapy
NG101 Gene Therapy Group 2ExperimentalSingle subretinal injection of 3x10^9 vector genomes of NG101 AAV gene therapy
Receives: Genetic: NG101 AAV gene therapy
NG101 Gene Therapy Group 3ExperimentalSingle subretinal injection of 8x10^9 vector genomes of NG101 AAV gene therapy
Receives: Genetic: NG101 AAV gene therapy

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Adverse Events

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

    Measured: From Day 0 (pre-treatment) through Week 24

Secondary outcomes (9)

  1. Ocular and Non -Ocular Adverse Events after week 24

    Incidence and severity of ocular and non-ocular AEs from Day 0 (pre-treatment) through Weeks 52 and Week 260 for each cohort

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

  2. Systemic Immunogenic Response

    Change in concentration of Anti-NG101 Transgene protein (aflibercept) antibodies, Anti-AAV8 Neutralizing antibodies, and Anti-AAV8 Antibodies in serum samples

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

  3. Systemic Immunogenic Response

    Change in concentration of expressed aflibercept protein in serum samples

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

  4. Systemic Immunogenic Response

    Change in AAV vector (NG101) genome copies as measured by qPCR (quantitative polymerase chain reaction) in blood samples

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

  5. Systemic Immunogenic Response

    Change in concentration of expressed aflibercept protein in vitreous samples

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

  6. Signs of CNV (choroidal neovascularization) Activity

    Change of 1 or more signs of CNV activity assessed by OCT (Optical coherence tomography)

    Measured: From Day -7 to Weeks 8, 24, 52, and 104

  7. Central Subfield Thickness (CST)

    Change in CST assessed with OCT

    Measured: From Day -7 to Weeks 24, 52, 104, 156, 208, and 260

  8. Best Corrected Visual Acuity (BCVA)

    Change in BCVA assessed with Early Treatment Diabetic Retinopathy Study (ETDRS) scale

    Measured: From Day -7 to Weeks 24, 52, 104, 156, 208 and 260

  9. Cumulative Number of Rescue Therapy Injections

    The cumulative number of rescue therapy injections per subject to maintain CNV control in the Study Eye

    Measured: Through Weeks 24, 52, 104, 156, 208, and 260

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted9 Aug 2023
  2. Study start8 Sep 2023
  3. Primary completion27 May 2026
  4. Study completionJan 2030 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Model description
Dose Escalation Study with 3 dose cohorts
Masking
None (open label)
Enrolment
20 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05984927
Sponsor's protocol ID
NG101WA-01
Status last verified
Sep 2026
First submitted
24 Jul 2023
Last updated
10 Sep 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing NG101 AAV gene therapy in people with Age-Related Macular Degeneration, run by Elisigen, Inc. at 4 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 8 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 10 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in Canada, United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Adverse Events (time frame: From Day 0 (pre-treatment) through Week 24). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05984927.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Sep 2026; AzTrials last refreshed its copy on 26 Oct 2026.

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