EXG102-031 for Wet Age-Related Macular Degeneration

Not currently recruiting at 1 trial location

Overseen By Senior Director, Clinical Development

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Exegenesis Bio

Must be taking: Anti-VEGF

Disqualifiers: Retinal detachment, Glaucoma, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 1

Duration: 1 day

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called EXG102-031, an experimental injection for individuals with wet age-related macular degeneration (nAMD). In this condition, abnormal blood vessels grow and leak in the eye, causing vision problems. The trial aims to determine if EXG102-031 can safely reduce this abnormal vessel growth and leakage. Suitable candidates for the trial include those diagnosed with nAMD, with an active lesion in one eye, and who have undergone cataract surgery in that eye. Participants will receive a single injection in the eye, and the trial will assess its safety for broader use. As a Phase 1 trial, participants will be among the first to receive this new treatment, aiding researchers in understanding its effects in people.

Do I have to stop taking my current medications for the trial?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that EXG102-031 is likely to be safe for humans?

Research shows that EXG102-031 is undergoing tests to determine its safety for people. The treatment is in the early testing stages, so researchers are still assessing its safety. In earlier animal studies, EXG102-031 appeared safe and effective for treating wet age-related macular degeneration. However, results in animals don't always predict human outcomes.

Currently, no solid information from human studies confirms its safety. This trial is a key step to determine how well people tolerate the treatment and identify any side effects. As the trial progresses, more information will clarify the safety of EXG102-031 in humans. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

EXG102-031 is unique because it involves a new delivery method—a single subretinal injection directly into the study eye—potentially allowing for more targeted and effective treatment of wet age-related macular degeneration (AMD). Unlike the standard treatments that often require repeated eye injections, this approach aims to simplify the treatment process while maximizing its impact. Researchers are excited about EXG102-031 because it could offer a more efficient way to manage this condition, reducing the burden on patients and improving their quality of life.

What evidence suggests that EXG102-031 might be an effective treatment for wet age-related macular degeneration?

Research shows that EXG102-031 is designed to stop the growth of unusual blood vessels in the eye, a major issue in wet age-related macular degeneration (nAMD). This could help reduce fluid leakage that harms vision over time. Although limited information exists on its effectiveness in improving vision, the treatment aims to address the main cause of vision problems in nAMD by preventing these harmful vessels from forming. Early studies, including this trial where participants receive a single subretinal injection of EXG102-031, focus on ensuring the treatment's safety, paving the way for future research on its potential to improve vision. ^{1 6 7 8 9}

Who Is on the Research Team?

Arshad Khanani, MD

Principal Investigator

Sierra Eye Associates

Are You a Good Fit for This Trial?

This trial is for men and women over 50 with wet age-related macular degeneration (nAMD) who've had some response to previous treatments. They must have a certain level of vision clarity in the affected eye, which should be pseudophakic (after cataract surgery). Participants need to understand the study and consent to join. Those with severe scarring, recent eye surgeries, other major eye diseases, or untreated glaucoma can't join.

Inclusion Criteria

An ETDRS BCVA letter scores between 73 and 19 letters in the study eye

I responded to anti-VEGF treatment during screening.

I am 50 years old or older.

Exclusion Criteria

I had eye surgery in the affected eye within the last 3 months.

My eye has scarring or damage in the central vision area.

I currently have an eye infection.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single subretinal injection of EXG102-031 in the study eye

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

52 weeks

Regular visits as per study protocol

What Are the Treatments Tested in This Trial?

Interventions

• EXG102-031

Trial Overview

EXG102-031 is being tested for safety in patients with nAMD. The drug aims to block abnormal blood vessel growth in the eye that causes vision loss by leaking fluid into the macula. This phase of testing will determine if it's safe before checking its effectiveness on improving vision.

How Is the Trial Designed?

Group I: EXG102-031 Experimental Treatment 1 Intervention

Each participant will receive a single subretinal injection of EXG102-031 in the study eye. Participants will be enrolled into one of four dosage groups sequentially, and the dose administered will be determined based on which study group the participant is enrolled into.

EXG102-031 is already approved in United States for the following indications:

Approved in United States as EXG102-031 for:

Who Is Running the Clinical Trial?

Exegenesis Bio

Lead Sponsor

Trials

2

Recruited

20+

Published Research Related to This Trial

In a study of 655 eyes with wet age-related macular degeneration (AMD) receiving anti-VEGF therapy, visual acuity improvements after 12 months were similar across all groups, regardless of lesion activity level, indicating that persistent lesion activity does not necessarily lead to worse visual outcomes.

The number of injections required increased slightly with more active lesions, but the overall visual improvements remained consistent, suggesting that treatment effectiveness is maintained even in cases of higher lesion activity.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Outcomes of persistently active neovascular age-related macular degeneration treated with VEGF inhibitors: observational study data. Barthelmes, D., Walton, R., Campain, AE., et al.[2015]

In a study of 65 patients with newly diagnosed active neovascular age-related macular degeneration, the leakage area significantly increased during the pretreatment period, indicating rapid progression of the condition, but visual acuity did not deteriorate during this time.

Despite the increase in leakage area, there was no correlation between this progression and visual outcomes at 3 months or 1 year, suggesting that short-term changes in leakage may not predict long-term visual prognosis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Short-term progression of wet AMD and correlation with 1-year treatment results. Munk, M., Kiss, C., Sulzbacher, F., et al.[2012]

In a study of 20 patients with wet Age-Related Macular Degeneration treated with 3 intravitreal injections of Bevacizumab (Avastin), 30% of eyes showed improved visual acuity, while 55% remained stable, indicating that while many patients did not experience a change in vision, a significant portion reported improved quality of vision.

The treatment led to favorable changes in foveolar thickness and total macular volume in 66.66% and 77.77% of eyes, respectively, suggesting that Avastin effectively reduces retinal swelling, even if visual acuity does not always improve.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Avastin in age related macular degeneration]. Tălu, S., Demea, S., Demea, H., et al.[2015]

In a study of 57 participants with wet age-related macular degeneration (AMD), those who received dietary supplements as recommended by the AREDS2 showed a trend towards fewer anti-vascular endothelial growth factor injections over 3 years (21.89 vs. 26.00), although this difference was not statistically significant.

No significant differences were found in final visual acuity or central macular thickness between the supplement group and the control group, suggesting that while dietary supplementation may influence treatment frequency, it does not significantly impact overall visual outcomes in this study.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

AREDS2 Supplementation in Patients with Wet Age-Related Macular Degeneration. Siag, N., Moshkovsky, R., Golan, N., et al.[2023]

In a study of 130 eyes from 115 patients with age-related macular degeneration, those who received annual intravitreal anti-vascular endothelial growth factor injections maintained stable visual acuity over an average follow-up of 11.1 years.

Patients who did not receive at least one injection each year experienced a significant decline in visual acuity, highlighting the importance of continuous treatment for preserving vision in this condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

TEN-YEAR FOLLOW-UP OF PATIENTS WITH EXUDATIVE AGE-RELATED MACULAR DEGENERATION TREATED WITH INTRAVITREAL ANTI-VASCULAR ENDOTHELIAL GROWTH FACTOR INJECTIONS. Starr, MR., Kung, FF., Mejia, CA., et al.[2021]

Citations

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clinicaltrials.gov

clinicaltrials.gov/study/NCT05903794

A Study of EXG102-031 in Patients With wAMD (Everest)

An Open-label, Dose-escalation Study to Evaluate the Safety and Tolerability of Gene Therapy With EXG102-031 in Participants With Neovascular Age-related ...

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clinicaltrials.gov

clinicaltrials.gov/study/NCT06817343

A Long-term Follow up Study of EXG102-031 in Patients ...

Before EXG102-031 can be tested for its efficacy (if it makes vision better), it must be tested to see if it is safely tolerated to confirm it can continue to ...

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withpower.com

withpower.com/trial/exg102-031-for-wet-age-related-macular-degeneration-d37fd

EXG102-031 for Wet Age-Related Macular Degeneration

What data supports the effectiveness of the drug EXG102-031 for ... Short-term progression of wet AMD and correlation with 1-year treatment results.

4.

scge.mcw.edu

scge.mcw.edu/platform/data/clinicalTrials/report/NCT05903794

A Study of EXG102-031 in Patients With wAMD (Everest)

Before EXG102-031 can be tested for its efficacy (if it makes vision better), it must be tested to see if it is safely tolerated to confirm it can continue to ...

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trial.medpath.com

trial.medpath.com/clinical-trial/f0c31f0779b7365f/gene-therapy-exg102-031-neovascular-amd

A Long-term Follow Up Study of EXG102-031 in Patients with ...

EXG102-031 was made to block the extra vessel formation which would lead to less leakage affecting the vision. Before EXG102-031 can be tested for its efficacy ...

6.

clinicaltrials.gov

clinicaltrials.gov/study/NCT06183814

A Study of EXG102-031 in Participants With wAMD

This Open-label, Dose-escalation Phase I/IIa Study is designed to evaluate the safety and efficacy of EXG102-031 intraocular Injection in participants with wet( ...

7.

retinalphysician.com

retinalphysician.com/issues/2024/april/gene-therapy-for-wet-age-related-macular-degeneration/

Gene Therapy for Wet Age-related Macular Degeneration

EXG102-031 is undergoing phase 1 trials to determine its safety and tolerability profile. Exegenesis Bio aims to enroll 6 participants for ...

8.

ctv.veeva.com

ctv.veeva.com/study/a-long-term-follow-up-study-of-exg102-031-in-patients-with-wamd-everest-ltfu

A Long-term Follow Up Study of EXG102-031 in Patients with ...

This study is designed to fulfill the long-term safety monitoring of EXG102-031. Participants that enroll in this long-term follow-up study have ...

9.

bioworld.com

bioworld.com/articles/697209-exg-102-031-demonstrates-preclinical-safety-and-efficacy-in-models-of-neovascular-amd

EXG-102-031 demonstrates preclinical safety and efficacy ...

EXG-102-031 demonstrates preclinical safety and efficacy in models of neovascular AMD. Fundus image of eye with age-related macular degeneration ...